RecruitingPhase 3NCT05568719

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

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


Sponsor

Pfizer

Enrollment

173 participants

Start Date

Dec 28, 2022

Study Type

INTERVENTIONAL

Conditions

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

Sex: MALEMin Age: 18 Years

Inclusion Criteria1

  • 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 Criteria1

  • None

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Interventions

DIAGNOSTIC_TESTTesting 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


Locations(16)

The Regents of the University of California||UC Davis Health

Rancho Cordova, California, United States

UC Davis Health

Sacramento, California, United States

UC Davis Ambulatory Care Clinic

Sacramento, California, United States

UC Davis Hemophilia Treatment Center

Sacramento, California, United States

UC Davis Medical Center

Sacramento, California, United States

UCSF Outpatient Hematology Clinic

San Francisco, California, United States

USF Health Morsani Center For Advanced Healthcare

Tampa, Florida, United States

The University of South Florida Board of Trustees

Tampa, Florida, United States

Mississippi Center For Advanced Medicine

Madison, Mississippi, United States

Cornell University||Joan & Sanford I. Weill Medical College

New York, New York, United States

Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, United States

The Childrens Hospital of Philadelphia Division of Hematology

Philadelphia, Pennsylvania, United States

Washington Institute for Coagulation

Seattle, Washington, United States

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Sydney Local Health District (RPAH Zone)

Camperdown, New South Wales, Australia

Ege Universitesi Hastanesi

Izmir, İ̇zmir, Turkey (Türkiye)

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NCT05568719


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