A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

a study on Hemophilia

Summary

Eligibility
for males ages 18-65 (full criteria)
Location
at San Francisco, California and other locations
Dates
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 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-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) prior to providing consent at the screening visit for this study.
  • Documented 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 >= 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 15 months from screening visit
  • Use of restricted therapies (e.g., blood products, acetylsalicylic acid [aspirin] or ibuprofen, other investigational therapy, and by-passing agents)
  • Previously dosed 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 infection with either Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 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 - Clinical Research Center accepting new patients
    San Francisco California 94143 United States
  • UCSF - 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 Hemophilia Research Study
Study Type
Interventional
Participants
Expecting 55 study participants
Last Updated
Contact us about this trial