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
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
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1-800-718-1021
- 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
- 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
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Descriptive Information | |||||||
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Brief Title ICMJE | 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 ICMJE | 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]. | ||||||
Detailed Description | Not Provided | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 3 | ||||||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment | ||||||
Condition ICMJE | Hemophilia B | ||||||
Intervention ICMJE | Biological: PF-06838435/ fidanacogene elaparvovec
Gene Therapy | ||||||
Study Arms ICMJE | Experimental: PF-06838435/ fidanacogene elaparvovec
Intervention: Biological: PF-06838435/ fidanacogene elaparvovec | ||||||
Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Recruiting | ||||||
Estimated Enrollment ICMJE | 55 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | May 23, 2027 | ||||||
Estimated Primary Completion Date | May 25, 2022 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria
Exclusion Criteria
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years to 65 Years (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | Australia, Brazil, Canada, France, Germany, Greece, Japan, Saudi Arabia, Sweden, Taiwan, Turkey, United Kingdom, United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT03861273 | ||||||
Other Study ID Numbers ICMJE | C0371002 2018-003086-33 ( EudraCT Number ) | ||||||
Has Data Monitoring Committee | Not Provided | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Pfizer | ||||||
Study Sponsor ICMJE | Pfizer | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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PRS Account | Pfizer | ||||||
Verification Date | December 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |