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

Last updated date
Study Location
Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
Rio de Janeiro, , 20211-030, Brazil
Contact
1-800-718-1021

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By email

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Hemophilia B
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18-65 years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- 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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Advanced Information
Descriptive Information
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
 (submitted: March 1, 2019)
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

  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Australia,   Brazil,   Canada,   France,   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
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP