Phase 3
Completed N=1
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Source: ClinicalTrials.gov NCT03392974 ↗Enrolled (actual)
1
Serious AEs
0.0%
Results posted
Oct 2021
Primary outcomePrimary: Change of the Median Factor VIII (FVIII) Activity — 4.1 IU/dL
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change of the Median Factor VIII (FVIII) Activity |
4.1 | — |
| SECONDARY Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy |
-4058.24 | — |
| SECONDARY Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment |
5.77 | — |
Eligibility Criteria
Inclusion Criteria
- Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history.
- Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
- Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days.
- No previous documented history of a detectable FVIII inhibitor of less than 0.6 Bethesda Units (BU).
Exclusion Criteria
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
- Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver cirrhosis of any etiology or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
- Active Hepatitis C.
- Prior treatment with any vector/gene transfer agent.
Data sourced from ClinicalTrials.gov (NCT03392974). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.