Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
Recruiting now · Not applicable
Conditions studied: Charcot-Marie-Tooth Disease Type 1A
In brief
This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A using multi-omics analysis. The study is recruiting subjects between the ages of 10 and 30. The most common inherited neuropathy is Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a duplication of the gene expressing PMP22. CMT1A patients develop symptoms in early childhood with variable progression and there is no established therapy until now. Therapy must start in childhood, before peripheral nerves degenerate. However, we lack easily obtainable biomarkers in early disease stages. In CMT-MODs, we will identify disease and prognostic biomarkers in young CMT1A patients.
Key facts
- Study ID
- NCT07049588
- Run by
- Assistance Publique Hopitaux De Marseille
- People needed
- 55
- Starts
- 2025-06-24
- Expected to finish
- 2028-06-24
- Last updated by the study team
- 2025-07-03
Who can join
Age: 10 and older, up to 30. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- Healthy volunteer or patient who has given consent for participation in the study or, for minors, a healthy volunteer whose two parents have given consent for participation in the study.
- Patient with genetically confirmed CMT1A or with a parent whose diagnosis is genetically confirmed
- Patient able to walk with or without assistance
You may not qualify if…
- Healthy volunteer with neurological disorders
- Healthy volunteer or patient with a contraindication to MRI,
- Healthy volunteers or patient under 30 kg
- Helathy volunteer on long-term therapy
- Patient with other neuromuscular pathologies
- Patient in a period of exclusion from another research protocol at the time of signing the consent/non-opposition form
- Pregnant or breast-feeding women
- Subjects covered by articles L1121-5 to 1121-8 of the French Public Health Code (minors, adults under guardianship or trusteeship, patients deprived of their liberty, pregnant or breast-feeding women)
- Subjects who cannot read and understand the French language well enough to be able to give their consent to participate in research
Where it is running
- Assistance Publique - Hôpitaux de Marseille — Marseille, France (enrolling)
Full record on ClinicalTrials.gov
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