TerminatedPhase 2

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

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

United States, Australia10 participantsStarted 2022-08-08

Plain-language summary

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

Who can participate

Age range

2 Years – 3 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Confirmed diagnosis of DMD by prior genetic testing. Exclusion Criteria: * 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. * 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. * Abnormality in specified laboratory tests, including blood counts, liver and kidney function.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events

Timeframe: Through Week 52

Number of participants with abnormal hematology test results

Timeframe: Through Week 52

Number of participants with abnormal biochemistry test results

Timeframe: Through Week 52

Number of participants with abnormal urine analysis

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in neurological examinations

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in body weight

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in vital signs

Timeframe: Through Week 52

Trial details

NCT IDNCT05429372

SponsorPfizer

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-10-03

View on ClinicalTrials.gov More Muscular Dystrophy, Duchenne trials

Who can participate

Age range

2 Years – 3 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events

Timeframe: Through Week 52

Number of participants with abnormal hematology test results

Timeframe: Through Week 52

Number of participants with abnormal biochemistry test results

Timeframe: Through Week 52

Number of participants with abnormal urine analysis

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in neurological examinations

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in body weight

Timeframe: Through Week 52

Number of participants with abnormal and clinically relevant changes in vital signs

Timeframe: Through Week 52