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

NCT03861273

Active Not RecruitingPhase 3

Sponsor: Pfizer

Conditions: Hemophilia B

Interventions: PF-06838435/ fidanacogene elaparvovec

Countries: United States, Australia, Brazil, Canada, France, Germany, Greece, Italy

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).

Eligibility overview

Sex: MALE

Age: 18 Years to 65 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria

* Males who completed 6 months of 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%)
* Previous experience with FIX therapy (=\>50 documented exposure days to a FIX protein product)
* 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-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold (ie, positive for nAb).
* 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 IV immunoglobulin administration
* History of chronic infection or other chronic disease
* Any conditions associated with increased thromboembolic risk
* Concurrent clinically significant major disease or condition unsuitable for participation and/or may interfere with the interpretation of study results
* Laboratory values at screening visit that are abnormal or outside acceptable study limits
* Current unstable liver or biliary disease
* Currently on antiviral therapy for hepatitis B or C
* 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, hepatitis B virus deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity
* Significant liver disease
* Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count \<=200 mm3 and/or a viral load \>20 copies/mL
* Study and sponsor staff involved in the conduct of the study and their families
* 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 (67)
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • Aurora, Colorado, United States
  • Aurora, Colorado, United States
  • Indianapolis, Indiana, United States
  • Indianapolis, Indiana, United States
  • Indianapolis, Indiana, United States
  • Indianapolis, Indiana, United States
  • Madison, Mississippi, United States
  • Madison, Mississippi, United States
  • Hamilton, Pennsylvania, United States
  • Philadelphia, Pennsylvania, United States
  • Philadelphia, Pennsylvania, United States
  • Philadelphia, Pennsylvania, United States
  • Philadelphia, Pennsylvania, United States
  • Camperdown, New South Wales, Australia
  • Adelaide, South Australia, Australia
  • Melbourne, Victoria, Australia
  • Murdoch, Western Australia, Australia
  • Herston, Australia
  • Vitória, Espírito Santo, Brazil
  • Campinas, São Paulo, Brazil
  • São Paulo, São Paulo, Brazil
  • Rio de Janeiro, Brazil
  • Hamilton, Ontario, Canada
  • Hamilton, Ontario, Canada
  • Toronto, Ontario, Canada
  • Bron, France
  • + 37 more on CT.gov