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Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT (INC-6603)

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ClinicalTrials.gov Identifier: NCT01203085
Recruitment Status : Recruiting
First Posted : September 16, 2010
Last Update Posted : September 13, 2017
Information provided by (Responsible Party):

August 9, 2010
September 16, 2010
September 13, 2017
April 2010
December 2019   (Final data collection date for primary outcome measure)
  • CMT Peds Scale Part 1: Symptoms [ Time Frame: 1 year ]
    The CMT Peds Scale Symptoms include foot and hand symptoms.
  • CMT Peds Score Part 2: Foot and Ankle Involvement [ Time Frame: 1 year ]
    Foot and ankle involvement includes foot posture index, range of ankle dorsiflexion, foot drop present/absent, and whether or not difficulty heel/toe walking.
  • CMT Peds Scale Part 3: Hand dexterity [ Time Frame: 1 year ]
    Hand dexterity involves hand dexterity testing and the nine-hole peg test.
  • CMT Peds Scale Part 4: Hand strength [ Time Frame: 1 year ]
    Hand strength includes grip strength, thumb-index pinch, and three point pinch.
  • CMT Peds Scale Part 5: Foot Strength [ Time Frame: 1 year ]
    Foot strength includes the strength of plantar- and dorsi-flexion, eversion, and inversion.
  • CMT Peds Score Part 6: Sensation [ Time Frame: 1 year ]
    Sensation includes pinprick and vibration sensations.
  • CMT Peds Scale Part 7: Balance [ Time Frame: 1 year ]
    Balance is assessed by the Bruininks-Oseretsky Test of Motor Proficiency, 2nd Edition (BOT-2).
  • CMT Peds Scale Part 8: Motor Function [ Time Frame: 1 year ]
    Motor function assessment includes long jump, 10 meter run/walk, stair climb, stair descend, and 6 minute walk test.
Same as current
Complete list of historical versions of study NCT01203085 on ClinicalTrials.gov Archive Site
Evaluate CMT Pediatric Scale (CMT Peds Scale) in CMT natural history study [ Time Frame: 6 months to 1 year ]
The sections of the CMT Peds Scale which are found to be clinically/functionally useful after one year of analysis will be carried forward for all pediatric patients every 6 months to one year.
Same as current
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Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT
Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth
The primary goal of this project is to develop and test a Charcot Marie Tooth disease (CMT) Pediatric Scale for use in evaluation in natural history CMT study.
This project is to develop a new CMT Pediatric Scale (CMTPeds) for Children with CMT. Although there is a validated score (the CMTNS) which measures disease severity for CMT, it is not always applicable to children due to their limited ability to relay information about their symptoms. The CMTPeds scale is being developed and validated in order to measure disease severity in children and have outcome measures available for future clinical trials. Children (defined as 21 and under) being evaluated will be asked to perform functional tasks such as using stairs, walking in a hallway, and performing hand function tests. This information will be used to validate the CMTPeds score. It is important to have validated instruments to measure disease severity in childhood so these can be used with clinical treatment trials are available.
Observational Model: Case-Only
Time Perspective: Prospective
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Non-Probability Sample
Patients who are 21 years of age and under who are also enrolled in the 6601 study and have performed all tasks to complete the CMT Peds Scale will be recruited for participation. Participation entails allow the information collected in the 6601 study be used for validation in the current study.
Charcot Marie Tooth Disease
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Pediatric patients
All patients 21 years of age and under who are enrolled in the 6601 study and have undergone the pediatric scale tests.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
December 2019
December 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

All patients MUST be seen in person at one of the participating centers for enrollment in this study.

  • Children (< 21 years of age)
  • Known or probable inherited neuropathies classified as CMT1, CMT2, or CMT4

Exclusion Criteria:

  • Known diagnoses of acquired neuropathy including toxic (e. g. medication related neuropathies); metabolic (e.g. diabetic), immune mediated or inflammatory [acute inflammatory demyelinating polyradiculoneuropathy (AIDP) or chronic inflammatory demyelinating polyneuropathy (CIDP)] polyneuropathies; neuropathy related to leukodystrophy, congenital muscular dystrophy; and patients with severe general medical conditions.
  • Entirely normal conduction velocities of upper and lower limbs as this suggests that the subject may not have a neuropathy.
Sexes Eligible for Study: All
up to 21 Years   (Child, Adult)
Contact: Shawna M Feely, MS 319-384-6362 Shawna-Feely@uiowa.edu
Contact: Tiffany Grider, MS 319-384-6362 Tiffany-Grider@uiowa.edu
Australia,   Italy,   United Kingdom,   United States
1U54NS065712-01 ( U.S. NIH Grant/Contract )
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Not Provided
Michael Shy, University of Iowa
University of Iowa
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Muscular Dystrophy Association
  • University of Rochester
  • Children's Hospital of Philadelphia
  • University College London Hospitals
  • Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
  • Sydney Children's Hospitals Network
  • Nemours Children's Clinic
Principal Investigator: Michael E Shy, MD University of Iowa
University of Iowa
September 2017