Trial Profile
A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5
Status:
Active, no longer recruiting
Phase of Trial:
Phase I/II
Latest Information Update: 28 Sep 2023
Price :
$35
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At a glance
- Drugs Valoctocogene-roxaparvovec (Primary)
- Indications Haemophilia A
- Focus Adverse reactions
- Acronyms GENEr8-AAV5+
- Sponsors BioMarin Pharmaceutical
- 25 Sep 2023 Planned End Date changed from 1 Nov 2027 to 1 Jun 2024.
- 25 Sep 2023 Planned primary completion date changed from 1 Nov 2027 to 1 Jun 2024.
- 25 Sep 2023 Status changed from recruiting to active, no longer recruiting.