NCT05568719 · Pfizer
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
What this study is about
A study to learn about the long-term safety and effectiveness 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 the usual treatment.
View original scientific description
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.
Interventions
DIAGNOSTIC_TEST
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
Primary outcome measures
Incidence of thromboembolic events
Time frame: Day 1 to 10 years
Incidence of factor inhibitor development
Time frame: 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
Time frame: Day 1 to 10 years
Incidence of liver abnormalities
Time frame: Day 1 to 10 years
Factor activity level
Time frame: 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- -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.
Where
- Rancho Cordova, California
- Sacramento, California
- San Francisco, California
- Tampa, Florida
- Madison, Mississippi
- New York, New York
- Philadelphia, Pennsylvania
- Seattle, Washington
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 27, 2026 · Source of record for eligibility and locations