Ignite Creation Date:
2025-12-24 @ 2:05 PM
Ignite Modification Date:
2025-12-27 @ 9:35 PM
Study NCT ID:
NCT02576795
Status:
COMPLETED
Last Update Posted:
2025-04-10
First Post:
2015-10-05
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A
Status:
COMPLETED
Status Verified Date:
2025-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2014-003880-38 | EUDRACT_NUMBER | None | View |