Hemophilia APhase 3STASEYNCT03191799
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
A Phase 3 study testing Emicizumab in people with Hemophilia A, run by Hoffmann-La Roche at 72 sites in 24 countries.
Trial summary
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Emicizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 195 participants
- Where
- 72 sitesin 24 countries
- Study dates
- 5 Sep 2017to 19 Nov 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- As per investigator's judgement, a willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an electronic device or paper
- Aged 12 years or older at the time of informed consent
- Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII
- Documented treatment with bypassing agents or FVIII concentrates in the last 6 months (on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day before starting emicizumab
- Adequate hematologic, hepatic, and renal function
- For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use a highly effective contraceptive method with a failure rate of <1% per year during the treatment period and for at least five elimination half-lives (24 weeks) after the last dose of emicizumab
Who cannot join
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy (prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to enrollment). Patients receiving ITI therapy will be eligible following the completion of a 72-hour washout period prior to the first emicizumab administration
- History of illicit drug or alcohol abuse within 12 months prior to screening, as per the investigator's judgment
- High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), as per the investigator's judgment
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (e.g., certain autoimmune diseases) that may increase the risk of bleeding or thrombosis
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- History of clinically significant hypersensitivity reaction associated with monoclonal antibody therapies or components of the emicizumab injection
- Known human immunodeficiency virus (HIV) infection with CD4 count <200 cells/μL within 6 months prior to screening
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of antiretroviral therapy
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient's safe participation in and completion of the study or interpretation of the study results
- Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within five half-lives of last drug administration; A non-hemophilia-related investigational drug within last 30 days or five half-lives, whichever is shorter; or, Any concurrent investigational drug.
- Pregnancy or lactation, or intent to become pregnant during the study
- Positive serum pregnancy test result within 7 days prior to initiation of emicizumab (females only)
Trial locations and contacts
72 sites in 24 countries.
72 sites in 24 countries
Italy9 sites
AOU Policlinico S. Orsola Malpighi; U.O. Angiologia e Malattie della Coagulazione Marino Golinelli
Bologna, Emilia-Romagna, 40138
AOU Careggi; SOD Malattie Emorragiche
Florence, Tuscany, 50134
IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"
Milan, Lombardy, 20122
AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia
Naples, Campania, 80131
Azienda Ospedaliera di Padova; Centro Emofilia
Padova, Veneto, 35128
Azienda Uni Ria Policlinico P. Giaccone ; Divisione Di Ematologia E Trapianto
Palermo, Sicily, 90127
AOU di Parma; Dip Emergenza-Urgenza Centro Riferimento Regionale per l'emofilia
Parma, Emilia-Romagna, 43126
Universita' Degli Studi La Sapienza-Ist.Di Ematologia; Dip Biot Cel e Ematol
Rome, Lazio, 00161
AO Città Salute e Scienza D-Osp S. G. Battista Molinette; SSCVD Malattie Tromboti e Emorragiche
Turin, Piedmont, 10126
India7 sites
St. John's Medical College & Hospital; Department of Medicine
Bengaluru, Karnataka, 560034
Nil Ratan Sircar Medical College and Hospital; Department of Hematology
Kolkata, West Bengal, 700014
King Edward Memorial Hospital and Seth G.S. Medical College; Department of Hematology
Mumbai, Maharashtra, 400012
Fortis Hospitals Limited
Mumbai, Maharashtra, 400078
Sahyadri Speciality Hospital; Hematology & Bone Marrow Unit
Pune, Maharashtra, 411004
Grant Medical Foundation, Ruby Hall Clinic
Pune, 411001
Christian Medical College & Hospital; Department of Haematology
Vellore, Maharashtra, 632004
Germany6 sites
Vivantes Klinikum im Friedrichshain - Landsberger Allee, Angiologie und Hämostaseologie
Berlin, 10249
Universitätsklinikum Bonn; Institut für Experimentelle Hämatologie und Transfusionsmedizin
Bonn, 53127
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin; Johann Wolfgang Goethe-Univers.
Frankfurt/M., 60590
Universitätsklinikum Leipzig, Innere Medizin, Neurologie, Dermatologie; Zentrum für Hämostaseologie
Leipzig, 04103
Hämophilie-Zentrum Rhein Main GmbH
Mörfelden-Walldorf, 64546
Klinikum der Universität München, Campus Innenstadt; Hämostaseologische Ambulanz/Hämophiliezentrum
München, 80336
Russia5 sites
Regional State Budgetary Institution of Healthcare "Regional Cinilcal Hospital"; Pulmonology
Barnaul, Altayskiy Kray, 656024
Morozov Children's Municipal Clinical Hospital. Haematology department
Moscow, 119049
"Hematological Scientific Center
Moscow, 125167
City Outpatient Clinic #37, City Hemophilia Treatment Center
Saint Petersburg, 191186
Samara State Medical University
Samara, 443099
Spain5 sites
Hospital Universitario Vall de Hebron; Unidad de Hemofília
Barcelona, 08035
Hospital Universitario la Paz; Servicio de Hematologia
Madrid, 28046
Hospital Regional Universitario Carlos Haya; Servicio de Hematologia
Málaga, 29010
Hospital Universitario Virgen del Rocio; Servicio de Hematologia
Seville, 41013
Hospital Universitario la Fe; Servicio de Hematologia
Valencia, 46026
United Kingdom5 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH
The Royal London Hospital
London, E1 2ES
Royal Free Hospital
London, NW3 2QS
Royal Victoria Infirmary; Non-Malignant Haematology Research
Newcastle upon Tyne, NE1 4LP
Royal Hallamshire Hospital Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, S10 2JF
Brazil4 sites
Hospital das Clinicas - UNICAMP
Campinas, São Paulo, 13083-888
Centro de Hematologia E Hemoterapia Do Parana - Hemepar
Curitiba, Paraná, 80045-145
Faculdade de Medicina de Ribeirão Preto - Universidade de Sao Paulo
Ribeirão Preto, São Paulo, 14051-140
Instituto Estadual de Hema
Rio de Janeiro, Rio de Janeiro, 20211-030
Canada4 sites
Kaye Edmonton Clinic
Edmonton, Alberta, T6G 2V2
McMaster University Health Sciences Center
Hamilton, Ontario, L8N 3Z5
St. Michael's Hospital
Toronto, Ontario, M5B 1W8
CancerCare Manitoba; Neuro-Oncology
Winnipeg, Manitoba, R2H 2A6
Australia3 sites
Royal Prince Alfred Hospital; Haematology
Camperdown, New South Wales, 2050
Alfred Hospital
Melbourne, Victoria, 3004
Fiona Stanley Hospital
Murdoch, Western Australia, 6150
Hungary3 sites
Magyar Honvédség Egészségügyi Központ; Országos Haemophilia Központ
Budapest, 1134
Országos Vérellátó Szolgálat - Győri Regionális Vérellátó Központ
Győr, 9023
University of Pecs, I st Dept of Internal Medicine
Pécs, 7624
Mexico3 sites
Hospital General de México
Distrito Federal, Mexico CITY (federal District), 06726
CENTRO MEDICO NACIONAL SIGLO XXI; Banco de Sangre
Mexico City, Mexico CITY (federal District), 06720
Hospital de Especialidades Centro Medico Nacional La Raza; Haematology
Mexico City, 02990
Portugal3 sites
Hospital Geral; Servico de Imuno-Hemoterapia
Coimbra, 3041-853
Hospital de Santa Maria; Servico de Imuno-Hemoterapia; Servico de Imuno-Hemoterapia
Lisbon, 1649-035
Hospital de Sao Joao; Servico de Imuno-Hemoterapia; Servico de Imuno-Hemoterapia
Porto, 4200-319
Colombia2 sites
Centro de Atención e Investigación Médica CAIMED S.A.S
Localidad Puente Aranda, 111611
Hospital Pablo Tobon Uribe
Medellín, 050034
Netherlands2 sites
Erasmus MC / location Sophia Kinderziekenhuis
Rotterdam, 3015 GJ
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
Sweden2 sites
Sahlgrenska Universitetssjukhuset; Koagulationscentrum
Gothenburg, 413 45
Karolinska Universitetssjukhuset; Koagulationsmottagningen
Solna, 171 64
Finland1 site
Helsingin yliopistollinen keskussairaala, Meilahden sairaala
Helsinki, 00290
Israel1 site
Sheba Medical Center - National Hemophilia Center
Tel HaShomer, 5262100
Panama1 site
Complejo Hospitalario Arnulfo Arias Madrid; Servicio de Hematología
Panama City, 0824
Poland1 site
Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
Warsaw, 02-776
Romania1 site
Louis Turcanu Emergency Clinical Hospital for Children; First Pediatric Clinic
Timișoara, 300011
Saudi Arabia1 site
King Faisal Specialist Hospital & Research Centre; Oncology
Riyadh, 11211
Switzerland1 site
Inselspital Bern; Hämatologie und Hämatologisches Zentrallabor
Bern, 3010
About this study
Official title: A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Approximately 200 participants, aged 12 or older, will be enrolled in this study and are expected to be enrolled at approximately 85 sites globally. Participants will receive an initial weekly dose of prophylactic emicizumab subcutaneously for 4 weeks, followed by a weekly maintenance dose subcutaneously for the remainder of the 2-year treatment period.
- Conditions
- Hemophilia A
- Being tested
- Emicizumab
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmicizumabDrug
Also called Hemlibra, RO5534262, ACE910
Initial dosing will be 3 mg/kg/week subcutaneously for 4 weeks; Maintenance dosing will follow at 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period
| Study group | Role | What they receive |
|---|---|---|
| 1.5 mg/kg Emicizumab QW | Experimental | Participants will receive initial weekly doses of prophylactic emicizumab subcutaneously for 4 weeks, followed by maintenance doses consisting of half the initial dose, administered subcutaneously for the remainder of the 2-year treatment period Receives: Drug: Emicizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale
Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. aPCC = activated prothrombin complex concentrate
Measured: From Baseline until study completion (up to 2 years)
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs
Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. The AE rate per 100 patient-years was computed as follows: AE Rate = (Number of AEs observed/ Total patient-years at risk)*100. Total patient-years at risk is the sum over all patients of the time intervals (in years) between start of study therapy (study day 1) and the end of follow up.
Measured: From Baseline until study completion (up to 2 years)
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline
The World Health Organization (WHO) toxicity grading scale was used for determining the severity of laboratory abnormalities (i.e., test results outside of the reference range) for hematology and biochemistry parameters; Grade 0 is normal and Grades 1 to 4 represent worsening levels of the parameter outside of the normal range in the specified direction of the abnormality (high and low are above and below the range, respectively). Not every laboratory abnormality qualified as an adverse event (AE). A laboratory test result was reported as an AE if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment; resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. Baseline was defined as the last available assessment prior to first receipt of study drug. Abs = absolute count; SGOT/AST = aspartate aminotransferase; SGPT/ALT = alanine aminotransferase
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Body Temperature at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Systolic Blood Pressure at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Pulse Rate at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Respiratory Rate at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Change From Baseline in Body Weight at Specified Timepoints
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Measured: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Secondary outcomes (39)
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
The number of bleeds over the efficacy period was analyzed as an ABR using a negative binomial regression model. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all bleed types, bleeds due to surgery/procedure were excluded. Bleeds occurring after dose up-titration have been excluded.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
The number of bleeds over the efficacy period was calculated as: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all types, bleeds due to surgery/procedure and bleeds occurring after dose up-titration were excluded.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
The number of bleeds over the efficacy period was calculated as: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all types, bleeds due to surgery/procedure and bleeds occurring after dose up-titration were excluded.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds
Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds
All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds
Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds
The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds
The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds
The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Measured: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. The Total Score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. The Total Score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflective of better quality of life. An improvement larger than the responder threshold was defined as a decrease from baseline of at least 7 points in the Haem-A-QoL Total score.
