Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

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Trial Status Active Not Recruiting

Trial Identifier

NCT03370913

Condition

Hemophilia A, Hemophilia a

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as “Post FVIII Prophylaxis to Last Visit”). The study will also assess the impact of BMN 270 (compared to FVIII prophylaxis) on: the number of bleeding episodes requiring exogenous FVIII treatment in “Post FVIII Prophylaxis to Last Visit”, FVIII activity as measured by chromogenic sustrate assay at Week 104 following intravenous infusion of BMN 270, usage of exogenous FVIII replacement therapy in “Post FVIII Prophylaxis to Last Visit”, health-related quality of life patient-reported outcomes at week 104 following intravenous infusion of BMN 270. The study will also evaluate the safety of the BMN 270.

Eligibility Criteria

Birth Sex

Male

Age

From 18 Years

Healthy Volunteers

No

Drug/Treatment:

valoctocogene roxaparvovec

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

144

Study Started:

2017-12-19

Study Updated:

2023-12-20

Trial Locations

  • Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center

    Los Angeles, California, United States

  • UC Davis Hemophilia Treatment Center

    Sacramento, California, United States

  • University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center

    San Diego, California, United States

  • UCSF Medical Center

    San Francisco, California, United States

  • University of Colorado

    Aurora, Colorado, United States

  • St. Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders

    Tampa, Florida, United States

  • Ann & Robert H. Lurie Children's Hospital of Chicago, Department of Hematology

    Chicago, Illinois, United States

  • James Graham Brown Cancer Center

    Louisville, Kentucky, United States

  • University of Michigan, Pediatric Hematology and Oncology

    Ann Arbor, Michigan, United States

  • Wayne State University, Detroit Medical Center

    Detroit, Michigan, United States

  • University of Minnesota

    Minneapolis, Minnesota, United States

  • Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology

    Saint Louis, Missouri, United States

  • UNC Hemophilia and Thrombosis Center

    Chapel Hill, North Carolina, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, United States

  • The Royal Adelaide Hospital (RAH)

    Adelaide, , Australia

  • Royal Brisbane and Women's Hospital

    Brisbane, , Australia

  • Alfred Hospital

    Melbourne, , Australia

  • Fiona Stanley Hospital

    Perth, , Australia

  • Royal Prince Alfred Hospital

    Sydney, , Australia

  • University Hospital Leuven

    Leuven, , Belgium

  • Campinas Estadual University (UNICAMP) / Campinas Hemocentro / Hematologia E Hemoterapia Center

    Campinas, , Brazil

  • Parana's Hematology And Hemotherapy Center (HEMEPAR)

    Curitiba, , Brazil

  • Arthur De Siqueira Cavalcanti Hematology State Institute

    Rio De Janeiro, , Brazil

  • Sao Paulo University Clinical Hospital

    São Paulo, , Brazil

  • Holy Spirit Hematology and Hemotherapy Center

    Vitória, , Brazil

  • Regional University Hospital of Lille (CHRU de Lille)

    Lille, , France

  • Hopital de la Timone Marseille - Assistance Publique des Hopitaux de Marseille

    Marseille, , France

  • Vivantes Clinic im Friedrichshain- Landsberger Allee

    Berlin, , Germany

  • University Clinic Bonn

    Bonn, , Germany

  • Chaim Sheba Medical Center

    Ramat Gan, , Israel

  • Maggiore Polyclinic Hospital, IRCCS Ca' Granda, Center for Hemophilia and Thrombosis Angelo Bianchi Bonomi

    Milan, , Italy

  • Department of Pediatrics, Kyung Hee University Hospital at Gangdong

    Seoul, , Korea, Republic of

  • Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center

    Johannesburg, , South Africa

  • Hospital Teresa Herrera

    A Coruna, , Spain

  • University Hospital Virgen del Rocio (HUVR)

    Seville, , Spain

  • Changhua Christian Hospital

    Changhua, , Taiwan

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung, , Taiwan

  • Taichung Veterans General Hospital

    Taichung, , Taiwan

  • National Taiwan University Hospital

    Taipei, , Taiwan

  • Tri-Service General Hospital

    Taipei, , Taiwan

  • Queen Elizabeth Hospital

    Birmingham, , United Kingdom

  • Addenbrookes Hospital

    Cambridge, , United Kingdom

  • Glasgow Royal Infirmary, Department of Hematology

    Glasgow, , United Kingdom

  • Barts and The London School of Medicine and Dentistry, Haemophilia Centre

    London, , United Kingdom

  • Hammersmith

    London, , United Kingdom

  • St Thomas' Hospital

    London, , United Kingdom

  • Churchill Hospital, Oxford Hemophilia and Thrombosis Center

    Oxford, , United Kingdom

  • University Hospital Southampton NHS Foundation Trust

    Southampton, , United Kingdom

Inclusion Criteria

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.

Exclusion Criteria

1. Detectable pre-existing antibodies to the adeno-associated virus 5 (AAV5) capsid.
2. Any evidence of active infection or any immunosuppressive disorder, except for HIV infection
3. Any evidence of active infection or any immunosuppressive disorder, including HIV infection (effective as of Protocol Amendment 3)
4. Significant liver dysfunction.
5. Prior liver biopsy showing significant fibrosis.
6. Evidence of any bleeding disorder not related to hemophilia A.
7. Platelet count of < 100 x 10^9/L. 8. Creatinine ≥ 1.5 mg/dL. 9. Liver cirrhosis of any etiology as assessed by liver ultrasound. 10. Chronic or active hepatitis B. 11. Active Hepatitis C. 12. Active malignancy, except non-melanoma skin cancer. 13. History of hepatic malignancy. 14. History of arterial or venous thromboembolic events. 15. Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation

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