An observational study in Dystrophinopathies, sponsored by Assistance Publique - Hôpitaux de Paris. Recruiting at 1 site in France. Open to participants aged 5 Years to 12 Years. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Assistance Publique - Hôpitaux de Paris · Observational
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.
We propose a unique longitudinal study in which DMD children aged 5-12 years old will first be engaged in a deep evaluation of a range of cognitive, behavioral, physiological and neural functions (identification of biomarkers based on ERG) and an eligible subgroup of patients will then enter a second study phase (last 2 years) aimed at developing targeted cognitive remediation strategies:
399 studies on the registry are indexed under Cognition Disorders; 74 are open to participants now.
This study's planned enrollment of 110 is below the median of 137 across 108 observational studies indexed under Cognition Disorders.
Browse Cognition Disorders studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,592 studies on the registry; 1,033 are open to participants now.
Counted across the registry records on this site, refreshed daily.
DMD diagnosis confirmed by a genetic analysis
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
DMD diagnosis confirmed by a genetic analysis
DMD diagnosis confirmed whose neuropsychological data are eligible for remediation
Nature and severity of sensory and neuropsychological disturbances
Specify the nature and severity of sensory and neuropsychological disturbances (cognitive, executive, emotional, behavioral and neuropsychiatric) according to the patient's genotype.
Time frame: 12 Months
Correlations between sensory and neuropsychological measures
Analyze the correlations between sensory (retinal/visual) and neuropsychological measures, in order to determine if retinal defects represent a signature of neuropsychological impairment severity.
Time frame: 24 Months
Correlation between neuropsychological and functional imaging parameters
Analyze correlation between neuropsychological and functional imaging parameters.
Time frame: 24 Months
Correlation between sensory and functional imaging parameters
Analyze correlation between sensory and functional imaging parameters.
Time frame: 24 Months
Plan to share: No
No publications or documents are linked to this record.
Started Sep 2026 (actual). Primary completion is estimated for Sep 2028, 24 months after the start.
Of 43 non-industry observational studies under Cognition Disorders, half reached primary completion within 41 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 89. This study's planned 24 months is below that.
Counting every sponsor, half of 44 studies reached it within 41 months; a quarter within 13 and three quarters within 89.
How these figures are worked out, and which studies are left out of them.
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