Hemophilia aNCT05605678
Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
An observational study of Hemophilia a and Hemophilia B, run by ApcinteX Ltd at 45 sites in 16 countries.
Trial summary
The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia a, Hemophilia B
- How many people
- 108 participants
- Where
- 45 sitesin 16 countries
- Study dates
- 9 Dec 2022to 29 Nov 2024
- 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with severe HemA with or without inhibitors, or moderately severe to severe HemB without inhibitors or HemB with inhibitors treated with SOC on-demand or prophylactic hemophilia therapy.
Who may be able to join
- Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age
- Who are capable of providing written informed consent (adolescent assent and parental/guardian consent when appropriate) for participation after reading the information and consent form and having the opportunity to discuss the study with the Investigator or their designee
- With historically documented severe HemA (defined as factor VIII [FVIII] less than (<) 0.01 International Units per milliliter (IU/mL) [<1 percent {%}]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX [FIX] <=0.02 IU/mL [<=2%]) without inhibitors. Participants must be currently included in a prophylactic treatment program or if undergoing an on-demand treatment regimen must have had >=6 documented acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months before enrollment OR: Historically documented HemB (defined as FIX <=0.05 IU/mL [<=5%]) with inhibitors with a historical or ongoing high titer inhibitor [>=5 Bethesda Units/mL] based on medical records or laboratory reports) and an ABR of >=6 in the 6 months before enrollment
- Who are able to use a diary to document bleeding events and associated treatment
Who cannot join
- With known thrombophilia
- With body weight greater than (>)150 kilogram (kg) or body mass index >40
- With known current inadequate hematologic function (eg, platelet count <100,000 per microliter [/mcL] and/or hemoglobin level <10 grams per deciliter [g/dL], <100 g/L), hepatic function (that is, total bilirubin >1.5*upper limit of normal [ULN] [excluding Gilbert syndrome], aspartate transferase and/or alanine aminotransferase levels >3*ULN; clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver) or renal function (that is, serum creatinine >2*ULN; based on medical records or available laboratory reports)
- With previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
- With history of intolerance to subcutaneous injections
- With known current uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mm Hg); diastolic blood pressure >100 mm Hg; based on medical records)
Show all 14Show fewer
- With active cancer or requires therapy for cancer, except for basal cell carcinoma
- With concurrent participation in an interventional clinical trial
- With current or planned use of emicizumab
- With prior, ongoing, or planned treatment with gene therapy for hemophilia
- With history of or other evidence of recent alcohol or drug abuse as determined by the Investigator or their designee (in the 12 months before enrollment)
- With known Human Immunodeficiency Virus (HIV) infection with CD4 count (or T-cell count) of <200 cells/mcL within 24 weeks before enrollment
- With current or planned treatment with anticoagulant or antiplatelet drugs
- With any other significant conditions or comorbidities that, in the opinion of the Investigator or their designee, would make the participant unsuitable for enrollment
Trial locations and contacts
45 sites in 16 countries.
45 sites in 16 countries
United Kingdom9 sites
Kent Canterbury Hospital
Canterbury, CT1 3NG
University Hospital of Wales
Cardiff, Cardiff, CF14 4XW
Glasgow Royal Infirmary
Glasgow, Glasgow, G4OSF
Barts and London School of Medicine and Dentistry
London, London, E1 2AT
Royal Free London NHS Foundation Trust
London, London, NW3 2QG
Imperial College Healthcare NHS Trust
London, London, W2 1NY
Central Manchester University Hospitals NHS Foundation Trust - Manchester Royal Infirmary
Manchester, M13 9WL
Royal Victoria Infirmary
Newcastle upon Tyne, Tyne and Wear, NE1 4LP
Southampton General Hospital
Southampton, Southampton, S016 6YD
France4 sites
Hôpital Bicêtre
Le Kremlin-Bicêtre, 94270
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
Lyon, Rhone, 69500
CHU Hotel Dieu
Nantes, 44000
Hopital Necker - Enfants Malades
Paris, Paris, 75015
Spain4 sites
Hospital Universitari Vall d'Hebron
Barcelona, 8035
Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil
Málaga, Malaga, 29010
Hospital Clínico Universitario Virgen de la Arrixaca
Murcia, Murcia, 30120
Hospital Universitario Miguel Servet
Zaragoza, Zaragoza, 50009
Taiwan4 sites
China Medical University Hospital
Taichung, Taichung, 40447
Chung Shan Medical University
Taichung, 40201
Taichung Veterans General Hospital
Taichung, 40705
National Taiwan University Hospital
Taipei, 100226
Canada3 sites
Hamilton Health Sciences Corporation
Hamilton, Hamilton, L8S 4K1
Hamilton Health Sciences Corporation
Hamilton, Ontario, L8S4K1
Unity Health Toronto
Toronto, Toronto, M5B 1W8
Germany3 sites
Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde
Berlin, State of Berlin, 10249
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, Saxony, 1307
University Hospital Frankfurt
Frankfurt am Main, Hesse, 60590
India3 sites
St. Johns Medical College
Bangalore, Bangalore, 560034
Christian Medical College & Hospital
Ludhiana, Punjab, 141008
K J Somaiya Super Speciality Hospital & Research Centre
Mumbai, Mumbai, 400022
United States2 sites
University of Michigan
Ann Arbor, Michigan, 48109
East Carolina univeristy
Greenville, South Carolina, 27834
Brazil2 sites
Fundacao de Desenvolvimento da Unicamp FUNCAMP
Campina, São Paulo, 13083-878
HEMOES - Centro de Hemoterapia e Hematologia do ES
Vitória, Vitória, 29047105
Italy2 sites
Azienda Ospedaliero-Universitaria Careggi
Florence, 50134
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, Milano, 20122
Poland2 sites
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, Podkarpackie Voivodeship, 35-302
Kl Hemat Now Krwi i Trans USK
Wroclaw, Wrocław, 50-367
South Africa2 sites
King Edward VIII Hospital
Durban, Durban, 4001
Phoenix Pharma (Pty) Ltd
Port Elizabeth, Eastern Cape, 6001
Turkey (Türkiye)2 sites
Ege University Hospital
Izmir, İzmir, 35100
Ege University Medical Faculty Pediatric Hospital
Izmir, 35100
Armenia1 site
Centre of Haematology named after prof. R. O. Yeolian
Yerevan, Yerevan, 0014
Australia1 site
Royal Prince Alfred Hospital
Camperdown, Camperdown, NSW 2050
About this study
Official title: A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Participants in the study may be eligible to enroll in future planned interventional studies to be conducted by Sponsor.
- Conditions
- Hemophilia a, Hemophilia B
- Being tested
- No Intervention
- Keywords
- Hemophilia, Prospective
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| All Eligible Participants | All Participants diagnosed with severe HemA with or without inhibitors, moderately severe to severe HemB, or HemB with inhibitors will be enrolled and continue to receive their usual hemophilia treatment regimen under SOC therapy. Bleeding episodes and treatment data will be collected during the prospective follow up period in a diary. No intervention will be administered as part of this study. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
Measured: Minimum 12 weeks
Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
Measured: Minimum 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2022
- Study start9 Dec 2022
- Primary completion29 Nov 2024
- Study completion29 Nov 2024
Why it stopped: Study terminated due to business and strategic decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia aStudies of the same condition, open and under way.
- 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
- Prospective
- Enrolment
- 108 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApcinteX Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Centessa Pharmaceuticals plc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05605678
- Sponsor's protocol ID
- AP-0105 (PRESent-5)
- Status last verified
- Feb 2025
- First submitted
- 31 Oct 2022
- Last updated
- 7 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There is no plan to share IPD due to the Sponsor's strategic and data-driven decision to discontinue the global clinical development of SerpinPC.
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
- ClinicalTrials.gov ID
- NCT05605678
- Acronym / sponsor ID
- AP-0105 (PRESent-5)
- Conditions
- Hemophilia a, Hemophilia B
- Keywords
- Hemophilia, Prospective
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study terminated due to business and strategic decision
- Enrolment
- 108 participants (actual)
- Lead sponsor
- ApcinteX LtdIndustry (pharma, biotech, devices)
- Collaborators
- Centessa Pharmaceuticals plc
- Study start
- 9 Dec 2022 (actual)
- Primary completion
- 29 Nov 2024 (actual)
- Study completion
- 29 Nov 2024 (actual)
- First posted
- 4 Nov 2022 (actual)
- 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.
The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Participants in the study may be eligible to enroll in future planned interventional studies to be conducted by Sponsor.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Eligible Participants | – | Other: No InterventionAll Participants diagnosed with severe HemA with or without inhibitors, moderately severe to severe HemB, or HemB with inhibitors will be enrolled and continue to receive their usual hemophilia treatment regimen under SOC therapy. Bleeding episodes and treatment data will be collected during the prospective follow up period in a diary. No intervention will be administered as part of this study. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with severe HemA with or without inhibitors, or moderately severe to severe HemB without inhibitors or HemB with inhibitors treated with SOC on-demand or prophylactic hemophilia therapy.
Key inclusion criteria (4 listed)
- Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age
- Who are capable of providing written informed consent (adolescent assent and parental/guardian consent when appropriate) for participation after reading the information and consent form and having the opportunity to discuss the study with the Investigator or their designee
- With historically documented severe HemA (defined as factor VIII [FVIII] less than (<) 0.01 International Units per milliliter (IU/mL) [<1 percent {%}]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX [FIX] <=0.02 IU/mL [<=2%]) without inhibitors. Participants must be currently included in a prophylactic treatment program or if undergoing an on-demand treatment regimen must have had >=6 documented acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months before enrollment OR: Historically documented HemB (defined as FIX <=0.05 IU/mL [<=5%]) with inhibitors with a historical or ongoing high titer inhibitor [>=5 Bethesda Units/mL] based on medical records or laboratory reports) and an ABR of >=6 in the 6 months before enrollment
- Who are able to use a diary to document bleeding events and associated treatment
Key exclusion criteria (14 listed)
- With known thrombophilia
- With body weight greater than (>)150 kilogram (kg) or body mass index >40
- With known current inadequate hematologic function (eg, platelet count <100,000 per microliter [/mcL] and/or hemoglobin level <10 grams per deciliter [g/dL], <100 g/L), hepatic function (that is, total bilirubin >1.5*upper limit of normal [ULN] [excluding Gilbert syndrome], aspartate transferase and/or alanine aminotransferase levels >3*ULN; clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver) or renal function (that is, serum creatinine >2*ULN; based on medical records or available laboratory reports)
- With previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
- With history of intolerance to subcutaneous injections
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 |
|---|---|---|
| No InterventionThis is a non-interventional study. | Other | All Eligible Participants |
Primary outcome
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
Time frame: Minimum 12 weeks
ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
Time frame: Minimum 12 weeks
Results and safety
The study was stopped early (reason given: Study terminated due to business and strategic decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2025.
- Individual participant dataSharing planned: No. There is no plan to share IPD due to the Sponsor's strategic and data-driven decision to discontinue the global clinical development of SerpinPC.
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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.
The record lists no contact. Use the official record to reach the sponsor.
45 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia a and Hemophilia B, run by ApcinteX Ltd at 45 sites in 16 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 4 inclusion and 14 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: stopped early, as of the sponsor's last update on 7 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United Kingdom, France, Spain, Taiwan, Canada and 11 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 was stopped early (reason given: Study terminated due to business and strategic decision). Results are not available in the source record.
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?
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds (time frame: Minimum 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Feb 2025.