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 range

3 Years – 65 Years

Sex

ALL

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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