Clinical trials: Hemophilia A and B
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Congenital Bleeding Disorder, Haemophilia A
To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives
This survey is conducted in South America. The purpose is to identify the key psychosocial issues affecting patients with haemophilia.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Haemophilia A With Inhibitors
- Trial name
- HERO (NCT02241694)
- Testing
- No treatment given
- Where
- Brazil
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B, Severe, Hemophilia A, Severe
Liver Biopsy In Haemophilia Gene Therapy
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at >1% any time after gene transfer following AAV mediated gene transfer.
- Trial name
- NCT04817462
- Testing
- Liver biopsy
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- University College, London
Hemophilia A, Hemophilia B
World Bleeding Disorders Registry
The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient…
- Conditions
- Hemophilia A, Hemophilia B, Von Willebrand Diseases
- Trial name
- WBDR (NCT03327779)
- Where
- Canada
- Volunteers
- 20,000 people
- Sponsor
- World Federation of Hemophilia
Hemophilia A
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the…
- Trial name
- NCT03588299
- Testing
- BAY2599023 (DTX201)
- Where
- 13 sites in United States, Bulgaria and 4 more
- Volunteers
- 11 people
- Sponsor
- Bayer
Hemophilia A
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately…
- Trial name
- NCT00543439
- Testing
- Moroctocog alfa (AF-CC)
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 66 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Trial of NovoSeven® in Haemophilia - Joint Bleeds
This trial is conducted in the United States of America (USA). This study compares the effectiveness and safety of NovoSeven® to FEIBA (FEIBA VH) in haemophilia patients with inhibitors being treated for joint bleeds.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT00108797
- Testing
- eptacog alfa (activated), Feiba VH
- Where
- 20 sites in United States
- Volunteers
- 42 people
- Sponsor
- Novo Nordisk A/S
Mild Hemophilia
Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%)
Currently, few recommendations exist for patients with mild hemophilia, who represent approximately 60% of the hemophilia population.
- Trial name
- echoSAHM (NCT07719647)
- Testing
- Joint Ultrasound (HEAD-US), Blood Sample for Coagulation Factor Measurement
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals