Summary

Eligibility
for males ages 18 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Andrew Leavitt

Description

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.

Official Title

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 Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Keywords

Hemophilia A Gene Therapy Clotting Disorders Blood Disorder Blood Coagulation Disorders Inherited Blood Coagulation disorders Hematologic Diseases Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases Inborn Factor VIII Coagulants valoctocogene roxaparvovec valoctocogene roxaparvovec Open Label

Eligibility

You can join if…

Open to males ages 18 years and up

  1. Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history, at the time of signing the informed consent.
  2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
  3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs).
  4. No previous documented history of a detectable FVIII inhibitor, and results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least one week apart within the past 12 months.

You CAN'T join if...

  1. Detectable pre-existing antibodies to the AAV5 capsid.
  2. Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
  3. Significant liver dysfunction.
  4. Prior liver biopsy showing significant fibrosis.
  5. Evidence of any bleeding disorder not related to hemophilia A.
  6. Platelet count of < 100 x 109/L.

  7. Creatinine ≥ 1.5 mg/dL.
  8. Liver cirrhosis of any etiology as assessed by liver ultrasound.
  9. Chronic or active hepatitis B.
  10. . Active Hepatitis C.
  11. . Active malignancy, except non-melanoma skin cancer.
  12. . History of hepatic malignancy.
  13. . History of arterial or venous thromboembolic events.
  14. . Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation.

Locations

  • UCSF Medical Center
    San Francisco California 94143-0106 United States
  • UC Davis Hemophilia Treatment Center
    Sacramento California 95817 United States

Lead Scientist at UCSF

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT03370913
Phase
Phase 3
Study Type
Interventional
Last Updated