NCT07125898Recruiting
Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
Source: ClinicalTrials.gov · Last updated 22 Sept 2026
Brief summary (registry, in English)
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 110 (estimated)
- First submitted
- 11 Aug 2025
- Start date
- 16 Sept 2026
- Primary completion date
- Sept 2028
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Diseases
- Autism
- NCT number
- NCT07125898
What you commit to
- Study type: Observational
- Planned participants: 110
- From 16 Sept 2026 to the primary completion date, Sept 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment with methylphenidate: In case of hyperactive patients, the treatment will be transitorily interrupted the week before testing.
Lab values
- Cardiac dysfunction with left ventricular ejection fraction \< 35%,
Other
- Severe intellectual deficiency with IQ \< 55, and IQ \< 70 for the deep neurocognitive evaluation (executive and socio-cognitive evaluation),
- Cataract except if operated (pseudophakic),
- High intraocular pressure,
- Respiratory dysfunction with force vital capacity \< 70%,
- Difficulties in fine motor skills with D3 MFM scale \< 75%
Who the study is looking for
- French citizenship, affiliated to the French Social Security,
- 5 to 12 years old,
- DMD diagnosis confirmed by a genetic analysis predicting breaking in the reading frame of the DMD gene with knowledge of the limits of the mutation,
- Follow-up in a French referral or a skills center belonging to Filnemus.
Age in the registry: 5 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hopital Necker Enfants Malades Coordinator of this site? Confirm its status | Paris | France | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.