NCT05568719

Brief 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.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
173

participants targeted

Target at P25-P50 for phase_3

Timeline
167mo left

Started Dec 2022

Longer than P75 for phase_3

Geographic Reach
7 countries

23 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress20%
Dec 2022Feb 2040

First Submitted

Initial submission to the registry

October 3, 2022

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 6, 2022

Completed
3 months until next milestone

Study Start

First participant enrolled

December 28, 2022

Completed
17.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 25, 2040

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 25, 2040

Last Updated

May 27, 2026

Status Verified

May 1, 2026

Enrollment Period

17.2 years

First QC Date

October 3, 2022

Last Update Submit

May 25, 2026

Conditions

Hemophilia AHemophilia B

Keywords

bleedingadeno-associated virus based vectorgene therapygiroctocogene fitelparvovecfidanacogene elaparvovecfactor VIIIfactor IX

Outcome Measures

Primary Outcomes (5)

  • Incidence of thromboembolic events

    Day 1 to 10 years

  • Incidence of factor inhibitor development

    FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).

    Day 1 to 10 years

  • Incidence of hepatic malignancy

    Day 1 to 10 years

  • Incidence of liver abnormalities

    Day 1 to 10 years

  • Factor activity level

    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.

    Day 1 to 10 years

Secondary Outcomes (9)

  • Total ABR (treated or untreated; (excluding bleeds related to surgery)

    Day 1 to 10 years

  • Incidence of and time from vector infusion to resumption of prophylaxis

    Day 1 to 10 years

  • AIR of exogenous factor (excluding infusions related to surgery)

    Day 1 to 10 years

  • Consumption of exogenous factor (excluding infusions related to surgery)

    Day 1 to 10 years

  • Incidence of Non-hepatic malignancy

    Day 1 to 10 years

  • +4 more secondary outcomes

Study Arms (2)

Hemophilia A / giroctocogene fitelparvovec

OTHER

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

Diagnostic Test: Testing of hepatic AAV Vector integration

Hemophilia B / fidanacogene elaparvovec

OTHER

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

Diagnostic Test: Testing of hepatic AAV Vector integration

Interventions

Testing of hepatic AAV Vector integrationDIAGNOSTIC_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 A / giroctocogene fitelparvovecHemophilia B / fidanacogene elaparvovec

Eligibility Criteria

Age18 Years+
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (23)

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

Rancho Cordova, California, 95670, United States

RECRUITING

UC Davis Health

Sacramento, California, 95816, United States

RECRUITING

UC Davis Ambulatory Care Clinic

Sacramento, California, 95817, United States

RECRUITING

UC Davis Hemophilia Treatment Center

Sacramento, California, 95817, United States

RECRUITING

UC Davis Medical Center

Sacramento, California, 95817, United States

RECRUITING

UCSF Outpatient Hematology Clinic

San Francisco, California, 94143, United States

NOT YET RECRUITING

USF Health Morsani Center For Advanced Healthcare

Tampa, Florida, 33612, United States

RECRUITING

The University of South Florida Board of Trustees

Tampa, Florida, 33620, United States

RECRUITING

Mississippi Center For Advanced Medicine

Madison, Mississippi, 39110, United States

TERMINATED

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

New York, New York, 10065, United States

RECRUITING

Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, 10065, United States

RECRUITING

The Childrens Hospital of Philadelphia Division of Hematology

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

The Childrens Hospital of Philadelphia Division of Hematology

Philadelphia, Pennsylvania, 19146, United States

RECRUITING

Washington Institute for Coagulation

Seattle, Washington, 98101, United States

NOT YET RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

RECRUITING

Sydney Local Health District (RPAH Zone)

Camperdown, New South Wales, 2050, Australia

RECRUITING

Unity Health Toronto, St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

NOT YET RECRUITING

Kyung Hee University Hospital at Gangdong

Seoul, 05278, South Korea

NOT YET RECRUITING

Skåne University Hospital

Malmö, 21428, Sweden

NOT YET RECRUITING

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

NOT YET RECRUITING

National Taiwan University Hospital

Taipei, 10041, Taiwan

NOT YET RECRUITING

Ege Universitesi Hastanesi

Izmir, İ̇zmir, 35100, Turkey (Türkiye)

RECRUITING

Acibadem Adana Hospital

Adana, 01130, Turkey (Türkiye)

NOT YET RECRUITING

Related Publications (5)

  • 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.

    PMID: 22456059BACKGROUND

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

    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.

    PMID: 25059285BACKGROUND

  • 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.

    PMID: 12709033BACKGROUND

  • 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.

    PMID: 18929522BACKGROUND

Related Links

MeSH Terms

Conditions

Hemophilia AHemophilia BHemorrhage

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Central Study Contacts

Pfizer CT.gov Call Center

CONTACT

1-800-718-1021ClinicalTrials.gov_Inquiries@pfizer.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Model Details: Non-investigational study
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 3, 2022

First Posted

October 6, 2022

Study Start

December 28, 2022

Primary Completion (Estimated)

February 25, 2040

Study Completion (Estimated)

February 25, 2040

Last Updated

May 27, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will share

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

More information

Locations

KR(1)SE(1)AU(2)TW(2)TU(2)US(14)CA(1)