UK Pharmaceutical Company started a Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
BioMarin Pharmaceutical is recruiting patients for the clinical trial of Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors.
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
The researchers plan that December 2020 will be the study start date. The indicative completion of the clinical trial will be expected in June 2027.
Among primary outcome measures are the Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
The study will take place at the Royal Free Hospital, London, United Kingdom.
Patients with the following pathologies:
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
- Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
- Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
are excluded from participation.
The page dedicated to this clinical trial can be found here: https://ichgcp.net/clinical-trials-registry/NCT04684940
