Prospective pilot study of L-dopa treatment in patients with a neurodevelopmental disorder linked to a pathogenic variant of the CTNNB1 gene
Date de révision : 29/04/2026
7 participants
Homme Femme
Jusqu'à 17 ans
Critères :
Critères d'inclusion :
- Ages 1 to 15 inclusive
- Carrier of a pathogenic variant of CTNNB1.
- Patient presenting with dystonia.
- Patient willing to comply with the contraception requirements outlined in the protocol
Critères d'exclusion :
- Contraindication to treatment with L-dopa and carbidopa or one of its excipients
- Current treatment with L-dopa, dopamine agonist or dopamine blocker,
- Failure to obtain informed consent signed by both parents or legal guardians, and to obtain the child's assent if possible.
- Patient not enrolled in or benefiting from a social security scheme
- Person participating in another research project with an exclusion period still in progress
- Patient presenting with a gastro-duodenal ulcer.
- Patient presenting with open-angle glaucoma
- Patient presenting with orthostatic hypotension
- Person who is pregnant or who wishes to become pregnant within 12 months after inclusion.
Lieux et contacts
Informations pratiques pour participer à cet essai.
Contacts
Sites des essais
Adresse
Centre Hospitalier Universitaire De Montpellier
34295 Montpellier Cedex 5 France
Comment postuler ?
Pour participer à cet essai clinique, veuillez contacter la personne responsable de l'étude. Vous trouverez ses coordonnées dans la section "Lieux et contacts", puis "Contacts".