Evaluating the safety and effectiveness of gene therapies for Hemophilia A and B
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
This study is testing two gene therapies for Hemophilia A and B to see how safe and effective they are for patients who have already taken part in previous trials.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 173 (estimated) |
| Ages | 18 Years and up |
| Sex | Male |
| Sponsor | Pfizer Industry-sponsored |
| Locations | 19 sites (Rancho Cordova, California and 18 other locations) |
| Trial ID | NCT05568719 on ClinicalTrials.gov |
What this trial studies
This study investigates the long-term safety and efficacy of two gene therapies, giroctocogene fitelparvovec for Hemophilia A and fidanacogene elaparvovec for Hemophilia B, in patients who have previously participated in Pfizer-sponsored clinical trials. Participants will undergo regular assessments based on standard care protocols to monitor their health outcomes. The study aims to gather data on the integration of the adeno-associated virus vector in the liver and its impact on bleeding episodes.
Who should consider this trial
Good fit: Ideal candidates are patients with Hemophilia A or B who have previously received investigational gene therapies in Pfizer-sponsored studies.
Not a fit: Patients who have not participated in prior Pfizer-sponsored studies involving these gene therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could significantly improve the management of Hemophilia A and B, potentially reducing bleeding episodes and enhancing patients' quality of life.
How similar studies have performed: Other studies involving gene therapies for hemophilia have shown promising results, indicating a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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. Exclusion Criteria: -None
Where this trial is running
Rancho Cordova, California and 18 other locations
- The Regents of the University of California||UC Davis Health — Rancho Cordova, California, United States (Recruiting)
- UC Davis Health — Sacramento, California, United States (Recruiting)
- UC Davis Ambulatory Care Clinic — Sacramento, California, United States (Recruiting)
- UC Davis Hemophilia Treatment Center — Sacramento, California, United States (Recruiting)
- UC Davis Medical Center — Sacramento, California, United States (Recruiting)
- UCSF Outpatient Hematology Clinic — San Francisco, California, United States (Not_yet_recruiting)
- USF Health Morsani Center For Advanced Healthcare — Tampa, Florida, United States (Recruiting)
- The University of South Florida Board of Trustees — Tampa, Florida, United States (Recruiting)
- Mississippi Center For Advanced Medicine — Madison, Mississippi, United States (Terminated)
- Cornell University||Joan & Sanford I. Weill Medical College — New York, New York, United States (Recruiting)
- Weill Cornell Medical College-New York Presbyterian Hospital — New York, New York, United States (Recruiting)
- The Childrens Hospital of Philadelphia Division of Hematology — Philadelphia, Pennsylvania, United States (Recruiting)
- Washington Institute for Coagulation — Seattle, Washington, United States (Not_yet_recruiting)
- Royal Prince Alfred Hospital — Camperdown, New South Wales, Australia (Recruiting)
- Sydney Local Health District (RPAH Zone) — Camperdown, New South Wales, Australia (Recruiting)
- Kyung Hee University Hospital at Gangdong — Seoul, South Korea (Not_yet_recruiting)
- Skåne University Hospital — Malmö, Sweden (Not_yet_recruiting)
- National Taiwan University Hospital — Taipei, Taiwan (Not_yet_recruiting)
- Ege Universitesi Hastanesi — Izmir, İ̇zmir, Turkey (Türkiye) (Recruiting)
Study contacts
- Study coordinator: Pfizer CT.gov Call Center
- Email: ClinicalTrials.gov_Inquiries@pfizer.com
- Phone: 1-800-718-1021
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.