A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
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Phase
Phase 3
Status
Terminated
Study type
Interventional
Design
Na · None · Other
Enrollment
7actual
Sites
12
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Why the sponsor stopped it
All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
As filed on the registry record, in the sponsor's own words.
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2023-03-13 | actual | When the study began enrolling |
| Primary completion | 2025-09-24 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-09-24 | actual | The whole study's end, after follow-up |
| First posted | 2023-01-19 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-10-21 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Duchenne Muscular Dystrophy
Intervention
- Biological: fordadistrogene movaparvovec
Primary outcomes
what the primary-completion date above is the date OFNumber of participants with serious adverse events
measured At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of participants with serious adverse events
measured At least annually from 5 through 10 years after dosing in the interventional study.
Number of participants with adverse events considered related to treatment
measured At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of particpants with adverse events considered related to treatment
measured At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with malignancy adverse event
measured At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in electrocardiogram (ECG) assessments
measured Annually from 5 through 10 years after dosing in the interventional study.
Number of participants with clinically significant findings in cardiac troponin I laboratory examinations
measured Annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in echocardiogram parameters
measured Annually from 5 through 10 years after dosing in the interventional study
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