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

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.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia A; Hemophilia B
• Intervention / Treatment: Diagnostic test: Testing of hepatic AAV Vector integration

• Study Type: Interventional
• Enrollment (Estimated): 173
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Pfizer CT.gov Call Center; Phone Number: 1-800-718-1021; Email: ClinicalTrials.gov_Inquiries@pfizer.com

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Recruiting
      • Royal Prince Alfred Hospital
    • Camperdown, New South Wales, Australia, 2050
      • Recruiting
      • Sydney Local Health District (RPAH Zone)
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5B 1W8
      • Not yet recruiting
      • Unity Health Toronto, St. Michael's Hospital
• South Korea
  • Seoul, South Korea, 05278
    • Not yet recruiting
    • Kyung Hee University Hospital at Gangdong
• Sweden
  • Malmö, Sweden, 21428
    • Not yet recruiting
    • Skane University Hospital
• Taiwan
  • Taichung, Taiwan, 40705
    • Not yet recruiting
    • Taichung Veterans General Hospital
  • Taipei, Taiwan, 10041
    • Not yet recruiting
    • National Taiwan University Hospital
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01130
    • Not yet recruiting
    • Acibadem Adana Hospital
  • İ̇zmir
    • Izmir, İ̇zmir, Turkey (Türkiye), 35100
      • Recruiting
      • Ege Universitesi Hastanesi
• United States
  • California
    • Rancho Cordova, California, United States, 95670
      • Recruiting
      • The Regents of the University of California||UC Davis Health
    • Sacramento, California, United States, 95817
      • Recruiting
      • UC Davis Medical Center
    • Sacramento, California, United States, 95816
      • Recruiting
      • UC Davis Health
    • Sacramento, California, United States, 95817
      • Recruiting
      • UC Davis Hemophilia Treatment Center
    • Sacramento, California, United States, 95817
      • Recruiting
      • UC Davis Ambulatory Care Clinic
    • San Francisco, California, United States, 94143
      • Not yet recruiting
      • UCSF Outpatient Hematology Clinic
  • Florida
    • Tampa, Florida, United States, 33612
      • Recruiting
      • USF Health Morsani Center for Advanced Healthcare
    • Tampa, Florida, United States, 33620
      • Recruiting
      • The University of South Florida Board of Trustees
  • Mississippi
    • Madison, Mississippi, United States, 39110
      • Terminated
      • Mississippi Center for Advanced Medicine
  • New York
    • New York, New York, United States, 10065
      • Recruiting
      • Weill Cornell Medical College-New York Presbyterian Hospital
    • New York, New York, United States, 10065
      • Recruiting
      • Cornell University||Joan & Sanford I. Weill Medical College
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • The Childrens Hospital of Philadelphia Division of Hematology
    • Philadelphia, Pennsylvania, United States, 19146
      • Recruiting
      • The Childrens Hospital of Philadelphia Division of Hematology
  • Washington
    • Seattle, Washington, United States, 98101
      • Not yet recruiting
      • Washington Institute for Coagulation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Study Population

Male participants over the age of 18 with Hemophilia A or Hemophilia B who have received prior treatment with investigational giroctocogene fitelparvovec or fidanacogene eleparvovec, respectively.

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: 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	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
Other: 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	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total ABR (treated or untreated; (excluding bleeds related to surgery)	ABR (Annual Bleed Rate): number of bleeding episodes per year. This includes treated and untreated bleeds. The ABR or the annualized number of bleeding episodes per year, will be derived for each participant for each observation period by using the following formula: ABR = (Number of bleeds / Days in observation period) x 365.25 days/year.	Day 1 to 10 years
Incidence of and time from vector infusion to resumption of prophylaxis	Describe incidence of resumption of prophylaxis resumption and the time (in days) to resumption of prophylaxis after receiving vector infusion.	Day 1 to 10 years
AIR of exogenous factor (excluding infusions related to surgery)	The AIR or the annualized number of FIX infusions per year, will be derived for each participant for each observation period by using the following formula: AIR = (Number of FIX infusions / Days in observation period) x 365.25 days/year.	Day 1 to 10 years
Consumption of exogenous factor (excluding infusions related to surgery)	The annualized TFC in international units (IU) will be derived for each participant for each observation period using the following formula: Annualized TFC = (Total units of FIX infused (IU)/ Days in observation period) x 365.25 days/year	Day 1 to 10 years
Incidence of Non-hepatic malignancy		Day 1 to 10 years
Incidence of Auto-immune disorders		Day 1 to 10 years
Incidence of SAEs	An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; development of a clinical thrombotic event; development of factor inhibitor; development of a hepatic malignancy; development of drug-related elevated hepatic transaminases that fail to improve with immunosuppressive regimens; occurrence of a malignancy with reasonable possibility of being related to study drug.	Day 1 to 10 years
All cause mortality	All-cause mortality was defined as the death due to any cause during the course of study. Incidence rate was defined as the total number of participants with admissible events divided by the total (for all qualifying participants) time at risk for the cohort/treatment group of interest. Incidence rate of all-cause deaths was reported in this outcome measure.	Day 1 to 10 years
EQ-5D-5L dimension and VAS scores	The EQ-5D-5L comprises a 5-item health status measure and a visual analog rating scale/feeling thermometer. Using the 5-dimensional Health State Classification, participants are asked to respond to five questions on different aspects of their health status that assess the following: Mobility; Self-care; Usual activities; Pain/Discomfort; Anxiety/Depression	Day 1 to 10 years

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

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

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): December 28, 2022
• Primary Completion (Estimated): February 25, 2040
• Study Completion (Estimated): February 25, 2040

Study Registration Dates

• First Submitted: October 3, 2022
• First Submitted That Met QC Criteria: October 3, 2022
• First Posted (Actual): October 6, 2022

Study Record Updates

• Last Update Posted (Actual): May 27, 2026
• Last Update Submitted That Met QC Criteria: May 25, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: bleeding; gene therapy; factor IX; giroctocogene fitelparvovec; factor VIII; adeno-associated virus based vector; fidanacogene elaparvovec
• Additional Relevant MeSH Terms: Pathologic Processes; Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Hemophilia A; Hemorrhage; Hemophilia B
• Other Study ID Numbers: C0371017; NCT05568719 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No