Clinical Trials in France / NCT07125898
Recruiting
Observational
Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
NCT07125898 · tracked via the Priya Life Science France tracker
Condition(s) studied
Dystrophinopathies
Study summary
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.
Eligibility
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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,
* Cardiac dysfunction with left ventricular ejection fraction \< 35%,
* Respiratory dysfunction with force vital capacity \< 70%,
* Difficulties in fine motor skills with D3 MFM scale \< 75%
* Treatment with methylphenidate: In case of hyperactive patients, the treatment will be transitorily interrupted the week before testing.
Primary outcome measure(s)
- Nature and severity of sensory and neuropsychological disturbances — 12 Months
Specify the nature and severity of sensory and neuropsychological disturbances (cognitive, executive, emotional, behavioral and neuropsychiatric) according to the patient's genotype.
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Hôpital Necker Enfants Malades | Paris | Île-de-France Region | Recruiting |
More Assistance Publique - Hôpitaux de Paris trials in France
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07125898 on ClinicalTrials.gov ↗ ← All trials in France