Clinical trial NCT05568719: Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hemophilia A
Hemophilia B
Keywords: bleeding
adeno-associated virus based vector
gene therapy
giroctocogene fitelparvovec
fidanacogene elaparvovec
factor VIII
factor IX
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-10-06
Enrollment: 263
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: Non-investigational study
Primary purpose: OTHER

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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of thromboembolic events
timeFrame: Day 1 to 10 years
measure: Incidence of factor inhibitor development
timeFrame: Day 1 to 10 years
measure: Incidence of hepatic malignancy
timeFrame: Day 1 to 10 years
measure: Incidence of liver abnormalities
timeFrame: Day 1 to 10 years
measure: Factor activity level
timeFrame: Day 1 to 10 years
Secondary outcomes:
measure: Total ABR (treated or untreated; (excluding bleeds related to surgery)
timeFrame: Day 1 to 10 years
measure: Incidence of and time from vector infusion to resumption of prophylaxis
timeFrame: Day 1 to 10 years
measure: AIR of exogenous factor (excluding infusions related to surgery)
timeFrame: Day 1 to 10 years
measure: Consumption of exogenous factor (excluding infusions related to surgery)
timeFrame: Day 1 to 10 years
measure: Incidence of Non-hepatic malignancy
timeFrame: Day 1 to 10 years
measure: Incidence of Auto-immune disorders
timeFrame: Day 1 to 10 years
measure: Incidence of SAEs
timeFrame: Day 1 to 10 years
measure: All cause mortality
timeFrame: Day 1 to 10 years
measure: EQ-5D-5L dimension and VAS scores
timeFrame: Day 1 to 10 years
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Health
state: California
city: Sacramento
country: United States
facility: UC Davis Ambulatory Care Clinic
state: California
city: Sacramento
country: United States
facility: UC Davis Hemophilia Treatment Center
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: San Francisco
country: United States
facility: UCSF Outpatient Hematology Clinic
state: California
city: Tampa
country: United States
facility: USF Health Morsani Center For Advanced Healthcare
state: Florida
city: Madison
country: United States
facility: Mississippi Center For Advanced Medicine
state: Mississippi
city: Philadelphia
country: United States
facility: The Childrens Hospital of Philadelphia Division of Hematology
state: Pennsylvania
city: Seattle
country: United States
facility: Washington Institute for Coagulation
state: Washington
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: İzmir
country: Turkey
facility: Ege Universitesi Hastanesi
state: İ̇zmir
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: 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.
citation: WFH guidelines: https://www1.wfh.org/publication/files/pdf-1472.pdf
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.
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.
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.