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Treatment for Charcot-Marie-Tooth Disease (INC-6601 Trial)

N/A
Recruiting
Led By Michael E Shy, MD
Research Sponsored by Michael Shy
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Summary

This is an observational longitudinal study to determine the natural history and genotype-phenotype correlations of disease causing mutations in Charcot Marie Tooth disease (CMT) type 1B (CMT1B), 2A (CMT2A), 4A (CMT4A), and 4C (CMT4C). The investigators will also be determine the capability of the newly developed CMT Pediatric Scale (CMT Peds scale) and the Minimal Dataset to measure impairment and perform longitudinal measurements in patients with multiple forms of CMT over a five year window

Eligible Conditions
  • Charcot-Marie-Tooth Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Charcot Marie Tooth Neuropathy Score (CMTNS)
Minimal dataset

Find a Location

Who is running the clinical trial?

The National Hospital for Neurology and NeurosurgeryUNKNOWN
Nemours Children's HospitalUNKNOWN
3 Previous Clinical Trials
540 Total Patients Enrolled
1 Trials studying Charcot-Marie-Tooth Disease
200 Patients Enrolled for Charcot-Marie-Tooth Disease
University of MinnesotaOTHER
1,413 Previous Clinical Trials
1,556,403 Total Patients Enrolled
2 Trials studying Charcot-Marie-Tooth Disease
1,250 Patients Enrolled for Charcot-Marie-Tooth Disease
~650 spots leftby Dec 2026
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