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

Condition(s) studied

Investigational drug(s) / intervention(s)

Testing of hepatic AAV Vector integration: Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated

Study summary

A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.

Eligibility

Primary outcome measure(s)

• Incidence of thromboembolic events — Day 1 to 10 years
• Incidence of factor inhibitor development — Day 1 to 10 years
  FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).
• Incidence of hepatic malignancy — Day 1 to 10 years
• Incidence of liver abnormalities — Day 1 to 10 years
• Factor activity level — Day 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.

Trial sites (39)

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