Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
About this trial
This is an interventional treatment trial for Hemophilia A With Inhibitor focused on measuring Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic DIseases, Factor VIII, Coagulants, Hemophilia A, AAV5 vector
Eligibility Criteria
Inclusion Criteria:
- Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
History of a positive inhibitor result with the first positive result in the last 12 months.
Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
- Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor & hemophilia therapy Hx over previous 12 months.
- Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.
- Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion.
Exclusion Criteria:
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
- 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.
Sites / Locations
- Children's Hospital Los AngelesRecruiting
- UC Davis Hemophilia Treatment CenterRecruiting
- Hemocentro Da UNICAMPRecruiting
- Fondazione IRCCS Cà Granda Ospedale Maggiore PoliclinicoRecruiting
- Kyung Hee University Hospital at GangdongRecruiting
- Kaohsiung Medical University - Chung-Ho Memorial HospitalRecruiting
- National Taiwan University HospitalRecruiting
- Queen Elizabeth HospitalRecruiting
- Guy's and St Thomas' NHS Foundation TrustRecruiting
- Royal Free HospitalRecruiting
Arms of the Study
Arm 1
Experimental
Valoctocogene roxaparvovec Open Label
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg in Active Inhibitor Population (Part A) and Prior Inhibitor Population (Part B).