Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT (INC-6603)
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.
Charcot Marie Tooth Disease
|Study Design:||Observational Model: Case-Only
Time Perspective: Prospective
|Official Title:||Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth|
- CMT Peds Scale Part 1: Symptoms [ Time Frame: 1 year ] [ Designated as safety issue: No ]The CMT Peds Scale Symptoms include foot and hand symptoms.
- CMT Peds Score Part 2: Foot and Ankle Involvement [ Time Frame: 1 year ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]Hand dexterity involves hand dexterity testing and the nine-hole peg test.
- CMT Peds Scale Part 4: Hand strength [ Time Frame: 1 year ] [ Designated as safety issue: No ]Hand strength includes grip strength, thumb-index pinch, and three point pinch.
- CMT Peds Scale Part 5: Foot Strength [ Time Frame: 1 year ] [ Designated as safety issue: No ]Foot strength includes the strength of plantar- and dorsi-flexion, eversion, and inversion.
- CMT Peds Score Part 6: Sensation [ Time Frame: 1 year ] [ Designated as safety issue: No ]Sensation includes pinprick and vibration sensations.
- CMT Peds Scale Part 7: Balance [ Time Frame: 1 year ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]Motor function assessment includes long jump, 10 meter run/walk, stair climb, stair descend, and 6 minute walk test.
- Evaluate CMT Pediatric Scale (CMT Peds Scale) in CMT natural history study [ Time Frame: 6 months to 1 year ] [ Designated as safety issue: No ]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.
|Study Start Date:||April 2010|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
All patients 21 years of age and under who are enrolled in the 6601 study and have undergone the pediatric scale tests.
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.
|Contact: Shawna M Feely, MS||319-384-6362||Shawna-Feely@uiowa.edu|
|Contact: Tiffany Grider, MS||319-384-6362||Tiffany-Grider@uiowa.edu|
|United States, Florida|
|Nemours Children's Clinic||Recruiting|
|Orlando, Florida, United States|
|Contact: Shannon Henry, MA 407-650-7604 Shannon.Henry@nemours.org|
|Principal Investigator: Richard Finkel, MD|
|United States, Iowa|
|University of Iowa||Recruiting|
|Iowa City, Iowa, United States, 52242|
|Contact: Shawna M Feely, MS 319-384-6362 Shawna-Feely@uiowa.edu|
|Principal Investigator: Michael E Shy, MD|
|United States, New York|
|University of Rochester||Recruiting|
|Rochester, New York, United States, 14642|
|Contact: Janet Sowden 585-275-1267 email@example.com|
|Principal Investigator: David Herrmann, MD|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia||Recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Sabrina Yum, MD 215-590-1719 YUMS@email.chop.edu|
|Principal Investigator: Sabrina Yum, MD|
|University of Pennsylvania||Recruiting|
|Philadelphia, Pennsylvania, United States|
|Contact: Meredith Gage 215-349-5313 firstname.lastname@example.org|
|Principal Investigator: Steven Scherer, MD, PhD|
|Australia, New South Wales|
|Children's Hospital of Westmead||Recruiting|
|Sydney, New South Wales, Australia, 2145|
|Contact: Natalie Gabrael +61 2 9845 1904 email@example.com|
|Principal Investigator: Joshua Burns, PhD|
|C. Fondazione IRCCS Istituto Neurologico Carlo Besta||Recruiting|
|Contact: Davide Pareyson, MD (+39)02-23943001 firstname.lastname@example.org|
|Principal Investigator: Davide Pareyson, MD|
|National Hospital of Neurology and Neurosurgery||Recruiting|
|London, England, United Kingdom, WC1N 3BG|
|Contact: Jacky Molyneaux +44 207 380 6852 email@example.com|
|Principal Investigator: Mary Reilly, MD|
|Principal Investigator:||Michael E Shy, MD||University of Iowa|