Longitudinal Biomarkers with Selected Outcome Measures in CMT

Launched by UNIVERSITY MEDICAL CENTER GOETTINGEN · Jan 20, 2025

Keywords

ClinConnect Summary

This clinical trial is focused on understanding Charcot-Marie-Tooth Disease type 1A (CMT1A), a hereditary condition that affects the nerves and can lead to muscle weakness and sensory loss. The goal is to explore how this disease progresses in different individuals and identify factors that may influence how severe or mild the symptoms are. Researchers will follow participants over five years, collecting blood and skin samples to look for biological markers that can help in developing new treatments. They will also compare these samples with those from about 25 healthy volunteers to better understand the disease.

To be eligible for this study, participants should be between 18 and 65 years old and have a confirmed diagnosis of CMT. They should be able to understand and agree to participate in the study. Those who are pregnant, breastfeeding, or have certain other serious health issues will not be able to join. Participants can expect to undergo various assessments and provide samples, contributing to a valuable resource for research that aims to improve care for people with CMT. The study is currently recruiting, and it promises to provide important insights into this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Clinical CMT Diagnosis / Anamnestically Healthy Control Group
• • Genetic confirmation of CMT in adult patients
• • Ability to achieve the outcome measure at baseline
• • Age between 18 and 65 years
• • Capacity of all study participants to consent and signed informed consent, including patient or participant information and consent form
• Exclusion Criteria:
• • Pregnancy or breastfeeding period
• • Other relevant neurological or psychiatric disorders, acute or in the past history
• • Presence of a serious previous internal disease