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

Launched by UNIVERSITY OF IOWA · Sep 15, 2010

Keywords

ClinConnect Summary

This clinical trial is focused on developing a new scale to help doctors evaluate children who have Charcot Marie Tooth disease (CMT). CMT is a genetic condition that affects the nerves and can lead to weakness and difficulty in movement. The researchers want to create a tool that will help them better understand how the disease affects kids over time. The study is currently recruiting participants, and it is open to children under 21 years old who have been diagnosed with certain types of inherited neuropathies known as CMT1, CMT2, or CMT4.

To join the study, children must visit one of the participating centers for an in-person evaluation. Unfortunately, kids with other types of nerve problems, like those caused by medication or certain medical conditions, won't be able to participate. If a child has normal nerve function, that may also mean they aren’t a good fit for this trial. By taking part in this study, participants may help researchers learn more about CMT and improve the way it is measured and treated in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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.