BMN270-203 Gene Therapy Study in Patients with Antibodies Against AAV5
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 Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5
IRAS ID
238111
Contact name
Amit Nathwani
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2017-000662-29
Duration of Study in the UK
5 years, 1 months, 9 days
Research summary
This study is being conducted by BioMarin Pharmaceutical Inc. as a phase 1/2 study to gather information primarily about the safety and activity of BMN 270 (an Adeno-Associated Virus based gene therapy vector) in participants with severe Haemophilia A with pre-existing antibodies against AAV5.
Haemophilia 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 classified 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 and only one dose will be given in this study.
This will be the first time that this vector will be used in humans with pre-existing antibodies to AAV5 and the purpose of this study is to learn about the safety and any good or bad effects it has on the participants and Haemophilia A. The study will see if the immune system (which is a body’s way of protecting itself) will react to the vector by producing additional antibodies, which the body makes to protect itself against things like viruses and bacteria. The study will also explore the effects that the study drug has on bleeding episodes. It is expected that up to 10 adults aged 18 and older will take part in the study.
Participants will be in the study for up to 5 years.REC name
South Central - Oxford A Research Ethics Committee
REC reference
17/SC/0641
Date of REC Opinion
1 Feb 2018
REC opinion
Further Information Favourable Opinion