Hemophilia A Clinical Trial
Official title:
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit"). The study will also assess the impact of BMN 270 (compared to FVIII prophylaxis) on: the number of bleeding episodes requiring exogenous FVIII treatment in "Post FVIII Prophylaxis to Last Visit", FVIII activity as measured by chromogenic sustrate assay at Week 104 following intravenous infusion of BMN 270, usage of exogenous FVIII replacement therapy in "Post FVIII Prophylaxis to Last Visit", health-related quality of life patient-reported outcomes at week 104 following intravenous infusion of BMN 270. The study will also evaluate the safety of the BMN 270.
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT03834727 -
Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
|
||
Completed |
NCT03191799 -
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
|
Phase 3 | |
Completed |
NCT01599819 -
BAX 855 Dose-Escalation Safety Study
|
Phase 1 | |
Terminated |
NCT04541628 -
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
|
Phase 1/Phase 2 | |
Completed |
NCT02847637 -
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
|
Phase 3 | |
Completed |
NCT04072237 -
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
|
Phase 1 | |
Completed |
NCT04085458 -
Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)
|
Phase 4 | |
Completed |
NCT04565236 -
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
|
Phase 4 | |
Recruiting |
NCT05987449 -
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT04621916 -
Preventing Inhibitor Recurrence Indefinitely
|
Phase 4 | |
Not yet recruiting |
NCT02888223 -
Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
|
Phase 1 | |
Completed |
NCT02528968 -
National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
|
N/A | |
Completed |
NCT02225483 -
Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function
|
N/A | |
Completed |
NCT02199717 -
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
|
N/A | |
Completed |
NCT01217255 -
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
|
||
Terminated |
NCT00995046 -
Individually Tailored Prophylaxis in Patients With Severe Hemophilia A
|
N/A | |
Completed |
NCT00969319 -
Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America
|
N/A | |
Completed |
NCT00868530 -
Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
|
Phase 3 | |
Completed |
NCT00839202 -
Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay
|
N/A | |
Completed |
NCT00629837 -
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
|
Phase 1 |