Clinical Trial: Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Observational

Official Title: Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: University of Iowa

Current Primary Outcome:

  • 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

    Original Primary Outcome: Same as current

    Current Secondary Outcome: 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.


    Original Secondary Outcome: Same as current

    Information By: University of Iowa

    Dates:
    Date Received: August 9, 2010
    Date Started: April 2010
    Date Completion:
    Last Updated: May 14, 2015
    Last Verified: May 2015