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

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BioMarin Pharmaceutical

Status and phase

Active, not recruiting
Phase 3

Conditions

Hemophilia A

Treatments

Biological: valoctocogene roxaparvovec

Study type

Interventional

Funder types

Industry

Identifiers

NCT03370913
2017-003215-19 (EudraCT Number)
270-301

Details and patient eligibility

About

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.

Enrollment

144 patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

144 participants in 1 patient group

valoctocogene roxaparvovec Open Label
Experimental group
Description:
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg
Treatment:
Biological: valoctocogene roxaparvovec

Trial documents
2

Trial contacts and locations

48

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Data sourced from clinicaltrials.gov

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