Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Completed

Conditions studied: Charcot Marie Tooth Disease

In brief

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.

Key facts

Study ID

NCT01203085

Run by

University of Iowa

People needed

500

Starts

2010-04-01

Expected to finish

2025-09-01

Last updated by the study team

2025-10-07

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Stanford University — Palo Alto, California, United States
• University of Connecticut/Connecticut Children's Medical Center — Hartford, Connecticut, United States
• Nemours Children's Clinic — Orlando, Florida, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Rochester — Rochester, New York, United States
• University of North Carolina — Chapel Hill, North Carolina, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Children's Hospital of Westmead — Sydney, New South Wales, Australia
• The Hospital for Sick Children — Toronto, Ontario, Canada
• C. Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy
• National Hospital of Neurology and Neurosurgery — London, England, United Kingdom
• Dubowitz Neuromuscular Centre — London, UK, United Kingdom

Full record on ClinicalTrials.gov

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