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Prospective pilot study of L-dopa treatment in patients with a neurodevelopmental disorder linked to a pathogenic variant of the CTNNB1 gene
EU CTIS ID: 2024-514852-34-01
What this study is testing
Evaluate the effect of treatment on patients' motor skills by assessing their motor skills before and after treatment with the 88-item Gross Motor Function Measure (GMFM-88).
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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
You likely can't join if
- 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.
See the full eligibility criteria
- 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
- 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.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.