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Clinical Trial Report

Gene Therapy Trial Report

Summary

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy


NCTID NCT04281485 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name PF-06939926
Compound Alias Fordadistrogene movaparvovec
Compound Description AAV9.hCK.Hopti-Dys3978.spA
Sponsor Pfizer
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 122
Results Posted Not Available

Therapy Information


Target Gene/Variant Mini-dystrophin
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
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 2E14 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase3
Submit Date 2020-02-11
Completion Date 2039-04-15
Last Update 2025-01-13

Participation Criteria


Eligible Age 4 Years - 7 Years
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 49
Locations Russian Federation,United States,Japan,United Kingdom,Switzerland,Spain,Canada,Belgium,Taiwan,Korea, Republic of,Italy,Israel,Australia,France,Germany

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Phase 3 study did not meet its primary efficacy endpoint, discontinuation was announced Q3 2024

Resources/Links