Status:
COMPLETED
Biomarkers and Validation of Selected Outcome Measures (CMTNSmod)
Lead Sponsor:
University Medical Center Goettingen
Collaborating Sponsors:
University Hospital Muenster
Ludwig-Maximilians - University of Munich
Conditions:
Charcot-Marie-Tooth Disease, Type IA
Eligibility:
All Genders
3-65 years
Brief 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 pr...
Detailed Description
Novel treatments are being developed for CMT. Intervention efficacy evaluation is hampered by slow disease progression and lack of sensitive outcome measures. The investigators have previously shown t...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 11 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 30 2019
Estimated Enrollment :
156 Patients enrolled
Trial Details
Trial ID
NCT03386266
Start Date
August 11 2017
End Date
September 30 2019
Last Update
November 5 2020
Active Locations (1)
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1
University Medical Center Goettingen
Goettigen, Lower Saxony, Germany, 37075