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

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05568719
• Other study ID #: C0371017
• Status: Recruiting
• Phase: Phase 3
• Start date: December 28, 2022
• Est. completion date: April 8, 2038

Study information

• Verified date: May 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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. Data collection and participant visits will be based on standard of care.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 263
• Est. completion date: April 8, 2038
• Est. primary completion date: April 8, 2038
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

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

Locations

Country	Name	City	State
Australia	Royal Prince Alfred Hospital	Camperdown	New South Wales
Turkey	Ege Universitesi Hastanesi	Izmir	I?zmir
United States	Mississippi Center For Advanced Medicine	Madison	Mississippi
United States	The Childrens Hospital of Philadelphia Division of Hematology	Philadelphia	Pennsylvania
United States	Washington Institute for Coagulation	Seattle	Washington
United States	USF Health Morsani Center For Advanced Healthcare	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia,  Turkey, 

References & 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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of thromboembolic events		Day 1 to 10 years
Primary	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
Primary	Incidence of hepatic malignancy		Day 1 to 10 years
Primary	Incidence of liver abnormalities		Day 1 to 10 years
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Incidence of Non-hepatic malignancy		Day 1 to 10 years
Secondary	Incidence of Auto-immune disorders		Day 1 to 10 years
Secondary	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
Secondary	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
Secondary	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

External Links

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