Clinical trials: Hemophilia A and B
Hemophilia A, Hemophilia B
Liver Biopsy Following Gene Therapy For Hemophilia
This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have…
- Trial name
- NCT05932914
- Testing
- Liver Biopsy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- St. Jude Children's Research Hospital
Hemophilia B
Phase I/IIa Study of FIXFc in Hemophilia B Patients
The primary objective of the study is to assess safety of FIXFc at doses ranging from 1 to 100 IU/kg.
- Trial name
- NCT00716716
- Testing
- rFIXFc
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia A, Hemophilia B
Validation and Reliability of the CHO-KLAT in Turkish
The aim of this study is to investigate the Turkish validity and reliability of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version2.0.
- Trial name
- NCT05439642
- Testing
- Evaluation of health related quality of life
- Where
- 4 sites in Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Yuzuncu Yil University
Congenital Bleeding Disorder, Haemophilia A
Impact of Pain on Functional Impairment and Quality of Life in Adults With Hemophilia
This study is conducted in the United States of America (USA). The aim of the study is to assess the impact of pain on functional impairment and quality of life in adult persons with hemophilia (PWH) with and without inhibitors…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT01988532
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 381 people
- Sponsor
- Novo Nordisk A/S
Hemophilia
In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)).
- Trial name
- NCT05617209
- Testing
- thrombin generation measurement
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Study of Recombinant Human Coagulation Factor VIIa for Injection (FⅦa) in Patients With Hemophilia.
Aim of this trial is to assess the pharmacokinetics and pharmacodynamics (PK/PD) of recombinant human coagulation factor VIIa for injection (FⅦa) in patients with hemophilia.
- Trial name
- NCT04768699
- Testing
- TQG203, TQG203/NovoSeven®
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Hemophilia A or B
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.
- Trial name
- HIZ (NCT07161687)
- Testing
- Marstacimab
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia B
A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients…
- Trial name
- paradigm™7 (NCT03075670)
- Testing
- N9-GP, ALPROLIX®
- Where
- 12 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Trial name
- NCT01757405
- Testing
- Recombinant Factor VIIa BI (rFVIIa BI)
- Where
- 16 sites in United States, Japan and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A…
- Trial name
- PERSEPT2 (NCT02448680)
- Testing
- Coagulation FVIIa (Recombinant)
- Where
- 9 sites in United States, Bulgaria and 4 more
- Volunteers
- 25 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A, Hemophilia B
Socialization of Adult Men With Congenital Hemophilia A or B
The goal of this pilot study is to understand the socialization of adult men with hemophilia living in the United States and their quality of life in order to improve comprehensive care for persons with congenital bleeding…
- Trial name
- PWBCD (NCT01510418)
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Dana-Farber Cancer Institute
Liver Cirrhosis, Hemophilia
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
The goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in…
- Conditions
- Liver Cirrhosis, Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT06014320
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Stanford University
Haemophilia B
A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B
The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP.
- Trial name
- Paradigm9 (NCT05365217)
- Testing
- Nonacog beta pegol
- Where
- 15 sites in China
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Healthy
A Study to Assess the Effects of 2 Different Prothrombin Complex Concentrates on the Pharmacodynamics of Rivaroxaban in Healthy Adult Volunteers
The purpose of this study is to assess the use of 2 different Prothrombin Complex Concentrates (PCCs) on their ability to reverse (normalize) the pharmacodynamic effects of rivaroxaban in healthy adult volunteers.
- Trial name
- NCT01656330
- Testing
- Rivaroxaban 20 mg twice daily, Rivaroxaban 20 mg once daily, Profilnine SD
- Where
- Belgium
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Janssen Research & Development, LLC
Hemophilia B
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and…
- Trial name
- NCT01174446
- Testing
- BAX 326, BeneFIX
- Where
- 25 sites in Argentina, Brazil and 12 more
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
Understanding Hemophilia A and B Drug Dosage Administration Patterns
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the…
- Trial name
- NCT03248141
- Testing
- Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Pfizer
Acquired Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.
- Trial name
- NCT01178294
- Testing
- OBI-1
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
Are There Differences Between Carriers of Haemophilia A and B?
This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed…
- Trial name
- NCT05217992
- Testing
- Genetic assessment (hemophilia testing)
- Where
- Belgium
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.
Hemophilia A, Hemophilia B
Virtual Reality for Hemophilia
Children with hemophilia are exposed to intravenous (IV) procedures that cause pain/anxiety and distraction methods can be employed to improve the patient experience.
- Trial name
- NCT03507582
- Testing
- Virtual Reality Distraction, Standard of Care Distraction
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital