Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults
NCT04370054
Last updated date
ABOUT THIS STUDY
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.
Study Location
University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
By phone
Pfizer Clinical Trials Contact Center
1-800-718-1021
Eligibility Criteria
condition
Hemophilia A
Sex
Male
Age
18-64 years
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Advanced Information
Descriptive Information | |||||||
---|---|---|---|---|---|---|---|
Brief Title ICMJE | Study to Evaluate the Efficacy and Safety of PF-07055480 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 (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 | 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: Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion Other Name: Gene Therapy | ||||||
Study Arms ICMJE | Experimental: PF-07055480
Single administration of PF-07055480 Intervention: Biological: 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 | Recruiting | ||||||
Estimated Enrollment ICMJE | 63 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Estimated Study Completion Date ICMJE | October 29, 2026 | ||||||
Estimated Primary Completion Date | August 12, 2022 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Main inclusion Criteria
Main exclusion Criteria
| ||||||
Sex/Gender ICMJE |
| ||||||
Ages ICMJE | 18 Years to 64 Years (Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE |
| ||||||
Listed Location Countries ICMJE | 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 |
| ||||||
IPD Sharing Statement ICMJE |
| ||||||
Responsible Party | Pfizer | ||||||
Study Sponsor ICMJE | Pfizer | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
| ||||||
PRS Account | Pfizer | ||||||
Verification Date | December 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |