BMN 270-302 Gene therapy study in severe Haemophilia A patients
Research type
Research Study
Full title
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector– Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels\n≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
IRAS ID
236312
Contact name
John Pasi
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2017-003573-34
Duration of Study in the UK
6 years, 4 months, 2 days
Research summary
This study is being conducted by BioMarin Pharmaceutical Inc. as a Phase 3, open label study in order to determine the safety and efficacy of BMN 270 (an Adeno-Associated Virus based gene therapy vector) in participants with severe\nHaemophilia A.\n\nHaemophilia A (HA) is an X-linked recessive bleeding disorder that affects approximately 1 in 5,000 males. It is caused by deficiency in the activity of coagulation factor VIII (FVIII), which is essential to blood clotting. Severe HA is\nclassified as FVIII activity less than 1%. Clinical manifestations of severe HA are frequent spontaneous bleeding episodes, predominantly in joints and soft tissues, with a substantially increased risk of death from haemorrhage when the brain is involved. Haemophilia A is well suited for a gene transfer approach because clinical manifestations are attributable to the lack of a single gene product (FVIII) that circulates in minute amounts in the plasma. BMN 270 is an AAV5 gene therapy drug that contains a FVIII gene. Phase 1/2 studies have demonstrated it can lead to expression of therapeutic amounts of FVIII and stop bleeding symptoms.\n\nAs a phase 3 programme, approximately 40 participants will receive a single dose of BMN 270 as an intravenous infusion. BMN 270 offers the potential of disease modification through continuous endogenous production of FVIII levels following a single intravenous administration and is designed to achieve stable, potentially life-long expression of active hFVIII in the blood.\n\nParticipants will be in the study for up to 5 years.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
17/SC/0639
Date of REC Opinion
23 Feb 2018
REC opinion
Further Information Favourable Opinion