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NCT01193075 · ClinicalTrials.gov registry record
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
A clinical trial of Charcot Marie Tooth Disease, sponsored by Michael Shy.
- Recruiting
- Registry status
- 5,000
- Enrollment target
- 20
- Study locations
NCT01193075: Recruiting study of Charcot Marie Tooth Disease, sponsored by Michael Shy.
NCT01193075 is a study of Charcot Marie Tooth Disease that is actively recruiting participants, run by Michael Shy. The registered enrollment target is 5,000 participants, above the 1,651-participant average among 7 other Charcot Marie Tooth Disease trials with a reported enrollment target (203% higher). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01193075, a study of Charcot Marie Tooth Disease, is actively recruiting participants, sponsored by Michael Shy.
- RECRUITING
- Registry status
- 5,000 participants
- Enrollment target
- 20
- Study locations
Study 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
Primary Outcome
Charcot Marie Tooth Neuropathy Score (CMTNS) is a composite measure of disability based on a person's symptoms, signs, and electrophysiology. It is based on a 36 point scale, with 9 items each worth up to 4 points. A higher score signifies increased disability.
Conditions Studied
Study Locations (20)
Florida
- University of Miami - Miami
- Nemours Children's Health - Orlando
- Nemours Children's Hospital - Orlando
California
- Cedars-Sinai Medical Center - Los Angeles
- Stanford University - Palo Alto
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- University of Pennsylvania - Philadelphia
Colorado
- University of Colorado Hospital - Aurora
Connecticut
- University of Connecticut/Connecticut Children's Medical Center - Hartford
District of Columbia
- Children's National Hospital - Washington D.C.
Iowa
- University of Iowa - Iowa City
Maryland
- Johns Hopkins University - Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5,000 participants |
| Start Date | 2010-04-01 |
| Est. Completion | 2026-12 |
What NCT01193075 shows while recruiting
NCT01193075 is an observational study that tracks outcomes without assigning an intervention. Its 5,000 participants enrollment target places it among the larger protocols in the corpus, above the 1,651-participant average among 7 other Charcot Marie Tooth Disease trials with a reported enrollment target (203% higher).
The record links to 1 condition, with Charcot Marie Tooth Disease appearing as the primary indexed condition, and to 0 interventions.
NCT01193075 names 20 study sites across 16 states, led by Florida, California, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT01193075 about?
NCT01193075 is a clinical study titled "Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others". 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 ...
What is the current status of trial NCT01193075?
This trial is currently recruiting. The enrollment target is 5,000 participants. The study started on 2010-04-01. Estimated completion is 2026-12.
What conditions does trial NCT01193075 study?
This clinical trial studies the following conditions: Charcot Marie Tooth Disease.
Who is sponsoring clinical trial NCT01193075?
This trial is sponsored by Michael Shy, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01193075 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, District of Columbia, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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