Could a gout drug help kids with a rare brain condition?
NCT ID NCT03776656
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the drug allopurinol in 8 children and adults with adenylosuccinate lyase deficiency (ADSL), a rare genetic disorder that can cause autism, seizures, and developmental delays. Researchers measured changes in thinking, daily skills, and seizure control over 12 months. The goal was to see if allopurinol can improve quality of life by reducing harmful metabolites and easing symptoms.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Department of Pediatry. Reference centre of Hereditary diseases of the metabolism of child and adult. Necker - Enfants malades Hospital
Paris, 75015, France
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LA PITIE-SALPETRIERE Hospital, AP-HP
Paris, 75013, France