Summary

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

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 objectives of the study are to compare the annualized bleeding rate [ABR] of the gene therapy to routine prophylaxis from the lead-in study and to evaluate the impact that it may have on participant's Factor IX circulating activity [FIX:C].

Official Title

PHASE 3, OPEN LABEL, SINGLE ARM STUDY TO EVALUATE EFFICACY AND SAFETY OF FIX GENE TRANSFER WITH PF-06838435 (RAAV-SPARK100-HFIX-PADUA) 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 BeneGene 2 Hematological Diseases Annualized bleeding rate (ABR) Hemophilia A PF-06838435/ fidanacogene elaparvovec

Eligibility

You can join if…

Open to males ages 18-65

  • Males who completed 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004) and have > = 50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product
  • Moderately severe to severe hemophilia B (Factor IX activity < =2%)
  • Suspension of prophylaxis therapy for hemophilia B after administration of the study drug
  • Laboratory values (hemoglobin, platelets and creatinine) within study specified limits
  • Agree to contraception until components of the drug are eliminated from their body
  • Capable of giving signed informed consent

You CAN'T join if...

  • Anti-AAV Spark100 neutralizing antibodies (nAb) titer, above the established threshold of 1:1
  • History of inhibitor to Factor IX or inhibitor detected during screening.
  • Clinical signs or symptoms of decreased response to Factor IX
  • Hypersensitivity to Factor IX replacement product or intravenous immunoglobulin administration
  • History of chronic infection or other chronic disease
  • Any concurrent clinically significant major disease or condition
  • Laboratory values at screening visit that are abnormal or outside acceptable study limits
  • Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
  • Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 12 months from screening visit
  • Use of restricted therapies (e.g., blood products, acetylsalicylic acid [aspirin] or ibuprofen, other investigational therapy, and by-passing agents)
  • Participation in a gene therapy research trial at any time or in an interventional clinical study within 12 weeks of screening visit
  • Active hepatitis B or C; hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV) deoxyribonucleic acid (DNA) positivity, or hepatitis C virus (HCV) ribonucleic acid (RNA) positivity
  • Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 and/or viral load >20 copies/mL
  • Study and sponsor staff and their families involved in the conduct of the study
  • Unable to comply with study procedures
  • Sensitivity to heparin or heparin induced thrombocytopenia
  • Sensitivity to any of the study interventions, or components thereof, or drug or other allergy

Locations

  • UCSF IDS Pharmacy accepting new patients
    San Francisco California 94143 United States
  • University of California, San Francisco - Clinical Research Center accepting new patients
    San Francisco California 94143 United States
  • University of California, San Francisco - Outpatient Hematology Clinic accepting new patients
    San Francisco California 94143 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT03861273
Phase
Phase 3
Study Type
Interventional
Last Updated