Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2016
• Main ID: NCT02979145
• Date of registration: 29/11/2016
• Prospective Registration: No
• Primary sponsor: Sydney Children's Network
• Public title: Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)
• Scientific title: Development of the Charcot-Marie-Tooth Disease Infant Scale (CMTInfS) for Infants With CMT
• Date of first enrolment: October 2016
• Target sample size: 200
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02979145
• Study type: Observational
• Study design: Observational Model: Case-Only, Time Perspective: Prospective
• Phase: N/A

Countries of recruitment

Australia, Italy, United States

Key inclusion & exclusion criteria

Inclusion Criteria (patients with CMT):

- Patient 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.

- Patient has known or probable inherited neuropathy

- Patient participates in the INC Natural History Study (INC 6601)

Inclusion Criteria (controls):

- 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 (patients with CMT):

- Patient has a known condition of acquired neuropathy including toxic (e.g. medication
related), metabolic (e.g. diabetic), immune mediated or inflammatory (AIDP or CIDP)
neuropathies, a neuropathy related to leukodystrophy, or a congenital muscular
dystrophy.

- Patient has a severe general medical condition, as determined by the site Principal
Investigator.

- Patient has known normal nerve conductions of upper and lower limbs. This will be
considered as exclusion criteria because it documents that the child does not have a
large-fibre neuropathy. However, patients will not be required to have nerve
conduction studies (NCS) or electromyography (EMG).



Age minimum: N/A
Age maximum: 4 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Charcot-Marie-Tooth Disease

Intervention(s)

Other: No intervention involved

Primary Outcome(s)

CMT Infant Scale Part 1 [Time Frame: 1 year]

The CMT Infant Scale Part 3 [Time Frame: 1 year]

The CMT Infant Scale Part 2 [Time Frame: 1 year]

Secondary Outcome(s)

Evaluate CMT Infant Scale (CMTInfS) in CMT natural history study [Time Frame: 6 months - 1 year]

Secondary ID(s)

6611

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Hospital of Neurology and Neurosurgery

Cedars-Sinai Medical Center

Nemours Children's Hospital

Stanford University

Children's Hospital of Philadelphia

National Institutes of Health - Intramural Branch (Bethesda)

University of Iowa Health Care

University of Pennsylvania

University of Washington

Johns Hopkins University

University of Rochester

Dubowitz Neuromuscular Centre

Universiteit Antwerpen

University of Connecticut

University of Miami

University of Michigan

University of Utah

Vanderbilt University

Harvard/Massachusetts General Hospital

University of Minnesota - Clinical and Translational Science Institute

Besta Neurological Institute