C0371017: A phase 3 study of Safety and Effectiveness of GF and FE in Haemophilia A and B
Research type
Research Study
Full title
A Phase 3, Non-Investigational Product, Multi Country Cohort Study To Describe The Long-Term Safety and Effectiveness Of A Prior Single-Dose Treatment With Investigative Giroctocogene Fitelparvovec Or Fidanacogene Elaparvovec In Participants With Hemophilia A Or Hemophilia B, Respectively.
IRAS ID
1013359
Contact name
Rasid Kamal
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2025-523660-21
Clinicaltrials.gov Identifier
Research summary
This study will characterise the long-term safety and efficacy of giroctocogene fitelparvovec (haemophilia A gene therapy) and fidanacogene elaparvovec (haemophilia B gene therapy), in patients who received these treatments previously through participation in Pfizer-sponsored clinical trials. No investigational product will be given in this study. All participants will have severe or moderately severe haemophilia (factor levels <2%).
Patients will participate in the study until it has been 15 years since they received their gene therapy. The main study involves follow-up based on standard of care (SOC) assessments (i.e., assessments conducted according to SOC practices and SOC visit frequency). These assessments will serve as the basis for long-term safety and efficacy monitoring, throughout the follow-up period.
There is a substudy for participants who have completed at least the Year 3 visit in their previous study (i.e., have completed at least 3 years of post-infusion follow-up). The substudy will include protocol mandated safety and efficacy assessments, at 6-monthly visits, until participants are 6 years post gene therapy administration. Participants will then only in the main study, with standard of care visits and assessments. The assessments required in the substudy are: physical examination, height, weight, vital signs, joint X-rays, FVIII and FIX levels, haematology and clinical chemistry safety blood tests, liver ultrasound scans, haemostais and thrombophilia markers, and health questionnaires.
This study is funded by Pfizer Inc. The study plans to recruit 187 people, at 52 research sites, in 16 countries.REC name
London - West London & GTAC Research Ethics Committee
REC reference
26/LO/0156
Date of REC Opinion
20 Mar 2026
REC opinion
Further Information Favourable Opinion