A Phase 3 study to evaluate the efficacy and safety of BMN 270 gene transfer in patients with severe hemophilia A

Phase 1

• Conditions: Haemophilia A; MedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-003215-19-DE
• Lead Sponsor: BioMarin Pharmaceutical Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-11-20
• Last Update Posted: 8 October 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 134

Inclusion Criteria

- 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 115
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
- 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified