Summary

Eligibility
for males ages 18-65 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

Summary

This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).

Official Title

Phase 3, Open-label, Single-arm Study to Evaluate Efficacy and Safety of FIX Gene Transfer With PF-06838435 (rAAV-Spark100-hFIX-R338L) in Adult Male Participants With Moderately Severe to Severe Hemophilia B (FIX:C =2%) (BeneGene-2)

Keywords

Hemophilia B, PF 06838435, Hemophilia, Factor IX, FIX, Gene Therapy, Infusion, rAAV Spark100 hFIX Padua, rAAV Spark100-hFIX-R338L, R338L, BeneGene 2, Hematological Diseases, Annualized bleeding rate (ABR), Hemophilia A, PF-06838435/ fidanacogene elaparvovec

Eligibility

Locations

  • UCSF - Clinical Research Center
    San Francisco California 94143 United States
  • UCSF - Outpatient Hematology Clinic
    San Francisco California 94143 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
ID
NCT03861273
Phase
Phase 3 Hemophilia Research Study
Study Type
Interventional
Participants
About 51 people participating
Last Updated