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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

Last updated on December 5, 2019

FOR MORE INFORMATION
Study Location
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045 United States
Contact
1-800-718-1021
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) and have > = 50 documented exposure days to a FIX protein product
such as recombinant, plasma-derived or extended half-life FIX product

- Moderately severe to severe hemophilia B (Factor IX activity

- 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, above the established threshold
of 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)

- Participation 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

NCT03861273
Cheladv74
Recruiting
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

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[email protected]

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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 SummaryThis 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 DescriptionNot 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 July 21, 2026
Estimated Primary Completion DateJuly 23, 2021   (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) and have > = 50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product
  • 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, above the established threshold of 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)
  • Participation 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: Cheladv74 CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Australia,   Canada,   Turkey,   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 CommitteeNot 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:Cheladv74 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 Cheladv74's data sharing criteria and process for requesting access can be found at: http://cheladv74.ru/science/clinical_trials/trial_data_and_results/da....
URL:http://cheladv74.ru/science/clinical_trials/trial_data_and_results/da...
Responsible PartyCheladv74
Study Sponsor  ICMJE Cheladv74
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Cheladv74 CT.gov Call CenterCheladv74
PRS AccountCheladv74
Verification DateDecember 2019

ICMJE     Data element required by the
and the

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Contact a representative by phone, email, or visiting the study website. Please see the references below:

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Cheladv74 Clinical Trials Contact Center

1-800-718-1021

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Contact

[email protected]

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