Measured: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life. An improvement larger than the responder threshold was defined as a decrease from baseline of at least 10 points in the Haem-A-QoL Physical Health domain score.
Measured: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants
The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. The Total Score is derived from the scores for all domains and scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. The Total Score is derived from the scores for all domains and scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life. An improvement larger than the responder threshold was defined as a decrease of at least 5 points in the Haemo-QoL-SF Total score.
Measured: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life. An improvement larger than the responder threshold was defined as a decrease of at least 10 points in the Haemo-QoL-SF Physical Health domain score.
Measured: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants
The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints
The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The index utility score is based on the participant's assessment of their health on five scales: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. It ranges from -0.224 to 1, with 0 corresponding to death and 1 corresponding to full health; negative values correspond to health states worse than death. Higher scores indicate better quality of life.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints
The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The visual analogue scale (VAS) ranges from 0 to 100 points, on which the participant self-assesses their current health status; 0 being the worst health you can imagine and 100 being the best health you can imagine. Higher scores are reflective of better health.
Measured: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire
The EmiPref questionnaire asked participants to specify the treatment they would prefer to continue to receive after receiving treatment with their previous episodic or prophylactic regimen and subcutaneous (SC) emicizumab, or if they had no preference. The 95% confidence intervals were calculated for one sample binomial using the Pearson-Clopper method.
Measured: Month 3
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline
'Total ADA Negative' is the sum of all subjects who tested negative for ADA in the 2 following categories: 'ADA Negative', those who are pre-dose ADA negative or are missing pre-dose ADA data and who have all negative post-dose ADA results; and 'ADA Negative (Treatment Unaffected)', a subset who are pre-dose ADA positive but do not have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement. 'Total ADA Positive' is the sum of all subjects who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Boosted)', those who are pre-dose ADA positive and have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement; and 'ADA Positive (Treatment Induced)', those who are pre-dose ADA negative or missing data and who have at least one post-dose ADA positive sample.
Measured: Baseline (Week 1), Week 5, and at 3, 6, 9, 12, and 18 months, and at early termination/study completion (up to 24 months)
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints
The trough concentration is a measure of the plasma concentration of a study drug at the end of of the dosage interval.
Measured: Pre-dose at Weeks 2, 3, and 5, Months 3, 6, 12, and 18, and at treatment discontinuation (up to 2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2017
- Study start5 Sep 2017
- Primary completion19 Nov 2020
- Study completion19 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 195 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03191799
- Sponsor's protocol ID
- MO39129
- Other IDs
- 2016-004366-25, 2016
- Acronym
- STASEY
- Status last verified
- May 2021
- First submitted
- 15 Jun 2017
- Last updated
- 11 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Castaman G, Peyvandi F, Kremer Hovinga JA, Schutgens REG, Robson S, Moreno K, Jimenez-Yuste V. Surgical Experience from the STASEY Study of Emicizumab Prophylaxis in People with Hemophilia A with Factor VIII Inhibitors. TH Open. 2024 Jan 12;8(1):e42-e54. doi: 10.1055/s-0043-1777766. eCollection 2024 Jan.
