NCT03370913
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.
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.
Male
From 18 Years
No
valoctocogene roxaparvovec
Phase 3
Interventional
144
2017-12-19
2023-12-20
Los Angeles, California, United States
Sacramento, California, United States
San Diego, California, United States
San Francisco, California, United States
Aurora, Colorado, United States
Tampa, Florida, United States
Chicago, Illinois, United States
Louisville, Kentucky, United States
Ann Arbor, Michigan, United States
Detroit, Michigan, United States
Minneapolis, Minnesota, United States
Saint Louis, Missouri, United States
Chapel Hill, North Carolina, United States
Columbus, Ohio, United States
Adelaide, , Australia
Brisbane, , Australia
Melbourne, , Australia
Perth, , Australia
Sydney, , Australia
Leuven, , Belgium
Campinas, , Brazil
Curitiba, , Brazil
Rio De Janeiro, , Brazil
São Paulo, , Brazil
Vitória, , Brazil
Lille, , France
Marseille, , France
Berlin, , Germany
Bonn, , Germany
Ramat Gan, , Israel
Milan, , Italy
Seoul, , Korea, Republic of
Johannesburg, , South Africa
A Coruna, , Spain
Seville, , Spain
Changhua, , Taiwan
Kaohsiung, , Taiwan
Taichung, , Taiwan
Taipei, , Taiwan
Taipei, , Taiwan
Birmingham, , United Kingdom
Cambridge, , United Kingdom
Glasgow, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
Oxford, , United Kingdom
Southampton, , United Kingdom
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.
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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