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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Development of the Charcot-Marie-Tooth Disease Infant Scale (CMTInfS) for Infants With CMT

Brief Summary: The purpose of this study is to develop and validate a clinical outcome measure to evaluate disability and disease progression of children 3 years of age and younger (infants and toddlers) with various types of Charcot-Marie-Tooth disease (CMT).

Detailed Summary: Most forms of CMT begin in childhood and progress throughout a person's lifetime. Current research suggests that treatment to slow disease progression may be most effective if introduced early in a patient's life before muscle weakness and sensation loss develop, as it may be easier to slow disease progression than to reverse disability that is already in place. Clinical outcome measures have been developed for adults (CMT Neuropathy Score) and for children 3 years of age and older (CMT Pediatric Scale). However, no CMT-specific clinical outcome measure currently exists to measure disease severity or progression in children from birth to 3 years of age. It is the goal of this study to develop and validate the CMT Infant Scale (CMTInfS) to meet this need.
Sponsor: Sydney Children's Network

Current Primary Outcome:

  • CMT Infant Scale Part 1 [ Time Frame: 1 year ]
    The CMT Infant Scale physical assessment
  • The CMT Infant Scale Part 2 [ Time Frame: 1 year ]
    The CMT Infant Scale lower limb and gross motor items
  • The CMT Infant Scale Part 3 [ Time Frame: 1 year ]
    The CMT Infant Scale upper limb and fine motor items


Original Primary Outcome: Same as current

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

The sections of the CMT Infant Scale which are found to be clinically/functionally useful after one year of analysis will be carried forward for all infant patients every 6 months to one year.


Original Secondary Outcome: Same as current

Information By: Sydney Children's Network

Dates:
Date Received: November 29, 2016
Date Started: October 2016
Date Completion:
Last Updated: November 29, 2016
Last Verified: November 2016