Clinical trials: Hemophilia A and B, active
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or…
- Trial name
- BENEGENE-2 (NCT03861273)
- Testing
- PF-06838435/ fidanacogene elaparvovec
- Where
- 65 sites in United States, Australia and 14 more
- Volunteers
- 51 people
- Sponsor
- Pfizer
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Hemophilia B
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05630651
- Testing
- ZS801
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Haemophilia A, Haemophilia B
Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years.
- Trial name
- NCT06831734
- Testing
- Concizumab
- Where
- 23 sites in Japan
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia B
Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor.
- Trial name
- NCT06285071
- Testing
- Concizumab
- Where
- 11 sites in Japan
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per…
- Trial name
- NCT05203679
- Testing
- Single dose intravenous injection of BBM-H901
- Where
- 9 sites in China
- Volunteers
- 32 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Hemophilia B
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual…
- Trial name
- NCT05709288
- Testing
- BBM-H901
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors
The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use.
- Trial name
- NCT06312475
- Testing
- KN057
- Where
- China
- Volunteers
- 53 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia A, Hemophilia B
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study…
- Trial name
- NCT05145127
- Testing
- PF-06741086
- Where
- 78 sites in United States, Argentina and 22 more
- Volunteers
- 245 people
- Sponsor
- Pfizer
Hemophilia A and B
Study of a Smart Sharps Disposal Device in Patients With Hemophilia
This pilot study will have patients use a smart sharps disposal container at home for disposal of needles used to administer their hemophilia drugs.
- Trial name
- NCT07421154
- Testing
- The smart Sharps device is given to the patient and they use it to throw needles away in. It is not internal to the patient.
- Where
- 2 sites in United States
- Volunteers
- 23 people
- Sponsor
- University of Pennsylvania
Hemophilia B
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
- Trial name
- NCT01687608
- Testing
- AskBio009
- Where
- 15 sites in United States
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Factor VIII Deficiency, Factor IX Deficiency
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
- Trial name
- PedNet (NCT02979119)
- Where
- 39 sites in Austria, Belgium and 17 more
- Volunteers
- 4,000 people
- Sponsor
- PedNet Haemophilia Research Foundation
Hemophilia B
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05641610
- Testing
- ZS801
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A, Hemophilia A and B
A Phase III Clinical Trial Evaluating SR604 Injection for Prophylactic Treatment in Patients With Hemophilia
This is a single-arm, multicenter Phase III clinical trial designed to evaluate the efficacy and safety of prophylactic treatment with SR604 Injection in hemophilia A/B patients with or without inhibitors.
- Conditions
- Hemophilia A, Hemophilia A and B, Hemophilia B
- Trial name
- NCT07876674
- Testing
- SR604
- Volunteers
- 90 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Phase IV multi-center, US-centric, open-label, safety study enrolling participants with Hemophilia A or B with inhibitors, 12 years of age and older, who are either on long term prophylactic treatment (e.g., emicizumab) at risk…
- Trial name
- NCT04647227
- Testing
- coagulation factor VIIa [recombinant]-jncw
- Where
- 21 sites in United States
- Volunteers
- 55 people
- Sponsor
- American Thrombosis and Hemostasis Network
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris