Hemophilia ANCT02476942
A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment
An observational study of Hemophilia A, run by Hoffmann-La Roche at 35 sites in 12 countries.
Trial summary
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Bypassing Agents and FVIII Replacement
- How many people
- 221 participants
- Where
- 35 sitesin 12 countries
- Study dates
- 26 May 2015to 31 Mar 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.
Who may be able to join
- Cohort A: Patients greater than or equal to (>/=) 12 years of age at time of informed consent
- Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/= 5 Bethesda units [BU])
- Cohort B: Pediatric patients less than (<) 12 years of age
- Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/=5 BU)
- Cohort C: Patients >/=12 years of age
- Cohort C: Diagnosis of congenital hemophilia A and FVIII activity <1 percent (%)
- Cohort C: No prior history of a positive inhibitor against FVIII
Who cannot join
- Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study)
- Bleeding disorder other than congenital hemophilia A
- Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor
- Previous or concomitant thromboembolic disease
- Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL)
Trial locations and contacts
35 sites in 12 countries.
35 sites in 12 countries
United States10 sites
Winship Cancer Institute
Atlanta, Georgia, 30322
University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045
Children's Hospital of Michigan; Pediatrics
Detroit, Michigan, 48201
Tulane Uni Health Sciences Center
New Orleans, Louisiana, 70112-2699
Cornell Univ Medical College; Hematology-Oncolog
New York, New York, 10021
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science Uni ; Dept of Pediatrics
Portland, Oregon, 97201
Santa Monica Oncology Center
Santa Monica, California, 90403
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, 98104
Georgetown Uni Medical Center; Lombardi Cancer Center
Washington D.C., District of Columbia, 20007
Japan6 sites
Nagoya University Hospital
Aichi, 466-8560
Hyogo College of Medicine Hospital
Hyōgo, 663-8501
St. Marianna University School of Medicine Hospital
Kanagawa, 216-8511
Hospital of the University of Occupational and Environmental Health,Japan
Kitakyushu-shi, 807-8556
Nara Medical University Hospital
Nara, 634-8521
Tokyo Medical University Hospital
Tokyo, 160-0023
Poland4 sites
Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii
Gdansk, 80-952
SPSK Nr1 Klinika Hematoo&Transpl.Szpiku
Lublin, 20-081
ALVAMED Lekarskie Gabinety Specjalistyczne
Poznan, 61-828
Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych
Warsaw, 02-776
China3 sites
Peking Union Medical College Hospital
Beijing, 100730
Nanfang Hospital, Southern Medical University
Guangzhou, 510515
Tianjin Institute of Hematology & Blood Diseases Hospital
Tianjin, 300020
Spain3 sites
Hospital Universitario la Paz; Servicio de Hematologia
Madrid, 28046
Hospital Universitario Virgen del Rocio; Servicio de Hematologia
Seville, 41013
Hospital Universitario la Fe; Servicio de Hematologia
Valencia, 46026
Australia2 sites
Royal Prince Alfred Hospital; Haematology
Camperdown, New South Wales, 2050
The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit
Melbourne, Victoria, 3004
Italy2 sites
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
Germany1 site
Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)
Bonn, 53127
South Africa1 site
Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center
Johannesburg, 2193
About this study
Official title: A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
- Conditions
- Hemophilia A
- Being tested
- Bypassing Agents, FVIII Replacement
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bypassing AgentsDrug
Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
FVIII ReplacementDrug
Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A: Adults and Adolescents with FVIII Inhibitors | Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. Receives: Drug: Bypassing Agents | |
| Cohort B: Children with FVIII Inhibitors | Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. | |
| Cohort C: Adults and Adolescents without FVIII Inhibitors | Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed. Receives: Drug: FVIII Replacement |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Bleeds
Measured: Approximately 6 months (from Baseline until study completion)
Secondary outcomes (3)
European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients
Measured: Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients
Measured: Approximately 6 months (every 4 weeks from Baseline until study completion)
Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score
Measured: Approximately 6 months (every 4 weeks from Baseline until study completion)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2015 (estimated)
- Study start26 May 2015
- Primary completion31 Mar 2017
- Study completion31 Mar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 221 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
- NCT02476942
- Sponsor's protocol ID
- BH29768
- Status last verified
- May 2017
- First submitted
- 3 Jun 2015
- Last updated
- 30 May 2017
- Results posted
- No
Publications
Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9.
PubMed 32776489
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment
- ClinicalTrials.gov ID
- NCT02476942
- Acronym / sponsor ID
- BH29768
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 221 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 26 May 2015 (actual)
- Primary completion
- 31 Mar 2017 (actual)
- Study completion
- 31 Mar 2017 (actual)
- First posted
- 22 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A: Adults and Adolescents with FVIII Inhibitors | – | Drug: Bypassing AgentsAdults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. |
| Cohort B: Children with FVIII Inhibitors | – | –Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed. |
| Cohort C: Adults and Adolescents without FVIII Inhibitors | – | Drug: FVIII ReplacementAdults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.
Key inclusion criteria (7 listed)
- Cohort A: Patients greater than or equal to (>/=) 12 years of age at time of informed consent
- Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/= 5 Bethesda units [BU])
- Cohort B: Pediatric patients less than (<) 12 years of age
- Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/=5 BU)
- Cohort C: Patients >/=12 years of age
Key exclusion criteria (5 listed)
- Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study)
- Bleeding disorder other than congenital hemophilia A
- Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor
- Previous or concomitant thromboembolic disease
- Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL)
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 |
|---|---|---|
| Bypassing AgentsEpisodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions. | Drug | Cohort A: Adults and Adolescents with FVIII Inhibitors |
| FVIII ReplacementEpisodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions. | Drug | Cohort C: Adults and Adolescents without FVIII Inhibitors |
Primary outcome
Number of Bleeds
Time frame: Approximately 6 months (from Baseline until study completion)
3 secondary outcomes
European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients
Time frame: Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients
Time frame: Approximately 6 months (every 4 weeks from Baseline until study completion)
Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score
Time frame: Approximately 6 months (every 4 weeks from Baseline until study completion)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2017.
- Derived from this study: PubMed 32776489Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9.
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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
35 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Hoffmann-La Roche at 35 sites in 12 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 7 inclusion and 5 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 30 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States, Japan, Poland, China, Spain and 7 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Bleeds (time frame: Approximately 6 months (from Baseline until study completion)). 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 NCT02476942, 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 30 May 2017.