Understanding biomarkers and outcomes in Charcot-Marie-Tooth disease
Longitudinal Biomarkers With Selected Outcome Measures In CMT
This study is trying to see how Charcot-Marie-Tooth disease type 1A changes over time by looking at blood and skin samples from patients and healthy people to find better ways to track the disease and develop new treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 75 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | University Medical Center Goettingen Academic / other |
| Locations | 1 site (Göttingen, Lower Saxony) |
| Trial ID | NCT06794489 on ClinicalTrials.gov |
What this trial studies
This observational study aims to investigate the progression of Charcot-Marie-Tooth disease type 1A (CMT1A) by identifying risk factors that influence the disease course. Over five years, researchers will analyze biomarkers from blood and skin samples of CMT patients, alongside a control group of healthy volunteers, to validate sensitive outcome measures. The study seeks to document symptom variability and disease dynamics, which could support the development of novel therapies for this hereditary neuropathy. By correlating molecular analyses with clinical data, the research hopes to establish practical biomarkers for monitoring disease severity and progression.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 to 65 with a clinical diagnosis and genetic confirmation of CMT.
Not a fit: Patients with other significant neurological or psychiatric disorders, or those who are pregnant or breastfeeding, may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to the identification of reliable biomarkers that improve monitoring and treatment outcomes for patients with CMT1A.
How similar studies have performed: While there have been preliminary studies exploring biomarkers in CMT, this approach is novel and aims to establish practical biomarkers for clinical use.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Göttingen, Lower Saxony
- University Medical Centre — Göttingen, Lower Saxony, Germany (Recruiting)
Study contacts
- Study coordinator: Michael W Sereda, Prof. MD
- Email: sereda@mpinat.mpg.de
- Phone: +49 551 3964162
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.