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Gene Therapy Trial Report

Summary

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


NCTID NCT04370054 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name Giroctogogene fitelparvovec
Sponsor Pfizer
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 77 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant F8
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Liver
Delivery System Viral transduction
Vector Type AAV2/6
Editor Type none
Dose 1 Phase 1/2: 9E11 vg/kg (n=2)
Dose 2 Phase 1/2: 2E12 vg/kg (n=2)
Dose 3 Phase 1/2: 1E13 vg/kg (n=2)
Dose 4 Phase 1/2: 3E13 vg/kg (n=5)
Dose 5 Phase 3: 3E13 vg/kg (n=75)

Study Record Dates


Current Stage Phase3
Submit Date 2020-04-21
Completion Date 2028-10-25
Last Update 2025-08-17

Participation Criteria


Eligible Age 18 Years - 64 Years
Standard Ages Adult
Sexes Eligible for Study MALE
Eligibility 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 36
Locations United States,Japan,United Kingdom,Spain,Greece,Saudi Arabia,Canada,South Korea,Sweden,Taiwan,Turkey (Türkiye),Brazil,Italy,Australia,France,Germany

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo

Resources/Links