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Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

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ClinicalTrials.gov Identifier: NCT04370054
Recruitment Status : Active, not recruiting
First Posted : April 30, 2020
Last Update Posted : March 4, 2024
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE April 21, 2020
First Posted Date  ICMJE April 30, 2020
Last Update Posted Date March 4, 2024
Actual Study Start Date  ICMJE August 18, 2020
Estimated Primary Completion Date June 17, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 16, 2022)
Total annualized bleeding rate (ABR) [ Time Frame: 15 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 29, 2020)
Annualized bleeding rate (ABR) [ Time Frame: 12 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 11, 2023)
  • FVIII activity levels [ Time Frame: 15 months ]
  • Annualized bleeding rate (ABR) [ Time Frame: 15 months ]
  • Annualized infusion rate (AIR) of exogenous Factor VIII Activity [ Time Frame: 15 months ]
  • Annualized FVIII consumption [ Time Frame: 15 months ]
  • Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location [ Time Frame: 15 months ]
  • Change in joint health using HJHS (Hemophilia Joint Health Score) [ Time Frame: Yearly up to 5 years ]
    HJHS is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints.
  • Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: Yearly up to 5 years ]
    HAL is a disease specific measure of the impact of hemophilia on functional abilities in adults. The instrument consists of 42 items across 7 domains, utilizing a past month recall period. Each item is rated on a scale of 0-6 with higher recoded scores indicating more functional limitations.
  • Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) [ Time Frame: Yearly up to 5 years ]
    Haem-A-QoL is a disease specific measure of health-related quality of life in patients with hemophilia. Intended for adults, the instrument uses a 4-week recall period to assess health across 10 domains consisting of 46 items. Each item is rated on a scale of 0-6 with lower scores indicating better health-related quality of life.
  • Incidence and severity of AEs [ Time Frame: 5-year study period ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 29, 2020)
  • Steady state FVIII activity levels [ Time Frame: 12 months ]
  • Annualized infusion rate (AIR) of exogenous Factor VIII Activity [ Time Frame: 12 months ]
  • Annualized FVIII consumption [ Time Frame: 12 months ]
  • Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location [ Time Frame: 12 months ]
  • Change in joint health using HJHS (Hemophilia Joint Health Score) [ Time Frame: 12 months ]
    HJHS is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints.
  • Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: 12 months ]
    HAL is a disease specific measure of the impact of hemophilia on functional abilities in adults. The instrument consists of 42 items across 7 domains, utilizing a past month recall period. Each item is rated on a scale of 0-6 with higher recoded scores indicating more functional limitations.
  • Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) [ Time Frame: 12 months ]
    Haem-A-QoL is a disease specific measure of health-related quality of life in patients with hemophilia. Intended for adults, the instrument uses a 4-week recall period to assess health across 10 domains consisting of 46 items. Each item is rated on a scale of 0-6 with lower scores indicating better health-related quality of life.
  • Incidence and severity of AEs [ Time Frame: 5-year study period ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Official Title  ICMJE Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants With Moderately Severe to Severe Hemophilia A(FVIII:C≤1%)
Brief Summary C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia A
Intervention  ICMJE Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Other Name: Gene Therapy
Study Arms  ICMJE Experimental: PF-07055480 (giroctocogene fitelparvovec)
Single administration of PF-07055480
Intervention: Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 11, 2023)
76
Original Estimated Enrollment  ICMJE
 (submitted: April 29, 2020)
63
Estimated Study Completion Date  ICMJE October 25, 2028
Estimated Primary Completion Date June 17, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main inclusion Criteria

  • Males who have been followed on routine Factor VIII prophylaxis therapy during the lead-in study (C0371004) and have > = 150 documented exposure days to a Factor VIII protein product
  • Moderately severe to severe hemophilia A (Factor VIII activity < =1%)
  • Suspension of FVIII prophylaxis therapy post study drug infusion

Main exclusion Criteria

  • Anti-AAV6 neutralizing antibodies
  • History of inhibitor to Factor VIII
  • Laboratory values at screening visit that are abnormal or outside acceptable study limits
  • Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
  • Conditions associated with increased thromboembolic risk such as inherited or acquired thrombophilia, or a history of thrombotic events
  • Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
  • Active hepatitis B or C
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 18 Years to 64 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Brazil,   Canada,   France,   Germany,   Greece,   Italy,   Japan,   Korea, Republic of,   Saudi Arabia,   Spain,   Sweden,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04370054
Other Study ID Numbers  ICMJE C3731003
2019-004451-37 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
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/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Current Responsible Party Pfizer
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Pfizer
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date March 2024

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