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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01203085
Registration number
NCT01203085
Ethics application status
Date submitted
9/08/2010
Date registered
16/09/2010
Date last updated
10/05/2024
Titles & IDs
Public title
Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT
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Scientific title
Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth
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Secondary ID [1]
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1U54NS065712-01
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Secondary ID [2]
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INC-6603
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Universal Trial Number (UTN)
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Trial acronym
INC-6603
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Charcot Marie Tooth Disease
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Condition category
Condition code
Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Pediatric patients - All patients 21 years of age and under who are enrolled in the 6601 study and have undergone the pediatric scale tests.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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CMT Peds Scale Part 1: Symptoms
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Assessment method [1]
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The CMT Peds Scale Symptoms include foot and hand symptoms.
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Timepoint [1]
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1 year
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Primary outcome [2]
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CMT Peds Score Part 2: Foot and Ankle Involvement
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Assessment method [2]
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Foot and ankle involvement includes foot posture index, range of ankle dorsiflexion, foot drop present/absent, and whether or not difficulty heel/toe walking.
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Timepoint [2]
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1 year
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Primary outcome [3]
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CMT Peds Scale Part 3: Hand dexterity
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Assessment method [3]
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Hand dexterity involves hand dexterity testing and the nine-hole peg test.
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Timepoint [3]
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1 year
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Primary outcome [4]
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CMT Peds Scale Part 4: Hand strength
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Assessment method [4]
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Hand strength includes grip strength, thumb-index pinch, and three point pinch.
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Timepoint [4]
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1 year
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Primary outcome [5]
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CMT Peds Scale Part 5: Foot Strength
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Assessment method [5]
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Foot strength includes the strength of plantar- and dorsi-flexion, eversion, and inversion.
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Timepoint [5]
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1 year
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Primary outcome [6]
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CMT Peds Score Part 6: Sensation
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Assessment method [6]
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Sensation includes pinprick and vibration sensations.
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Timepoint [6]
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1 year
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Primary outcome [7]
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CMT Peds Scale Part 7: Balance
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Assessment method [7]
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Balance is assessed by the Bruininks-Oseretsky Test of Motor Proficiency, 2nd Edition (BOT-2).
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Timepoint [7]
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1 year
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Primary outcome [8]
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CMT Peds Scale Part 8: Motor Function
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Assessment method [8]
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Motor function assessment includes long jump, 10 meter run/walk, stair climb, stair descend, and 6 minute walk test.
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Timepoint [8]
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1 year
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Secondary outcome [1]
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Evaluate CMT Pediatric Scale (CMT Peds Scale) in CMT natural history study
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Assessment method [1]
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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.
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Timepoint [1]
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6 months to 1 year
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Eligibility
Key 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
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Minimum age
No limit
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Maximum age
21
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/04/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2024
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Actual
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Sample size
Target
500
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Children's Hospital of Westmead - Sydney
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Recruitment postcode(s) [1]
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2145 - Sydney
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Recruitment outside Australia
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Connecticut
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Country [3]
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United States of America
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State/province [3]
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Florida
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Country [4]
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United States of America
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State/province [4]
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Iowa
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Country [5]
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United States of America
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State/province [5]
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Michigan
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Country [6]
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United States of America
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State/province [6]
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New York
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Country [7]
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United States of America
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State/province [7]
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Pennsylvania
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Country [8]
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Italy
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State/province [8]
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Milan
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Country [9]
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United Kingdom
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State/province [9]
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England
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Country [10]
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United Kingdom
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State/province [10]
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UK
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Funding & Sponsors
Primary sponsor type
Other
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Name
University of Iowa
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Address
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Country
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Other collaborator category [1]
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Government body
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Name [1]
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National Institute of Neurological Disorders and Stroke (NINDS)
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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Muscular Dystrophy Association
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Address [2]
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Country [2]
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Other collaborator category [3]
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Other
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Name [3]
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University of Rochester
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Address [3]
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Other collaborator category [4]
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Other
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Name [4]
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Children's Hospital of Philadelphia
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Address [4]
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Other collaborator category [5]
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Other
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Name [5]
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University College London Hospitals
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Address [5]
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Other collaborator category [6]
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Other
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Name [6]
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Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
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Address [6]
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Other collaborator category [7]
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Other
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Name [7]
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Sydney Children's Hospitals Network
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Address [7]
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Country [7]
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Other collaborator category [8]
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Other
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Name [8]
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Nemours Children's Clinic
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Address [8]
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01203085
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Michael E Shy, MD
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Address
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University of Iowa
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Shawna M Feely, MS
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Address
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Country
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Phone
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319-384-6362
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01203085
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