Status:
COMPLETED
Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT
Lead Sponsor:
University of Iowa
Collaborating Sponsors:
National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Dystrophy Association
Conditions:
Charcot Marie Tooth Disease
Eligibility:
All Genders
Up to 21 years
Brief Summary
The primary goal of this project is to develop and test a Charcot Marie Tooth disease (CMT) Pediatric Scale for use in evaluation in natural history CMT study.
Detailed Description
This project is to develop a new CMT Pediatric Scale (CMTPeds) for Children with CMT. Although there is a validated score (the CMTNS) which measures disease severity for CMT, it is not always applicab...
Eligibility Criteria
Inclusion
- All patients MUST be seen in person at one of the participating centers for enrollment in this study.
- Children (\< 21 years of age)
- Known or probable inherited neuropathies classified as CMT1, CMT2, or CMT4
Exclusion
- Known diagnoses of acquired neuropathy including toxic (e. g. medication related neuropathies); metabolic (e.g. diabetic), immune mediated or inflammatory \[acute inflammatory demyelinating polyradiculoneuropathy (AIDP) or chronic inflammatory demyelinating polyneuropathy (CIDP)\] polyneuropathies; neuropathy related to leukodystrophy, congenital muscular dystrophy; and patients with severe general medical conditions.
- Entirely normal conduction velocities of upper and lower limbs as this suggests that the subject may not have a neuropathy.
Key Trial Info
Start Date :
April 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 1 2025
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT01203085
Start Date
April 1 2010
End Date
September 1 2025
Last Update
October 7 2025
Active Locations (14)
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1
Stanford University
Palo Alto, California, United States, 94305
2
University of Connecticut/Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
3
Nemours Children's Clinic
Orlando, Florida, United States, 32827
4
University of Iowa
Iowa City, Iowa, United States, 52242