Long-Term Safety of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec for Hemophilia A or B

This study is looking at the long-term safety and effectiveness of two gene therapies, giroctocogene fitelparvovec and fidanacogene elaparvovec, for men with hemophilia A or B. These therapies were previously given to participants in other Pfizer studies. Researchers want to see if these treatments cause any serious side effects like blood clots (thromboembolic events), the body developing resistance to the treatment (factor inhibitor development), or liver cancer (hepatic malignancy) over a 10-year period. You can join this study only if you have already received one of these treatments in a specific Pfizer-sponsored clinical trial. The study plans to enroll 173 participants, but its current status is unclear.

Study design
This is an interventional study, meaning participants receive a specific treatment. It is designed to follow 173 men who previously received giroctocogene fitelparvovec or fidanacogene elaparvovec.
What's involved
Data collection and participant visits will be based on standard of care. This may include evaluation of AAV vector integration if a liver sample is obtained.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 10 years to monitor for specific safety events.

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NCT05568719

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Recruiting
PHASE3Ages 18+Interventional
Pfizer
~173 participants
Updated 2026-05-27 on ClinicalTrials.gov
What's tested:Testing of hepatic AAV Vector integration

At a glance

Recruiting sites
14 of 23 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of thromboembolic events
Measured over Day 1 to 10 years
+4 more outcomes measured
Hemophilia A
Hemophilia B
23 sites across 13 states
California6
Florida2
New York2
Pennsylvania2
New South Wales2
Taiwan2
Mississippi1
Washington1
  • Pfizer CT.gov Call Center · STUDY_DIRECTOR · Pfizer

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Eligibility criteria

Inclusion

Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.

Exclusion

None
  • Incidence of thromboembolic eventsDay 1 to 10 years
  • Incidence of factor inhibitor developmentDay 1 to 10 years

    FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).

  • Incidence of hepatic malignancyDay 1 to 10 years
  • Incidence of liver abnormalitiesDay 1 to 10 years
  • Factor activity levelDay 1 to 10 years

    Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.