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Trial record 1 of 1 for:    NCT04370054
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Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

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ClinicalTrials.gov Identifier: NCT04370054
Recruitment Status : Recruiting
First Posted : April 30, 2020
Last Update Posted : September 4, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 (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 completed at least 6 months of routine prophylaxis with FVIII products in the lead-in study C0371004.

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: Recombinant AAV2/6 Human Factor VIII Gene Therapy Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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%)
Actual Study Start Date : August 18, 2020
Estimated Primary Completion Date : August 12, 2022
Estimated Study Completion Date : October 29, 2026


Arm Intervention/treatment
Experimental: PF-07055480
Single administration of PF-07055480
Biological: Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Other Name: Gene Therapy




Primary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Steady state FVIII activity levels [ Time Frame: 12 months ]
  2. Annualized infusion rate (AIR) of exogenous Factor VIII Activity [ Time Frame: 12 months ]
  3. Annualized FVIII consumption [ Time Frame: 12 months ]
  4. Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location [ Time Frame: 12 months ]
  5. 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.

  6. 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.

  7. 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.

  8. Incidence and severity of AEs [ Time Frame: 5-year study period ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Main inclusion Criteria

  • Males who completed 6 months of 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
  • 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

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): NCT04370054


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, Washington
Bloodworks NW Recruiting
Seattle, Washington, United States, 98104
University of Washington Medical Center - Translational Research Unit (TRU) Recruiting
Seattle, Washington, United States, 98195
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT04370054    
Other Study ID Numbers: C3731003
2019-004451-37 ( EudraCT Number )
First Posted: April 30, 2020    Key Record Dates
Last Update Posted: September 4, 2020
Last Verified: September 2020
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
Keywords provided by Pfizer:
PF-07055480
SB-525
Hemophilia
Factor VIII
FVIII
Gene Therapy
AAV
AAV6
Annualized bleeding rate
ABR
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants