Brief Title
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
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)
Brief 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].
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Annualized bleeding rate (ABR)
Secondary Outcome
Annualized infusion rate (AIR) of exogenous Factor IX Activity
Condition
Hemophilia B
Intervention
PF-06838435/ fidanacogene elaparvovec
Study Arms / Comparison Groups
PF-06838435/ fidanacogene elaparvovec
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
55
Start Date
July 29, 2019
Completion Date
May 23, 2027
Primary Completion Date
May 25, 2022
Eligibility Criteria
Inclusion Criteria - 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 Exclusion Criteria - 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 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) - 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 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
Gender
Male
Ages
18 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Pfizer CT.gov Call Center, 1-800-718-1021, [email protected]
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03861273
Organization ID
C0371002
Secondary IDs
2018-003086-33
Responsible Party
Sponsor
Study Sponsor
Pfizer
Study Sponsor
Pfizer CT.gov Call Center, Study Director, Pfizer
Verification Date
December 2020