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Clinical trial

GENEr8-JPN: 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 Japanese Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

NCT06224907 is registered in ClinicalTrials.gov with BioMarin Pharmaceutical as lead sponsor.

NCT NCT06224907
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Registry record

As filed in ClinicalTrials.gov.

Title
GENEr8-JPN: 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 Japanese Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
Lead sponsor
BioMarin Pharmaceutical
Phase
Phase 3
Overall status
Active, not recruiting
Start date
December 25, 2023
Completion date
March 31, 2029
Enrollment
6 (estimated)
Sites
4

Conditions

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

hemophilia a