Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)
NCT ID: NCT02979145
Last Updated: 2016-12-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
200 participants
OBSERVATIONAL
2016-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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Patients with CMT
Two groups of patients will be included: Group 1 (Definitive): Children with known CMT where genetic testing confirms the diagnosis, or children with a clinical diagnosis including electrophysiology confirming the presence of CMT and a corresponding family history where a first or second degree relative has a genetic diagnosis; or Group 2 (At risk): A clinical diagnosis of CMT awaiting genetic testing or confirmatory electrophysiology and evidence of a genetic diagnosis in a first or second degree relative; or individuals identified as being at risk of a CMT diagnosis (prodromal patients), without the onset of signs or symptoms.
No intervention involved
Controls
Healthy controls will be included from unaffected family members or friends accompanying patients at INC sites. Healthy controls are defined as boys and girls aged 0-≤4 years without a diagnosis of CMT or any of the other study exclusion criteria.
No intervention involved
Interventions
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No intervention involved
Eligibility Criteria
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Inclusion Criteria
* Parent(s) or guardians have agreed for the child to take part in the study and have signed an informed consent form.
* Patient has known or probable inherited neuropathy
* Patient participates in the INC Natural History Study (INC 6601)
* Participant is ≤4 years of age
* Parent(s) or guardians have agreed for the child to take part in the study and have signed an informed consent form.
* Participant does NOT have an inherited neuropathy as determined by the investigator
* Participant is an unaffected friend or family member of a patient with CMT (patient does not have to be included in the study)
Exclusion Criteria
* Patient has a severe general medical condition, as determined by the site Principal Investigator.
4 Years
ALL
Yes
Sponsors
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University of Iowa
OTHER
Children's Hospital of Philadelphia
OTHER
University of Pennsylvania
OTHER
University of Rochester
OTHER
National Hospital of Neurology and Neurosurgery
UNKNOWN
Dubowitz Neuromuscular Centre
OTHER
University of Miami
OTHER
Carlo Besta Neurological Institute
OTHER
Johns Hopkins University
OTHER
Vanderbilt University
OTHER
University of Washington
OTHER
Nemours Children's Hospital
OTHER
National Institutes of Health (NIH)
NIH
Stanford University
OTHER
Cedars-Sinai Medical Center
OTHER
Harvard/Massachusetts General Hospital
UNKNOWN
University of Michigan
OTHER
University of Minnesota
OTHER
University of Utah
OTHER
University of Connecticut
OTHER
Universiteit Antwerpen
OTHER
Sydney Children's Hospitals Network
OTHER
Responsible Party
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Joshua Burns
Professor of Allied Health (Paediatrics)
Locations
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University of Iowa Health Care
Iowa City, Iowa, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
C. Besta Neurological Institute
Milan, , Italy
Countries
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Facility Contacts
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Shawna Feely, MS, CGC
Role: primary
Timothy Estilow, OT
Role: primary
Melissa Mandarakas, MPhty
Role: primary
Isabella Moroni
Role: primary
Related Links
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6611: Charcot-Marie-Tooth disease Infant Scale Recruitment Website
Other Identifiers
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6611
Identifier Type: -
Identifier Source: org_study_id