Hemophilia APhase 3NCT05568719
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
A Phase 3 study testing Testing of hepatic AAV Vector integration in people with Hemophilia A and Hemophilia B, run by Pfizer at 39 sites in 13 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- Testing of hepatic AAV Vector integration
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 173 participants
- Where
- 39 sitesin 13 countries
- Study dates
- 28 Dec 2022to 25 Feb 2040
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- -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.
Who cannot join
- -None
Trial locations and contacts
39 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
39 sites in 13 countries
United States15 sites, 14 recruiting
- Recruiting
Innovative Hematology, Inc.
Indianapolis, Indiana, 46260
- Terminated
Mississippi Center For Advanced Medicine
Madison, Mississippi, 39110
- Recruiting
Cornell University||Joan & Sanford I. Weill Medical College
New York, New York, 10065
- Recruiting
Weill Cornell Medical College-New York Presbyterian Hospital
New York, New York, 10065
- Recruiting
The Childrens Hospital of Philadelphia Division of Hematology
Philadelphia, Pennsylvania, 19104
- Recruiting
The Childrens Hospital of Philadelphia Division of Hematology
Philadelphia, Pennsylvania, 19146
- Recruiting
The Regents of the University of California||UC Davis Health
Rancho Cordova, California, 95670
- Recruiting
UC Davis Health
Sacramento, California, 95816
- Recruiting
UC Davis Ambulatory Care Clinic
Sacramento, California, 95817
- Recruiting
UC Davis Hemophilia Treatment Center
Sacramento, California, 95817
- Recruiting
UC Davis Medical Center
Sacramento, California, 95817
- Recruiting
UCSF Outpatient Hematology Clinic
San Francisco, California, 94143
- Recruiting
Washington Institute for Coagulation
Seattle, Washington, 98101
- Recruiting
USF Health Morsani Center For Advanced Healthcare
Tampa, Florida, 33612
- Recruiting
The University of South Florida Board of Trustees
Tampa, Florida, 33620
Taiwan5 sites, 5 recruiting
- Recruiting
Changhua Christian Hospital
Changhua, 500
- Recruiting
Chung-Ho Memorial Hospital, Kaohsiung Medical University
Kaohsiung City, 807
- Not yet recruiting
Chung Shan Medical University Hospital
Taichung, 402
- Recruiting
Taichung Veterans General Hospital
Taichung, 40705
- Recruiting
National Taiwan University Hospital
Taipei, 10041
Turkey (Türkiye)4 sites, 4 recruiting
- Recruiting
Acibadem Adana Hospital
Adana, 01130
- Recruiting
Gaziantep Universitesi Sahinbey Arastirma ve Uygulama Hastanesi
Gaziantep, 27310
- Recruiting
İstanbul Üniversitesi Onkoloji Enstitüsü
Istanbul, 34093
- Recruiting
Ege Universitesi Hastanesi
Izmir, İ̇zmir, 35100
Australia3 sites, 3 recruiting
- Recruiting
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
- Recruiting
Sydney Local Health District (RPAH Zone)
Camperdown, New South Wales, 2050
- Recruiting
The Alfred Hospital
Melbourne, Victoria, 3004
Japan3 sites, 3 recruiting
- Recruiting
Saitama Medical University Hospital
Iruma-gun, Saitama, 350-0495
- Recruiting
Nagoya University Hospital
Nagoya, Aichi-ken, 466-8560
- Recruiting
Sapporo Tokushukai Hospital
Sapporo, Hokkaido, 004-0041
France2 sites, 2 recruiting
- Recruiting
Hospices Civils de Lyon - Hopital Louis Pradel
Bron, Rhône, 69677
- Recruiting
Hôpital Universitaire Necker Enfants Malades
Paris, 75015
Canada1 site, 1 recruiting
- Recruiting
Unity Health Toronto, St. Michael's Hospital
Toronto, Ontario, M5B 1W8
Germany1 site, 1 recruiting
- Recruiting
Universitätsklinikum Bonn
Bonn, North Rhine-Westphalia, 53127
Greece1 site, 1 recruiting
- Recruiting
General Hospital of Athens Laiko
Athens, 11527
Saudi Arabia1 site, 1 recruiting
- Not yet recruiting
King Faisal Specialist Hospital and Research Center
Riyadh, 11211
South Korea1 site, 1 recruiting
- Recruiting
Kyung Hee University Hospital at Gangdong
Seoul, 05278
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario Rio Hortega
Valladolid, 47012
About this study
Official title: 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.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- Testing of hepatic AAV Vector integration
- Keywords
- bleeding, adeno-associated virus based vector, gene therapy, giroctocogene fitelparvovec, fidanacogene elaparvovec, factor VIII, factor IX
- Medical subject headings
- Hemophilia A, Hemophilia B, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Diagnostic Test: Testing of hepatic AAV Vector integration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of thromboembolic events
Measured: 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).
Measured: Day 1 to 10 years
Incidence of hepatic malignancy
Measured: Day 1 to 10 years
Incidence of liver abnormalities
Measured: 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.
Measured: Day 1 to 10 years
Secondary outcomes (9)
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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: Day 1 to 10 years
Incidence of Non-hepatic malignancy
Measured: Day 1 to 10 years
Incidence of Auto-immune disorders
Measured: 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.
Measured: 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.
Measured: 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
Measured: Day 1 to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2022
- Study start28 Dec 2022
- Primary completion25 Feb 2040 (estimated)
- Study completion25 Feb 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Non-investigational study
- Masking
- None (open label)
- Enrolment
- 173 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05568719
- Sponsor's protocol ID
- C0371017
- Other IDs
- NCT05568719
- Status last verified
- Oct 2026
- First submitted
- 3 Oct 2022
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
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.
PubMed 22456059WFH 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.
PubMed 25059285Blanchette 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.
PubMed 12709033Lillicrap 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.
PubMed 18929522