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

Completed · Phase 3

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT03370913

Run by

BioMarin Pharmaceutical

People needed

144

Starts

2017-12-19

Expected to finish

2024-11-20

Last updated by the study team

2025-03-25

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days (EDs).
• 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 may not qualify if…

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

Where it is running

• 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
• Tulane University Hematology & Medical Oncology — New Orleans, Louisiana, 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 — St 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.