BeneGene-2
Research type
Research Study
Full title
Phase 3, open-label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIXR338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C≤2%) (BeneGene-2)
IRAS ID
271917
Contact name
Jemma Capuozzo
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2018-003086-33
Clinicaltrials.gov Identifier
16,437, IND
Duration of Study in the UK
years, months, days
Research summary
The purpose of this study is to learn about the safety and effectiveness of the study drug PF-06838435 which is a gene therapy based treatment. The sponsor wants to see if the drug works to prevent bleeding episodes commonly experienced by patients with Haemophilia B.
Patients with Haemophilia B, bleed more frequently than those without Haemophilia B because it is more difficult to form a blood clot to stop bleeding. This is due to a deficiency of a protein called FIX/Factor 9 (referred to as “Factor IX”) that is needed to prevent bleeding. Specifically, their genes contain an error resulting in a lack of or a non-functional Factor IX.
Current treatment relies on replacement of non-functional Factor IX with Factor IX products. These products are administered intravenously. They are administered when needed and/or at scheduled intervals to try and prevent bleeding episodes.
The study drug is part of class of drugs called gene therapy. Gene therapy may be able to introduce a functioning copy of the gene to produce functional protein, in this case the Factor IX protein.
Unlike current approved treatments for Haemophilia B, the study medication is only intended to be administered once by infusion.
It is hoped that after receiving the study drug the body will start making sufficient Factor IX- protein to prevent the need for additional replacement therapy. The study drug will contain a virus to help deliver the gene to cells. Patients that have not created antibodies to the virus portion of the study drug will be allowed to receive study drug as long as they meet the rest of the study eligibility requirements.
There will be about 50 participants taking part in this study. The study is being conducted at about 31 different study sites in approximately 14 countries.REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1652
Date of REC Opinion
28 Jan 2020
REC opinion
Further Information Favourable Opinion