Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)
- Conditions
- Charcot-Marie-Tooth Disease
- Registration Number
- NCT02979145
- Lead Sponsor
- Sydney Children's Hospitals Network
- 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 Description
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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 200
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method CMT Infant Scale Part 1 1 year The CMT Infant Scale physical assessment
The CMT Infant Scale Part 2 1 year The CMT Infant Scale lower limb and gross motor items
The CMT Infant Scale Part 3 1 year The CMT Infant Scale upper limb and fine motor items
- Secondary Outcome Measures
Name Time Method Evaluate CMT Infant Scale (CMTInfS) in CMT natural history study 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.
Trial Locations
- Locations (4)
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
University of Iowa Health Care🇺🇸Iowa City, Iowa, United States