Skip to content
Pharma Observatory a Scientist.com platform

Clinical trial

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

NCT03520712 is registered in ClinicalTrials.gov with BioMarin Pharmaceutical as lead sponsor.

NCT NCT03520712
Email me this page as a PDF

Search trials, sites, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Get the NCT03520712 report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
BioMarin Pharmaceutical
Phase
Phase 1/2
Overall status
Terminated
Start date
April 24, 2018
Completion date
August 7, 2024
Enrollment
3 (actual)
Sites
9

Conditions

As the registry lists them; a condition with its own page is linked.

blood disorder, clotting disorders, gene therapy, hemophilia a