Clinical Trials Logo
  1. Home
  2. Hemophilia A
  3. NCT05768386

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) — GENEr8-LTE  

Hemophilia A Clinical Trial

Official title:
A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial

Clinical Trial Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.

Clinical Trial Description

Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05768386
Study type Observational
Source BioMarin Pharmaceutical
Contact
Status Enrolling by invitation
Phase
Start date January 1, 2023
Completion date January 2040
View Details
See also
  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