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NCT05429372TerminatedPhase 2

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY

Source: ClinicalTrials.gov · Last updated 21 Oct 2025

Reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003

Brief summary (registry, in English)

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Status (registry)
Terminated
Phase (registry)
Phase 2
Study type
Interventional
Enrollment
10 (actual)
First submitted
8 Oct 2021
Start date
8 Aug 2022
Primary completion date
3 Oct 2025
Lead sponsor
Pfizer
NCT number
NCT05429372
EudraCT number
2021-003379-33

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Positive test performed by Pfizer for neutralizing antibodies to AAV9.
  • Any prior treatment with gene therapy.
  • Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
  • Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.

Lab values

  • Abnormality in specified laboratory tests, including blood counts, liver and kidney function.

Other

  • Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.

Who the study is looking for

  • Confirmed diagnosis of DMD by prior genetic testing.

Age in the registry: 2 years to 3 years · Sex: Male · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (12)

Sites in registry order. Save your city in your profile to sort them by distance.

SiteCityCountrySite status
Perth Children's HospitalNedlandsAustraliaUnknown status
The Royal Children's Hospital MelbourneParkvilleAustraliaUnknown status
The Children's Hospital at WestmeadWestmeadAustraliaUnknown status
University of FloridaGainesvilleUnited StatesUnknown status
UF Health Shands HospitalGainesvilleUnited StatesUnknown status
The Children's Hospital Of PhiladelphiaPhiladelphiaUnited StatesUnknown status
University of Utah HospitalSalt Lake CityUnited StatesUnknown status
University of Utah Clinical Neurosciences CenterSalt Lake CityUnited StatesUnknown status
University of Utah Imaging and Neurosciences CenterSalt Lake CityUnited StatesUnknown status
University of Utah Hospital & Clinics Investigational Drug ServicesSalt Lake CityUnited StatesUnknown status
Primary Children's HospitalSalt Lake CityUnited StatesUnknown status
CTSI Clinical Research CenterSalt Lake CityUnited StatesUnknown status

Are you the coordinator? Confirm the details

This page is information, not medical advice. Discuss any trial with your doctor.