Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexMale
Age18+
SponsorPfizer
About this trial
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
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
None
Trial design
Design model
Single group
Treatments tested in this trial
Testing of hepatic AAV Vector integration
Diagnostic testEvaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated
Treatment groups
173 Participants
are divided into 2 treatment groupsGroup A: Hemophilia A / giroctocogene fitelparvovecOther 1 intervention
Group B: Hemophilia B / fidanacogene elaparvovecOther 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of thromboembolic events
Time frame
Day 1 to 10 years2
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 years3
Incidence of hepatic malignancy
Time frame
Day 1 to 10 years4
Incidence of liver abnormalities
Time frame
Day 1 to 10 yearsSecondary outcomes
1
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 years2
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 years3
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.
Time frame
Day 1 to 10 years4
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 Source ClinicalTrials.gov