ClinicalTrials.gov

History of Changes for Study: NCT04370054
Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults (AFFINE)
Latest version (submitted May 12, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 April 29, 2020 None (earliest Version on record)
2 May 18, 2020 Study Status
3 August 11, 2020 Study Status and Contacts/Locations
4 September 3, 2020 Recruitment Status, Study Status and Contacts/Locations
5 October 28, 2020 Study Status and Contacts/Locations
6 December 7, 2020 Study Status and Contacts/Locations
7 February 8, 2021 Study Status and Contacts/Locations
8 March 18, 2021 Study Status and Contacts/Locations
9 April 7, 2021 Study Status and Contacts/Locations
10 May 12, 2021 Study Status and Contacts/Locations
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Changes (Side-by-Side) for Study: NCT04370054
February 8, 2021 (v7) -- March 18, 2021 (v8)

Changes in: Study Status and Contacts/Locations

Open or close this module Study Identification
Unique Protocol ID: C3731003 C3731003
Brief Title: Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults (AFFINE)Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults (AFFINE)
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%) 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%)
Secondary IDs: 2019-004451-37 [EudraCT Number]2019-004451-37 [EudraCT Number]
Open or close this module Study Status
Record Verification: February 2021 March 2021
Overall Status: RecruitingRecruiting
Study Start: August 18, 2020 August 18, 2020
Primary Completion: August 12, 2022 [Anticipated ] August 12, 2022 [Anticipated ]
Study Completion: October 29, 2026 [Anticipated ] October 29, 2026 [Anticipated ]
First Submitted: April 21, 2020 April 21, 2020
First Submitted that
Met QC Criteria:
April 29, 2020 April 29, 2020
First Posted: April 30, 2020 [Actual ] April 30, 2020 [Actual ]
Last Update Submitted that
Met QC Criteria:
February 8, 2021 March 18, 2021
Last Update Posted: February 10, 2021 [Actual ] March 22, 2021 [Actual ]
Open or close this module Sponsor/Collaborators
Sponsor: Pfizer Pfizer
Responsible Party: Sponsor Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: YesYes
U.S. FDA-regulated Device: NoNo
Data Monitoring: Yes Yes
Open or close this module Study Description
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. 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.
Detailed Description:
Open or close this module Conditions
Conditions: Hemophilia A Hemophilia A
Keywords: PF-07055480
SB-525
Hemophilia
Factor VIII
FVIII
Gene Therapy
AAV
AAV6
Annualized bleeding rate
ABR
PF-07055480
SB-525
Hemophilia
Factor VIII
FVIII
Gene Therapy
AAV
AAV6
Annualized bleeding rate
ABR
Open or close this module Study Design
Study Type: InterventionalInterventional
Primary Purpose: TreatmentTreatment
Study Phase: Phase 3Phase 3
Interventional Study Model: Single Group Assignment Single Group Assignment
Number of Arms: 11
Masking: None (Open Label)None (Open Label)
Allocation: N/AN/A
Enrollment: 63 [Anticipated ] 63 [Anticipated ]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: PF-07055480
Single administration of PF-07055480
Biological: Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Other Names:
  • Gene Therapy
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Annualized bleeding rate (ABR)

[Time Frame: 12 months ]
Annualized bleeding rate (ABR)

[Time Frame: 12 months ]
Secondary Outcome Measures:
2. Steady state FVIII activity levels

[Time Frame: 12 months ]
Steady state FVIII activity levels

[Time Frame: 12 months ]
3. Annualized infusion rate (AIR) of exogenous Factor VIII Activity

[Time Frame: 12 months ]
Annualized infusion rate (AIR) of exogenous Factor VIII Activity

[Time Frame: 12 months ]
4. Annualized FVIII consumption

[Time Frame: 12 months ]
Annualized FVIII consumption

[Time Frame: 12 months ]
5. Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location

[Time Frame: 12 months ]
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location

[Time Frame: 12 months ]
6. Change in joint health using HJHS (Hemophilia Joint Health Score)
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.

[Time Frame: 12 months ]
Change in joint health using HJHS (Hemophilia Joint Health Score)
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.

[Time Frame: 12 months ]
7. Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL)
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.

[Time Frame: 12 months ]
Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL)
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.

[Time Frame: 12 months ]
8. Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
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.

[Time Frame: 12 months ]
Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
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.

[Time Frame: 12 months ]
9. Incidence and severity of AEs

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

[Time Frame: 5-year study period ]
Open or close this module Eligibility
Minimum Age: 18 Years 18 Years
Maximum Age: 64 Years 64 Years
Sex: Male Male
Gender Based: Yes Yes
Accepts Healthy Volunteers: NoNo
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

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
Open or close this module Contacts/Locations
Central Contact Person: Pfizer CT.gov Call Center
Telephone: 1-800-718-1021
Email: ClinicalTrials.gov_Inquiries@pfizer.com
Pfizer CT.gov Call Center
Telephone: 1-800-718-1021
Email: ClinicalTrials.gov_Inquiries@pfizer.com
Study Officials: Pfizer CT.gov Call Center
Study Director
Pfizer
Pfizer CT.gov Call Center
Study Director
Pfizer
Locations: United States, CaliforniaUnited States, California
UCSF IDS Pharmacy
[Recruiting]
San Francisco, California, United States, 94143
UCSF IDS Pharmacy
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Clinical Research Center
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Clinical Research Center
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Moffitt/Long Inpatient Hematology
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Moffitt/Long Inpatient Hematology
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Outpatient Hematology Clinic
[Recruiting]
San Francisco, California, United States, 94143
University of California, San Francisco - Outpatient Hematology Clinic
[Recruiting]
San Francisco, California, United States, 94143
United States, WashingtonUnited States, Washington
Bloodworks NW
[Recruiting]
Seattle, Washington, United States, 98104
Bloodworks NW
[Recruiting]
Seattle, Washington, United States, 98104
University of Washington Medical Center - Translational Research Unit (TRU)
[Recruiting]
Seattle, Washington, United States, 98195
University of Washington Medical Center - Translational Research Unit (TRU)
[Recruiting]
Seattle, Washington, United States, 98195
Korea, Republic of
Kyung Hee University Hospital at Gangdong
[Not yet recruiting]
Seoul, Korea, Republic of, 05278
Taiwan
National Taiwan University Hospital
[Not yet recruiting]
Taipei, Taiwan, 10002
TurkeyTurkey
Adana Acibadem Hospital
[Not yet recruiting]
Adana, Turkey, 01130
Adana Acibadem Hospital
[Not yet recruiting]
Adana, Turkey, 01130
Gaziantep University Sahinbey Research and Training Hospital
[Not yet recruiting]
Gaziantep, Turkey, 27310
Ege University Medical Faculty, Pediatric Hematology
[Not yet recruiting]
Izmir, Turkey, 35100
Ege University Medical Faculty
[Not yet recruiting]
Izmir, Turkey, 35100
Open or close this module IPDSharing
Plan to Share IPD: Yes
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.
Yes
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.
Supporting Information:
Supporting Information:
Time Frame:
Time Frame:
Access Criteria:
Access Criteria:
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requestsURL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Open or close this module References
Citations:
Links:
Description: To obtain contact information for a study center near you, click here.
Description: To obtain contact information for a study center near you, click here.
Available IPD/Information:

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