Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
Recruiting
N/A
Interventional Study
Charcot-Marie-Tooth Disease Type 1A
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
At a Glance
- Age
- 10 – 30
- Sex
- Any
- Study type
- Interventional
- Purpose
- Other
- Participants needed
- 55 (estimated)
- Sponsor
- Assistance Publique Hopitaux De Marseille · Other
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About This Trial
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 patien…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* 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…
Contacts