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Clinical Trials in Italy / NCT01193075
Recruiting Observational

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

NCT01193075 · tracked via the Priya Life Science Italy tracker
Sponsor
Michael Shy
Phase
Observational
Started
2010-04-01
Last updated
2025-10-07

Condition(s) studied

Charcot Marie Tooth Disease

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

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
Accepted
Inclusion Criteria: All patients must be seen in-person at a participating center for the initial visit. Inclusion Criteria - patients with CMT (all subtypes) 1. Patient has documented, pathogenic or likely pathogenic CMT-causing variant(s) OR 2. Patient has a first- or second-degree family member (parent, child, sibling, half-sibling, aunt, uncle, grandparent, or grandchild) with a documented pathogenic or likely pathogenic CMT-causing variant AND a clear link between that family member and the affected patient AND a phenotype consistent with the diagnosis i. A clear link is necessary for a second-degree relative. For example, if a grandparent is affected and has a pathogenic or likely pathogenic variant, and the parent does not have any signs, symptoms, or electrophysiology consistent with the diagnosis, there is no clear link unless the parent has also been found to have the pathogenic or likely pathogenic variant such as in cases with reduced penetrance ii. In cases where clear links are not available, genetic testing is required for the patient or the family member who is not clearly affected. 3. Patients who have a variant of uncertain significance, as determined by the laboratory performing the testing may still be included if one of the following circumstances applies: i. Variant is categorized as pathogenic or likely pathogenic per the ACMG variant interpretation guidelines. \[80, 81\] ii. Variant has been found in multiple affected people in a family and has not been found in unaffected family members. (Note - both affected and unaffected family members must be tested in this situation to be included). iii. The principal investigator and the site investigator agree that the variant(s) is (are) most likely pathogenic. 4. Patients whose clinical presentation is suggestive of CMT, but CMT type and variant are unknown will be characterized by the following categories: 1. Nerve conduction velocities: demyelinating, axonal, intermediate 2. Inheritance: dominant, recessive, X-linked, or unknown 5. Patient or patient's legally authorized representative has understood and signed an IRB approved consent form for the study. Teenagers (age 13 - 17 years) and cognitively impaired adults who are able to read and write must sign an assent form (depending on local ethics committee requirements). Inclusion Criteria - Controls 1. Person does not have a peripheral neuropathy, as determined by the investigator. 2. Person has understood and signed an IRB approved consent form for the study. Teenagers (age 13-17 years) must sign an assent form (depending on local ethics committee requirements). EXCLUSION CRITERIA 1. Patient has a variant of uncertain significance that cannot be further classified following methods listed in the Inclusion Criteria. 2. Patient does not wish to be a part of the study or has not signed an informed consent form. 3. Patient is deemed inappropriate by the Site PI.

Primary outcome measure(s)

Trial sites (22)

FacilityCityRegionStatus
Cedars-Sinai Medical Center Los Angeles California Recruiting
Stanford University Palo Alto California Recruiting
University of Colorado Hospital Aurora Colorado Recruiting
University of Connecticut/Connecticut Children's Medical Center Hartford Connecticut Recruiting
Children's National Hospital Washington D.C. District of Columbia Recruiting
University of Miami Miami Florida Recruiting
Nemours Children's Health Orlando Florida Recruiting
Nemours Children's Hospital Orlando Florida Recruiting
University of Iowa Iowa City Iowa Recruiting
Johns Hopkins University Baltimore Maryland Recruiting
Harvard/Massachusetts General Hospital Boston Massachusetts Recruiting
University of Michigan Ann Arbor Michigan Not Yet Recruiting
University of Minnesota Minneapolis Minnesota Recruiting
University of Rochester Rochester New York Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
University of Pennsylvania Philadelphia Pennsylvania Recruiting
St. Jude Children's Research Hospital Memphis Tennessee Recruiting
Seattle Children's Hospital Seattle Washington Not Yet Recruiting
University of Westmead Sydney New South Wales Recruiting
The Hospital for Sick Children Toronto Ontario Recruiting
C. Besta Neurological Institute Milan Milan Recruiting
National Hospital of Neurology and Neurosurgery London England Recruiting

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT01193075 on ClinicalTrials.gov ↗ ← All trials in Italy