Trial record 1 of 1 for:
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
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 |
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Recruitment Status :
Recruiting
First Posted : April 30, 2020
Last Update Posted : May 3, 2023
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Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A | Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy | Phase 3 |
| 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 : | July 1, 2024 |
| Estimated Study Completion Date : | July 30, 2028 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
| Arm | Intervention/treatment |
|---|---|
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Experimental: PF-07055480 (giroctocogene fitelparvovec)
Single administration of PF-07055480
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Biological: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Other Name: Gene Therapy |
Primary Outcome Measures :
- Total annualized bleeding rate (ABR) [ Time Frame: 15 months ]
Secondary Outcome Measures :
- 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: 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 ]
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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 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
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
| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
Locations
Show 38 study locations
Sponsors and Collaborators
Pfizer
Investigators
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
Additional Information:
| 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: | May 3, 2023 |
| Last Verified: | May 2023 |
| 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 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Pfizer:
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PF-07055480 SB-525 Hemophilia Factor VIII FVIII Gene Therapy |
AAV AAV6 Annualized bleeding rate ABR giroctocogene fitelparvovec AFFINE |
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 |