Multi-omic search for early biomarkers in CMT1A
A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A) (CMT-MODs)
University Medical Center Goettingen · NCT07476365
This project will test whether blood-based molecular signatures and qMRI measures in children and young adults with CMT1A can predict how the disease will progress.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 70 (estimated) |
| Ages | 10 Years to 30 Years |
| Sex | All |
| Sponsor | University Medical Center Goettingen (other) |
| Locations | 1 site (Göttingen) |
| Trial ID | NCT07476365 on ClinicalTrials.gov |
What this trial studies
This observational project collects blood samples and quantitative MRI from children, adolescents, and young adults (age 10–30) with genetically confirmed or family-diagnosed CMT1A. Researchers will perform transcriptomics and other multi-omic analyses on blood and correlate those signals with MRI biomarkers and clinical measures. Biomarker candidates will be informed by prior findings in CMT1A rat models and adult patient samples to build predictive models of future disease course. The aim is to find easily obtainable prognostic markers usable before peripheral nerves undergo advanced degeneration.
Who should consider this trial
Good fit: Ideal candidates are cooperative patients aged 10–30 with a genetic diagnosis of CMT1A (or clinical diagnosis plus an affected relative) who are able to walk with or without support.
Not a fit: Patients with other neuromuscular disorders, contraindications to qMRI, or those outside the 10–30 age range may not be eligible or likely to benefit from this protocol.
Why it matters
Potential benefit: If successful, this work could allow earlier identification of patients at higher risk of progression, enabling timely interventions and more personalized care planning.
How similar studies have performed: Preclinical data from CMT1A rats and gene-expression correlations in adult CMT1A patients support this multi-omic approach, but applying it in young patients is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion criteria: * collaborative children, adolescents and young adults aged 10-30 years * genetic diagnosis of CMT1A, or clinical diagnosis and genetic diagnosis in affected relatives * able to walk with/ without support. Exclusion Criteria: * neuromuscular disorders other than CMT1A * concomitant disease preventing correct patient evaluation and contraindication to qMRI
Where this trial is running
Göttingen
- University Medical Centre — Göttingen, Germany (RECRUITING)
Study contacts
- Principal investigator: Michael W Sereda — University Medical Centre Göttingen
- Study coordinator: Michael W Sereda, Prof. of Neurology
- 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.
Conditions: CMT, CMT - Charcot-Marie-Tooth Disease, CMT1A, CMT 1A, CMT1A children, blood-derived prognostic biomarkers, MRI biomarkers, transcriptomics