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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 (BENEGENE-2)

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ClinicalTrials.gov Identifier: NCT03861273
Recruitment Status : Recruiting
First Posted : March 4, 2019
Last Update Posted : August 26, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Description
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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].

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: PF-06838435/ fidanacogene elaparvovec Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 55 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : July 29, 2019
Estimated Primary Completion Date : July 23, 2021
Estimated Study Completion Date : July 21, 2026

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Arms and Interventions
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Arm Intervention/treatment
Experimental: PF-06838435/ fidanacogene elaparvovec Biological: PF-06838435/ fidanacogene elaparvovec
Gene Therapy


Outcome Measures
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Primary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: First 12 months post PF 06838435 infusion ]
  2. Vector derived FIX:C level [ Time Frame: Week 12 to 12 months post PF 06838435 infusion ]

Secondary Outcome Measures :
  1. Annualized infusion rate (AIR) of exogenous Factor IX Activity [ Time Frame: First 12 months post study drug infusion ]
  2. Annualized Factor IX Activity consumption [ Time Frame: 12 months post study drug infusion ]
  3. Annualized number of bleeding events of specific type: spontaneous and traumatic, and untreated [ Time Frame: 12 months post study drug infusion ]
  4. Frequency of target joint bleeds [ Time Frame: 12 months post study drug infusion ]
  5. Percentage of the participants without bleeds [ Time Frame: 12 months post study drug infusion ]
  6. Change in joint health as measured by the Hemophilia Joint Health Score (HJHS) instrument [ Time Frame: 12 months post study drug infusion ]
  7. Patient Reported Outcome (PRO) instrument - Hemophilia Quality of Life (Haem A QoL) [ Time Frame: 12 months post study drug infusion ]
  8. Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: 12 months post study drug infusion ]
  9. Patient Reported Outcome (PRO) instrument - Patient Global Impression of Change-Hemophilia (PGIC-H) [ Time Frame: 12 months post study drug infusion ]
  10. Annualized Bleeding Rate [ Time Frame: Annually for 6 years ]
  11. Vector derived Factor IX activity (FIX:C) level at steady state [ Time Frame: Annually for 6 years ]
  12. Annualized infusion rate (AIR) of exogenous Factor IX [ Time Frame: Annually for 6 years ]
  13. Annualized Factor IX consumption [ Time Frame: Annually for 6 years ]
  14. Annualized number of bleeding events of specific type: spontaneous and traumatic, and untreated [ Time Frame: Annually for 6 years ]
  15. Frequency of target joint bleeds [ Time Frame: Annually for 6 years ]
  16. Patient Reported Outcome (PRO) instrument - Hemophilia Quality of Life (Haem A QoL) [ Time Frame: Annually for 6 years ]
  17. Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: Annually for 6 years ]
  18. Patient Reported Outcome (PRO) instrument - Patient Global Impression of Change - Hemophilia (PGIC-H) [ Time Frame: Annually for 6 years ]
  19. Incidence and severity of all adverse events collected during the study [ Time Frame: For the duration of 6 years after PF-06838435 infusion ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03861273


Contacts
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Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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United States, Mississippi
Mississippi Center for Advanced Medicine Recruiting
Madison, Mississippi, United States, 39110
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:
To obtain contact information for a study center near you, click here.  This link exits the ClinicalTrials.gov site

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03861273     History of Changes
Other Study ID Numbers: C0371002
2018-003086-33 ( EudraCT Number )
First Posted: March 4, 2019    Key Record Dates
Last Update Posted: August 26, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
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/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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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
Keywords provided by Pfizer:
PF 06838435
Hemophilia
Factor IX
FIX
Gene Therapy
 Infusion
rAAV Spark100 hFIX Padua
BeneGene 2
Hematological Diseases
Annualized bleeding rate (ABR)
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked