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

Charcot Marie Tooth Disease Clinical Trial

— INC-6603  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01203085
• Other study ID #: INC-6603
• Secondary ID: 1U54NS065712-01
• Status: Recruiting
• Start date: April 2010
• Est. completion date: December 2024

Study information

• Verified date: May 2024
• Source: University of Iowa
• Contact: Shawna M Feely, MS
• Phone: 319-384-6362
• Email: Shawna-Feely[@]uiowa.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

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.

Related Conditions & MeSH terms

• Charcot Marie Tooth Disease
• Charcot-Marie-Tooth Disease
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes
• Tooth Diseases

Locations

Country	Name	City	State
Australia	Children's Hospital of Westmead	Sydney	New South Wales
Italy	C. Fondazione IRCCS Istituto Neurologico Carlo Besta	Milan
United Kingdom	Dubowitz Neuromuscular Centre	London	UK
United Kingdom	National Hospital of Neurology and Neurosurgery	London	England
United States	University of Michigan	Ann Arbor	Michigan
United States	University of Connecticut/Connecticut Children's Medical Center	Hartford	Connecticut
United States	University of Iowa	Iowa City	Iowa
United States	Nemours Children's Clinic	Orlando	Florida
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of Rochester	Rochester	New York

Sponsors (9)

Lead Sponsor	Collaborator
University of Iowa	Children's Hospital of Philadelphia, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Muscular Dystrophy Association, National Institute of Neurological Disorders and Stroke (NINDS), Nemours Children's Clinic, Sydney Children's Hospitals Network, University College London Hospitals, University of Rochester

Countries where clinical trial is conducted

United States,  Australia,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	CMT Peds Scale Part 1: Symptoms	The CMT Peds Scale Symptoms include foot and hand symptoms.	1 year
Primary	CMT Peds Score Part 2: Foot and Ankle Involvement	Foot and ankle involvement includes foot posture index, range of ankle dorsiflexion, foot drop present/absent, and whether or not difficulty heel/toe walking.	1 year
Primary	CMT Peds Scale Part 3: Hand dexterity	Hand dexterity involves hand dexterity testing and the nine-hole peg test.	1 year
Primary	CMT Peds Scale Part 4: Hand strength	Hand strength includes grip strength, thumb-index pinch, and three point pinch.	1 year
Primary	CMT Peds Scale Part 5: Foot Strength	Foot strength includes the strength of plantar- and dorsi-flexion, eversion, and inversion.	1 year
Primary	CMT Peds Score Part 6: Sensation	Sensation includes pinprick and vibration sensations.	1 year
Primary	CMT Peds Scale Part 7: Balance	Balance is assessed by the Bruininks-Oseretsky Test of Motor Proficiency, 2nd Edition (BOT-2).	1 year
Primary	CMT Peds Scale Part 8: Motor Function	Motor function assessment includes long jump, 10 meter run/walk, stair climb, stair descend, and 6 minute walk test.	1 year
Secondary	Evaluate CMT Pediatric Scale (CMT Peds Scale) in CMT natural history study	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.	6 months to 1 year

External Links

•   Inherited Neuropathy Consortium Website