Pfizer and Sangamo Therapeutics have reopened the recruitment of their phase 3 study evaluating giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe haemophilia A, the companies announced.

The reopening comes after the two partners voluntarily paused the trial in November 2021 after patients treated with the therapy experienced blood clotting protein Factor VIII activity greater than 150%, raising concerns that they may be at increased risk of harmful clotting events.

The US Food and Drug Administration (FDA) subsequently placed a clinical hold on the trial, before lifting it in March this year. However, the partners kept the voluntary pause in place while working to meet all necessary conditions, including approval of updated trial protocols by regulatory authorities.

People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot. The condition occurs in around one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.

The phase 3 AFFINE study is an open-label, multicentre, single arm study to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult male participants, aged 18-64 years, with moderately severe to severe haemophilia A.

Eligible study participants will have completed at least six months of routine FVIII prophylaxis therapy during the lead-in phase 3 study in order to collect pretreatment data for efficacy and selected safety parameters, the companies stated.

The primary endpoint in AFFINE is the impact of giroctocogene fitelparvovec on annualised bleeding rate (ABR) after 15 months of follow-up. This will be compared to ABR on prior FVIII prophylaxis replacement therapy.

The secondary endpoints include FVIII activity level after the onset of steady state and after 15 months following infusion of giroctocogene fitelparvovec.

Trial sites will begin to resume enrolment this month, with dosing expected to resume in October and all trial sites anticipated to be active by the end of 2022, the companies outlined.

Giroctocogene fitelparvovec has been granted Orphan Drug, Fast Track and regenerative medicine advanced therapy designations by the FDA, as well as Orphan Medicinal Product designation by the European Medicines Agency.

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Pfizer and Sangamo Therapeutics reopen recruitment for haemophilia A gene therapy trial - PMLiVE