Trial record 4 of 119 for:
Open Studies | "Tooth Diseases"
Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT (INC-6603)
This study is currently recruiting participants.
Verified October 2011 by Wayne State University
Sponsor:
Wayne State University
Collaborators:
Muscular Dystrophy Association
University of Rochester
Children's Hospital of Westmead
Children's Hospital of Philadelphia
National Hospital of Neurology and Neurosurgery
C. Besta Neurological Institute
University of Washington
Johns Hopkins University
Information provided by (Responsible Party):
Michael E. Shy, MD, Wayne State University
ClinicalTrials.gov Identifier:
NCT01203085
First received: August 9, 2010
Last updated: October 17, 2011
Last verified: October 2011
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Purpose
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.
| Condition |
|---|
|
Charcot Marie Tooth Disease |
| Study Type: | Observational |
| Study Design: | Observational Model: Case-Only Time Perspective: Prospective |
| Official Title: | Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth |
Resource links provided by NLM:
Genetics Home Reference related topics:
Charcot-Marie-Tooth disease
hereditary neuropathy with liability to pressure palsies
U.S. FDA Resources
Further study details as provided by Wayne State University:
Primary Outcome Measures:
- 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.
Secondary Outcome Measures:
- 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.
| Estimated Enrollment: | 500 |
| Study Start Date: | April 2010 |
| Groups/Cohorts |
|---|
|
Pediatric patients
All patients 21 years of age and under who are enrolled in the 6601 study and have undergone the pediatric scale tests.
|
Eligibility| Ages Eligible for Study: | up to 21 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Patients who are 21 years of age and under who are also enrolled in the 6601 study and have performed all tasks to complete the CMT Peds Scale will be recruited for participation. Participation entails allow the information collected in the 6601 study be used for validation in the current study.
Criteria
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01203085
Contacts
| Contact: Carly E Siskind, MS | 313-577-8317 | csiskind@med.wayne.edu |
| Contact: Lisa Rowe, BS | 313-577-1689 | lrowe@med.wayne.edu |
Locations
| United States, Michigan | |
| Wayne State University | Recruiting |
| Detroit, Michigan, United States, 48201 | |
| Contact: Lisa Rowe, BS 313-577-1689 lrowe@med.wayne.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 janet_sowden@urmc.rochester.edu | |
| Principal Investigator: David Herrmann, MD | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Donnette Paris 267-426-7167 Paris@email.chop.edu | |
| Principal Investigator: Richard Finkel, MD | |
| Australia, New South Wales | |
| Children's Hospital of Westmead | Recruiting |
| Sydney, New South Wales, Australia, 2145 | |
| Contact: Natalie Gabrael +61 2 9845 1904 natalig1@chw.edu.au | |
| Principal Investigator: Joshua Burns, PhD | |
| United Kingdom | |
| National Hospital of Neurology and Neurosurgery | Recruiting |
| London, England, United Kingdom, WC1N 3BG | |
| Contact: Jacky Molyneaux +44 207 380 6852 j.molyneaux@ion.ucl.ac.uk | |
| Principal Investigator: Mary Reilly, MD | |
Sponsors and Collaborators
Wayne State University
Muscular Dystrophy Association
University of Rochester
Children's Hospital of Westmead
Children's Hospital of Philadelphia
National Hospital of Neurology and Neurosurgery
C. Besta Neurological Institute
University of Washington
Johns Hopkins University
Investigators
| Principal Investigator: | Michael E Shy, MD | Wayne State University |
More Information
Additional Information:
No publications provided
| Responsible Party: | Michael E. Shy, MD, Professor, Wayne State University |
| ClinicalTrials.gov Identifier: | NCT01203085 History of Changes |
| Other Study ID Numbers: | INC-6603, 1U54NS065712-01 |
| Study First Received: | August 9, 2010 |
| Last Updated: | October 17, 2011 |
| Health Authority: | Australia: Human Research Ethics Committee United Kingdom: Research Ethics Committee United States: Institutional Review Board Italy: Research Ethics Committee |
Additional relevant MeSH terms:
|
Charcot-Marie-Tooth Disease Tooth Diseases Nerve Compression Syndromes Hereditary Sensory and Motor Neuropathy Nervous System Malformations Nervous System Diseases Heredodegenerative Disorders, Nervous System |
Neurodegenerative Diseases Polyneuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Congenital Abnormalities Genetic Diseases, Inborn Stomatognathic Diseases |
ClinicalTrials.gov processed this record on June 17, 2013