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Prospective pilot study of L-dopa treatment in CTNNB1-related neurodevelopmental disorder
2024-514852-34-01Phase 2Authorised
- Sponsor
- Centre Hospitalier Universitaire de Montpellier
- Start
- Aug 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of levodopa and carbidopa in Neurodevelopmental disorder (NDD) and dystonia, sponsored by Centre Hospitalier Universitaire de Montpellier. Primary endpoint: Change in GMFM-88 motor score at 6 months. Planned start Aug 2025; status: authorised.
02
Drugs
levodopacarbidopa
03
Conditions
Neurodevelopmental disorder (NDD)dystonia
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