Clinical trials: Severe Haemophilia B, open to volunteers
Hemophilia B
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
- Trial name
- NCT06003387
- Testing
- CSL222 (AAV5-hFIXco-Padua)
- Where
- 32 sites in United States, Australia and 13 more
- Volunteers
- 35 people
- Sponsor
- CSL Behring
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, 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
Hemophilia a and B
KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors
The goal of this clinical trial is to evaluate the safety and efficacy of KN057 in adult patients with severe Hemophilia A (coagulation factor FVIII activity <1%) or moderate-to-severe Hemophilia B (FIX activity ≤2%).
- Trial name
- NCT06747416
- Testing
- KN057
- Volunteers
- 24 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
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 B
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately…
- Trial name
- IX-TEND 3004 (NCT07080905)
- Testing
- CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 20 people
- Sponsor
- CSL Behring
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