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

NCT ID: NCT05568719

Last Updated: 2025-08-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

263 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-28

Study Completion Date

2040-02-25

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Non-investigational study
Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Hemophilia A / giroctocogene fitelparvovec

Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study

Group Type OTHER

Testing of hepatic AAV Vector integration

Intervention Type DIAGNOSTIC_TEST

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

Hemophilia B / fidanacogene elaparvovec

Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study

Group Type OTHER

Testing of hepatic AAV Vector integration

Intervention Type DIAGNOSTIC_TEST

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

Interventions

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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

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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UC Davis Health

Sacramento, California, United States

Site Status RECRUITING

UC Davis Ambulatory Care Clinic

Sacramento, California, United States

Site Status RECRUITING

UC Davis Hemophilia Treatment Center

Sacramento, California, United States

Site Status RECRUITING

UC Davis Medical Center

Sacramento, California, United States

Site Status RECRUITING

UCSF Outpatient Hematology Clinic

San Francisco, California, United States

Site Status NOT_YET_RECRUITING

USF Health Morsani Center For Advanced Healthcare

Tampa, Florida, United States

Site Status RECRUITING

Mississippi Center For Advanced Medicine

Madison, Mississippi, United States

Site Status TERMINATED

Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, United States

Site Status RECRUITING

The Childrens Hospital of Philadelphia Division of Hematology

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Washington Institute for Coagulation

Seattle, Washington, United States

Site Status NOT_YET_RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Site Status RECRUITING

Ege Universitesi Hastanesi

Izmir, İ̇zmir, Turkey (Türkiye)

Site Status RECRUITING

Countries

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United States Australia Turkey (Türkiye)

Central Contacts

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Pfizer CT.gov Call Center

Role: CONTACT

1-800-718-1021

References

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Berntorp E, Shapiro AD. Modern haemophilia care. Lancet. 2012 Apr 14;379(9824):1447-56. doi: 10.1016/S0140-6736(11)61139-2. Epub 2012 Mar 27.

Reference Type BACKGROUND
PMID: 22456059 (View on PubMed)

WFH guidelines: https://www1.wfh.org/publication/files/pdf-1472.pdf

Reference Type BACKGROUND

Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. No abstract available.

Reference Type BACKGROUND
PMID: 25059285 (View on PubMed)

Blanchette VS, McCready M, Achonu C, Abdolell M, Rivard G, Manco-Johnson MJ. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia. 2003 May;9 Suppl 1:19-26; discussion 26. doi: 10.1046/j.1365-2516.9.s1.12.x.

Reference Type BACKGROUND
PMID: 12709033 (View on PubMed)

Lillicrap D. Extending half-life in coagulation factors: where do we stand? Thromb Res. 2008;122 Suppl 4:S2-8. doi: 10.1016/S0049-3848(08)70027-6.

Reference Type BACKGROUND
PMID: 18929522 (View on PubMed)

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=C0371017

To obtain contact information for a study center near you, click here.

Other Identifiers

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NCT05568719

Identifier Type: REGISTRY

Identifier Source: secondary_id

C0371017

Identifier Type: -

Identifier Source: org_study_id

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