Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
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
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
(Show More)
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
Clinical Publications
(Abstract) One year data from ambulatory boys in a phase 1b, open-label study of fordadistrogene movaparvovec (PF-06939926) for Duchenne muscular dystrophy (DMD) - MDA 2022
(Video) CIFFREO Data Discussion 10 OCT 2024 EM USA DMD 0099
News and Press Releases
Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
SEC Form 10-Q: Pfizer, Inc. 2Q2024
Preclinical Publications
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
Related NCTID
Phase 1: NCT03362502
Phase 2: NCT05429372
Long Term Follow-Up: NCT05689164