Clinical trials: Severe Hemophilia A
Hemophilia A With Inhibitors
A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)
The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.
- Trial name
- NCT03093480
- Testing
- rFVIIIFc
- Where
- 37 sites in United States, Belgium and 8 more
- Volunteers
- 16 people
- Sponsor
- Bioverativ, a Sanofi company
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
Severe Haemophilia A
Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens
This trial is designed to assess if there is evidence of subclinical joint bleeding on MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically tailored prophylaxis regimens.
- Trial name
- PERSONAL (NCT02314325)
- Testing
- ADVATE [Antihemophilic Factor (Recombinant)]
- Where
- Ireland
- Volunteers
- 20 people
- Sponsor
- St. James's Hospital, Ireland
Hemophilia A
A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and…
- Trial name
- NCT05251090
- Testing
- ADVATE, FRSW107
- Where
- 6 sites in China
- Volunteers
- 13 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia A
Prophylaxis Versus On-demand Therapy Through Economic Report
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
- Trial name
- POTTER (NCT01159587)
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- Italy
- Volunteers
- 58 people
- Sponsor
- Bayer
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi
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
Severe Hemophilia A, Moderate Hemophilia A
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants aged ≥13 and <70 years…
- Trial name
- Beyond ABR (NCT05181618)
- Testing
- Emicizumab
- Where
- 26 sites in United States, Brazil and 10 more
- Volunteers
- 136 people
- Sponsor
- Hoffmann-La Roche