Actively Recruiting
Longitudinal Biomarkers With Selected Outcome Measures In CMT
Led by University Medical Center Goettingen · Updated on 2026-03-09
75
Participants Needed
1
Research Sites
86 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The goal of this study is to better understand the progression of CMT1A and identify risk factors influencing disease course. CMT1A, the most common hereditary peripheral neuropathy, shows high variability in individual phenotypes despite genetic similarity. Key objectives include analyzing determinants of phenotypic expression and documenting symptom variability over five years to capture disease dynamics. Although incurable, novel CMT therapies are in development. Proving efficacy is challenging due to slow progression and limited sensitive outcome measures. This study aims to validate biomarkers (DNA/epigenetics and RNA/RT-PCR) and sensitive outcome measures from blood and skin of CMT patients over five years to support clinical therapy trials. Approximately 25 healthy volunteers will serve as controls, providing blood and skin samples for biomarker validation. Additionally, the project will build a tissue collection (skin, blood, and cultured fibroblasts) from CMT patients of various subtypes for unrestricted scientific research, especially for the German CMT-NET network (NCT03386266). Scientific partners have free access to samples and data for research (commercial use is excluded). Currently, this collection includes over 100 standardized skin biopsies from CMT1A patients and is Germany's only repository for hereditary neuropathy tissue samples.
CONDITIONS
Official Title
Longitudinal Biomarkers With Selected Outcome Measures In CMT
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of CMT or healthy control group
- Genetic confirmation of CMT in adult patients
- Ability to complete the outcome measure at baseline
- Age between 18 and 65 years
- Ability to consent and provide signed informed consent
You will not qualify if you...
- Pregnancy or breastfeeding
- Presence of other relevant neurological or psychiatric disorders, current or past
- History of serious internal disease
AI-Screening
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Trial Site Locations
Total: 1 location
1
University Medical Centre
Göttingen, Lower Saxony, Germany, 37075
Actively Recruiting
Research Team
M
Michael W Sereda, Prof. MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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