CompletedNot Applicable

Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)

Germany156 participantsStarted 2017-08-11

Plain-language summary

CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures. Primary goal of the project is to identify and validate RNA and protein derived biomarkers in blood of CMT patients for selected outcome measures over 2 years. The investigators expect to develop more responsive outcome measures and circulating biomarkers to improve assessment of intervention efficacy in forthcoming therapeutic trials.

Who can participate

Age range3 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Clinical diagnosis of CMT1A * Genetic confirmation of PMP22 duplication (for adults patients) * Children aged 3-11, adolescents aged 12-17 and adults aged 18-65 years * Signed informed patient consent Exclusion Criteria: * Other causes of neurological and psychiatric disorders * Severe internistic disease * Patient known or suspected to be alcohol / drug abuser * Pregnancy, breast feeding period * Permanent Vitamin C intake * Participation an interventional clinical study up to 4 weeks prior to inclusion

What they're measuring

mRNA Expression Levels in blood samples from CMT1A patients

Timeframe: 3 years

mRNA Expression Levels in Skin biopsies from CMT1A patients

Timeframe: 3 years

Trial details

NCT IDNCT03386266

SponsorUniversity Medical Center Goettingen

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2019-09-30

View on ClinicalTrials.gov More Charcot-Marie-Tooth Disease, Type IA trials