PubMed 38222041
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
- ClinicalTrials.gov ID
- NCT03191799
- Acronym / sponsor ID
- STASEY / MO39129
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 195 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 5 Sep 2017 (actual)
- Primary completion
- 19 Nov 2020 (actual)
- Study completion
- 19 Nov 2020 (actual)
- First posted
- 19 Jun 2017 (actual)
- Results
- Posted 11 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Approximately 200 participants, aged 12 or older, will be enrolled in this study and are expected to be enrolled at approximately 85 sites globally. Participants will receive an initial weekly dose of prophylactic emicizumab subcutaneously for 4 weeks, followed by a weekly maintenance dose subcutaneously for the remainder of the 2-year treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1.5 mg/kg Emicizumab QW | Experimental | Drug: EmicizumabParticipants will receive initial weekly doses of prophylactic emicizumab subcutaneously for 4 weeks, followed by maintenance doses consisting of half the initial dose, administered subcutaneously for the remainder of the 2-year treatment period |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- As per investigator's judgement, a willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an electronic device or paper
- Aged 12 years or older at the time of informed consent
- Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII
- Documented treatment with bypassing agents or FVIII concentrates in the last 6 months (on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day before starting emicizumab
- Adequate hematologic, hepatic, and renal function
Key exclusion criteria (13 listed)
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy (prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to enrollment). Patients receiving ITI therapy will be eligible following the completion of a 72-hour washout period prior to the first emicizumab administration
- History of illicit drug or alcohol abuse within 12 months prior to screening, as per the investigator's judgment
- High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), as per the investigator's judgment
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmicizumabAlso: Hemlibra, RO5534262, ACE910Initial dosing will be 3 mg/kg/week subcutaneously for 4 weeks; Maintenance dosing will follow at 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period | Drug | 1.5 mg/kg Emicizumab QW |
Primary outcome
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale
Time frame: From Baseline until study completion (up to 2 years)
Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. aPCC = activated prothrombin complex concentrate
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs
Time frame: From Baseline until study completion (up to 2 years)
Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. The AE rate per 100 patient-years was computed as follows: AE Rate = (Number of AEs observed/ Total patient-years at risk)*100. Total patient-years at risk is the sum over all patients of the time intervals (in years) between start of study therapy (study day 1) and the end of follow up.
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
The World Health Organization (WHO) toxicity grading scale was used for determining the severity of laboratory abnormalities (i.e., test results outside of the reference range) for hematology and biochemistry parameters; Grade 0 is normal and Grades 1 to 4 represent worsening levels of the parameter outside of the normal range in the specified direction of the abnormality (high and low are above and below the range, respectively). Not every laboratory abnormality qualified as an adverse event (AE). A laboratory test result was reported as an AE if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment; resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. Baseline was defined as the last available assessment prior to first receipt of study drug. Abs = absolute count; SGOT/AST = aspartate aminotransferase; SGPT/ALT = alanine aminotransferase
Change From Baseline in Body Temperature at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Change From Baseline in Systolic Blood Pressure at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Change From Baseline in Pulse Rate at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Change From Baseline in Respiratory Rate at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Change From Baseline in Body Weight at Specified Timepoints
Time frame: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
39 secondary outcomes
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds
Time frame: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints
Time frame: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints
Time frame: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints
Time frame: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints
Time frame: At 3, 6, 12, and 18 months
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints
Time frame: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire
Time frame: Month 3
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline
Time frame: Baseline (Week 1), Week 5, and at 3, 6, 9, 12, and 18 months, and at early termination/study completion (up to 24 months)
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints
Time frame: Pre-dose at Weeks 2, 3, and 5, Months 3, 6, 12, and 18, and at treatment discontinuation (up to 2 years)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38222041Castaman G, Peyvandi F, Kremer Hovinga JA, Schutgens REG, Robson S, Moreno K, Jimenez-Yuste V. Surgical Experience from the STASEY Study of Emicizumab Prophylaxis in People with Hemophilia A with Factor VIII Inhibitors. TH Open. 2024 Jan 12;8(1):e42-e54. doi: 10.1055/s-0043-1777766. eCollection 2024 Jan.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
72 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Emicizumab in people with Hemophilia A, run by Hoffmann-La Roche at 72 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 72 sites in Italy, India, Germany, Russia, Spain and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale (time frame: From Baseline until study completion (up to 2 years)). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03191799, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2021.