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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
173
Evaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated
The Regents of the University of California||UC Davis Health
Rancho Cordova, California, United States
Incidence of thromboembolic events
Time frame: 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).
Time frame: Day 1 to 10 years
Incidence of hepatic malignancy
Time frame: Day 1 to 10 years
Incidence of liver abnormalities
Time frame: 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.
Time frame: Day 1 to 10 years
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.
Time frame: 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.
Time frame: Day 1 to 10 years
AIR of exogenous factor (excluding infusions related to surgery)
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UC Davis Health
Sacramento, California, United States
RECRUITINGUC Davis Ambulatory Care Clinic
Sacramento, California, United States
RECRUITINGUC Davis Hemophilia Treatment Center
Sacramento, California, United States
RECRUITINGUC Davis Medical Center
Sacramento, California, United States
RECRUITINGUCSF Outpatient Hematology Clinic
San Francisco, California, United States
NOT_YET_RECRUITINGUSF Health Morsani Center For Advanced Healthcare
Tampa, Florida, United States
RECRUITINGThe University of South Florida Board of Trustees
Tampa, Florida, United States
RECRUITINGMississippi Center For Advanced Medicine
Madison, Mississippi, United States
TERMINATEDCornell University||Joan & Sanford I. Weill Medical College
New York, New York, United States
RECRUITING...and 7 more locations
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.
Time frame: 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
Time frame: Day 1 to 10 years
Incidence of Non-hepatic malignancy
Time frame: Day 1 to 10 years
Incidence of Auto-immune disorders
Time frame: 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.
Time frame: 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.
Time frame: 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: 1. Mobility 2. Self-care 3. Usual activities 4. Pain/Discomfort 5. Anxiety/Depression
Time frame: Day 1 to 10 years