AFFINE
Research type
Research Study
Full title
Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C≤1%)
IRAS ID
289410
Contact name
Sirjana Gurung
Contact email
Sponsor organisation
Pfizer Inc.,
Eudract number
2019-004451-37
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
BB-IND 17250, IND; PF-07055480 (formerly SB-525), Compoud Name
Duration of Study in the UK
5 years, 4 months, 27 days
Research summary
Haemophilia A is caused by a gene that contains a mistake. Because of this, the body does not make enough factor 8 (hereafter referred to as “factor VIII”). Factor VIII is one of the proteins needed for blood to clot. At present, the main therapy options for haemophilia A involve intravenous factor VIII injections. These can be at the time of a bleed (on-demand) or at regular intervals (prophylaxis). The AFFINE clinical study will assess the safety and effectiveness of a study drug to see if a single infusion can reduce the bleeding episodes associated with haemophilia A over the long term, when compared to routine prophylaxis. The study drug uses a modified virus to introduce a functioning factor VIII gene into the body, enabling your body to produce factor VIII. This approach is known as gene therapy.
To take part, participants must have moderately severe to severe haemophilia A, be between 18 and 64 years of age, and have taken part in the lead-in study, C0371004, for at least 6 months of routine factor VIII prophylaxis.
Participation in the study will last for just over 5 years. After a 6-week screening period, participants will receive one infusion of the study drug and enter a follow-up period of 5 years with at least 18 study visits planned at the clinic. Frequent visits are planned during the first year, and then participants will be only required to come to a clinic every 6 months, or every year (from Year 3).Throughout the study, additional visits (at home using home health services when provided, or on-site), will also be required to provide some blood, urine, semen and saliva samples.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/1256
Date of REC Opinion
11 Feb 2021
REC opinion
Further Information Favourable Opinion