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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01203085




Registration number
NCT01203085
Ethics application status
Date submitted
9/08/2010
Date registered
16/09/2010

Titles & IDs
Public title
Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT
Scientific title
Development and Validation of CMT Pediatric Scale for Children With Charcot Marie Tooth
Secondary ID [1] 0 0
1U54NS065712-01
Secondary ID [2] 0 0
INC-6603
Universal Trial Number (UTN)
Trial acronym
INC-6603
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Charcot Marie Tooth Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
CMT Peds Scale Part 1: Symptoms
Timepoint [1] 0 0
1 year
Primary outcome [2] 0 0
CMT Peds Score Part 2: Foot and Ankle Involvement
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
CMT Peds Scale Part 3: Hand dexterity
Timepoint [3] 0 0
1 year
Primary outcome [4] 0 0
CMT Peds Scale Part 4: Hand strength
Timepoint [4] 0 0
1 year
Primary outcome [5] 0 0
CMT Peds Scale Part 5: Foot Strength
Timepoint [5] 0 0
1 year
Primary outcome [6] 0 0
CMT Peds Score Part 6: Sensation
Timepoint [6] 0 0
1 year
Primary outcome [7] 0 0
CMT Peds Scale Part 7: Balance
Timepoint [7] 0 0
1 year
Primary outcome [8] 0 0
CMT Peds Scale Part 8: Motor Function
Timepoint [8] 0 0
1 year
Secondary outcome [1] 0 0
Evaluate CMT Pediatric Scale (CMT Peds Scale) in CMT natural history study
Timepoint [1] 0 0
6 months to 1 year

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
Minimum age
No limit
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2024
Actual
Sample size
Target
500
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Children's Hospital of Westmead - Sydney
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
Italy
State/province [8] 0 0
Milan
Country [9] 0 0
United Kingdom
State/province [9] 0 0
England
Country [10] 0 0
United Kingdom
State/province [10] 0 0
UK

Funding & Sponsors
Primary sponsor type
Other
Name
University of Iowa
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Institute of Neurological Disorders and Stroke (NINDS)
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Muscular Dystrophy Association
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
University of Rochester
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Children's Hospital of Philadelphia
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
University College London Hospitals
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Address [6] 0 0
Country [6] 0 0
Other collaborator category [7] 0 0
Other
Name [7] 0 0
Sydney Children's Hospitals Network
Address [7] 0 0
Country [7] 0 0
Other collaborator category [8] 0 0
Other
Name [8] 0 0
Nemours Children's Clinic
Address [8] 0 0
Country [8] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Michael E Shy, MD
Address 0 0
University of Iowa
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Shawna M Feely, MS
Address 0 0
Country 0 0
Phone 0 0
319-384-6362
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified RDCRN data is submitted to an ORDR-designated repository. For the current grant cycle, that repository has been dbGaP.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR), Analytic code
When will data be available (start and end dates)?
(For Observational/Longitudinal/Natural History/Epidemiology studies): For the current grant cycle, available data will be released to the repository and will become available to the scientific community one year after publication of planned analyses, or after a period of 5 years from the date when the data were collected, whichever comes first.
Available to whom?
For the current grant cycle, once de-identified data is posted on dbGaP, a summary of the study is posted and individual participant data is accessed via a request through dbGaP.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs001553.v1.p1


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT01203085