BMN 270 Gene Therapy in Severe Haemophilia A Patients with Inhibitors
Research type
Research Study
Full title
A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Active or Prior Inhibitors
IRAS ID
279454
Contact name
Amit Nathwani
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2019-003213-34
Clinicaltrials.gov Identifier
017659 , IND
Duration of Study in the UK
6 years, 6 months, 0 days
Research summary
Haemophilia A (HA) is a genetic bleeding disorder that usually affects males causing increased bleeding in joints and soft tissue with increased risk of death from haemorrhage, bleeding in the brain. For the past decade, patients with HA have received infusions of clotting factor to treat episodes of bleeding. However, patients have developed antibodies to neutralise the effect of the clotting factor treatment leading to an increase in number of bleeding episodes. The alternative treatment for patient with resistance to the conventional clotting factor treatment is the newly approved drug emicizumab that causes an increase to multiple clotting factors but require chronic administrations.
The study drug BMN 270 is an AAV5-based gene therapy vector, it contains a functional gene for FVIII so that the body can make its own FVIII. BMN 270 attempts to treat HA by transferring a functional gene for FVIII into cells in the liver, so that the body will begin to make natural FVIII on its own.
So far, BMN 270 has been tested in over 140 patients with haemophilia A without inhibitors. After a single treatment with BMN 270, these patients have been able to produce enough natural FVIII to reduce their number of bleeding episodes and the amount of replacement FVIII they need to take to manage their haemophilia.
This will be the first research study conducted with BMN 270 in patients with active inhibitors. The study is divided into 2 parts; Part A will test BMN 270 in patients who currently have active inhibitors and are receiving regular emicizumab therapy, and Part B will test the drug in patients who have previously had inhibitors and are currently receiving prophylaxis (treatment to prevent disease) with replacement FVIII products. It is expected approximately 10 adult males aged 18 and older around the world will take part in each part of the study.REC name
South Central - Oxford A Research Ethics Committee
REC reference
20/SC/0232
Date of REC Opinion
25 Sep 2020
REC opinion
Further Information Favourable Opinion