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for males ages 18 years and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion:



This clinical trial is being conducted to learn more about a potential treatment (valoctocogene roxaparvovec) for people with severe hemophilia A. This research study will test and confirm the safety and effectiveness of the 6E13 vg/kg dose of the study drug (valoctocogene roxaparvovec) that contains the correct gene to make Factor VIII so that the body can make its own Factor VIII that functions properly. Only one dose of valoctocogene roxaparvovec is being given in this study, and this dose has been previously studied in another clinical trial in patients with hemophilia A. This is a phase 3 study which is meant to show that the study drug is safe and works to help treat hemophilia A. The study will see if liver cells are able to make Factor VIII that functions properly after receiving this study drug. The study will also examine the effects that the study drug has on how much Factor VIII concentrates patients have to inject into their veins and on their bleeding episodes after the study drug has been administered. Finally, the study will see if and how the body responds to the study drug - for example, whether liver cells become inflamed or whether the body makes antibodies (something the immune system makes to protect itself against things like bacteria and viruses) against the vector or the new Factor VIII gene.

Official Title

Phase 3 Open-Label Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec an AAV Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤1 IU/dL Receiving Prophylactic FVIII Infusions


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


You can join if…

Open to males ages 18 years and up

  • 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.
  • Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry. High-quality, well-documented historical data concerning bleeding episodes and FVIII usage over the previous 12 months must be available.
  • Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs).
  • No history of FVIII inhibitor, and results from a Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions (the most recent one of which should be tested at the central laboratory) at least one week apart within the past 12 months
  • HIV positive patients may be enrolled, only if the patient has a CD4 count > 200/mm3 and an undetectable viral load.

You CAN'T join if...

  • Detectable pre-existing antibodies to the AAV5 capsid.
  • Any evidence of active infection or any immunosuppressive disorder, except for HIV infection as described in the inclusion criterion above.
  • Significant liver dysfunction with any of the following abnormal laboratory results:
  • ALT (alanine transaminase) or AST >2X ULN;
  • Total bilirubin >2X ULN;
  • Alkaline phosphatase >2X ULN; or
  • INR (international normalized ratio) ≥ 1.4.

Subjects whose liver laboratory assessments fall outside of these ranges may undergo repeat testing and, if eligibility criteria are met on retest, may be enrolled after confirmation by the Medical Monitor. In addition, subjects with abnormal laboratory results related to confirmed benign liver conditions (eg, Gilbert's syndrome) are considered eligible for the study notwithstanding their abnormal laboratory results and may be enrolled after discussion with the Medical Monitor.

  • Prior liver biopsy showing significant fibrosis of 3 or 4 as rated on a scale of 0-4 on the Batts-Ludwig (Batts 1995) or METAVIR (Bedossa 1996) scoring systems, or an equivalent grade of fibrosis if an alternative scale is used.
  • Evidence of any bleeding disorder not related to hemophilia A.
  • Platelet count of < 100 x 109/L.

  • Creatinine ≥ 1.5 mg/dL.
  • Liver cirrhosis of any etiology as assessed by liver ultrasound.
  • Chronic or active hepatitis B as evidenced by positive serology testing and confirmatory HBV DNA testing. Refer to the Centers for Disease Control (CDC) table for the interpretation of serological test results in the Laboratory Manual.
  • Active Hepatitis C as evidenced by detectable HCV RNA or currently on antiviral therapy.
  • Active malignancy, except non-melanoma skin cancer.
  • History of hepatic malignancy.
  • History of arterial or venous thromboembolic events (eg, deep vein thrombosis,nonhemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolus),with the exception of catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing.
  • Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation.


  • UCSF Medical Center not yet accepting patients
    San Francisco, California, 94143-0106, United States
  • Lucile Packard Children's Hospital at Stanford not yet accepting patients
    Palo Alto, California, 94304, United States
  • UC Davis Hemophilia Treatment Center accepting new patients
    Sacramento, California, 95817, United States
  • Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center not yet accepting patients
    Los Angeles, California, 90007-2664, United States


accepting new patients
Start Date
Completion Date
BioMarin Pharmaceutical
Phase 3
Study Type
Last Updated
December 12, 2017
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