L-dopa Treatment for Children With CTNNB1 Gene Disorder
Treatments studied
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests whether L-dopa medication can help children with a rare genetic disorder (caused by changes in the CTNNB1 gene) that causes involuntary muscle movements called dystonia. Researchers want to see if this medicine can reduce these movements and improve daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is between 1 and 15 years old
- Child has been diagnosed with a pathogenic (disease-causing) variant in the CTNNB1 gene
- Child has dystonia (involuntary muscle contractions or repetitive movements)
- Child is not currently taking L-dopa, dopamine agonists, or dopamine blockers
- Child does not have peptic ulcers, glaucoma, or orthostatic hypotension (dizziness upon standing)
- Both parents or legal guardians must provide written consent, and the child must agree to participate if possible
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study uses a new DNA reading method to find hidden genetic causes of dystonia. It is for people with dystonia who had standard genetic testing that did not find a cause.
This study tests how a medicine called trihexyphenidyl works in children with dystonic cerebral palsy, a condition that causes muscle spasms. Researchers want to learn if a child’s genes affect how the medicine works and how the body processes it.
This study looks at ways to improve how the brain processes movement in children and young people with dystonia. You may be asked to complete study tasks while researchers compare people with dystonia to similar children without movement disorders.
This trial tests a one-time gene replacement therapy given into the fluid around the brain to help children with CTNNB1 neurodevelopmental syndrome. The goal is to see if replacing the faulty gene can improve development and reduce symptoms.
This study looks at how different types of dystonia (movement disorders) relate to genetics and brain findings from scans. You might help researchers understand why symptoms look different from person to person and what patterns may matter.
This study measures brain signals in children with dystonia to understand how the brain controls movement, with the goal of improving treatments like Deep Brain Stimulation (DBS). It also uses healthy children for comparison.
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