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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02979145




Registration number
NCT02979145
Ethics application status
Date submitted
29/11/2016
Date registered
1/12/2016
Date last updated
1/12/2016

Titles & IDs
Public title
Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)
Scientific title
Development of the Charcot-Marie-Tooth Disease Infant Scale (CMTInfS) for Infants With CMT
Secondary ID [1] 0 0
6611
Universal Trial Number (UTN)
Trial acronym
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
Patients with CMT - Two groups of patients will be included: Group 1 (Definitive): Children with known CMT where genetic testing confirms the diagnosis, or children with a clinical diagnosis including electrophysiology confirming the presence of CMT and a corresponding family history where a first or second degree relative has a genetic diagnosis; or Group 2 (At risk): A clinical diagnosis of CMT awaiting genetic testing or confirmatory electrophysiology and evidence of a genetic diagnosis in a first or second degree relative; or individuals identified as being at risk of a CMT diagnosis (prodromal patients), without the onset of signs or symptoms.

Controls - Healthy controls will be included from unaffected family members or friends accompanying patients at INC sites. Healthy controls are defined as boys and girls aged 0-=4 years without a diagnosis of CMT or any of the other study exclusion criteria.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
CMT Infant Scale Part 1
Assessment method [1] 0 0
The CMT Infant Scale physical assessment
Timepoint [1] 0 0
1 year
Primary outcome [2] 0 0
The CMT Infant Scale Part 2
Assessment method [2] 0 0
The CMT Infant Scale lower limb and gross motor items
Timepoint [2] 0 0
1 year
Primary outcome [3] 0 0
The CMT Infant Scale Part 3
Assessment method [3] 0 0
The CMT Infant Scale upper limb and fine motor items
Timepoint [3] 0 0
1 year
Secondary outcome [1] 0 0
Evaluate CMT Infant Scale (CMTInfS) in CMT natural history study
Assessment method [1] 0 0
The sections of the CMT Infant Scale which are found to be clinically/functionally useful after one year of analysis will be carried forward for all infant patients every 6 months to one year.
Timepoint [1] 0 0
6 months - 1 year

Eligibility
Key inclusion criteria
Inclusion Criteria (patients with CMT):

* Patient is =4 years of age
* Parent(s) or guardians have agreed for the child to take part in the study and have signed an informed consent form.
* Patient has known or probable inherited neuropathy
* Patient participates in the INC Natural History Study (INC 6601)

Inclusion Criteria (controls):

* Participant is =4 years of age
* Parent(s) or guardians have agreed for the child to take part in the study and have signed an informed consent form.
* Participant does NOT have an inherited neuropathy as determined by the investigator
* Participant is an unaffected friend or family member of a patient with CMT (patient does not have to be included in the study)
Minimum age
No limit
Maximum age
4 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Exclusion Criteria (patients with CMT):

* Patient has a known condition of acquired neuropathy including toxic (e.g. medication related), metabolic (e.g. diabetic), immune mediated or inflammatory (AIDP or CIDP) neuropathies, a neuropathy related to leukodystrophy, or a congenital muscular dystrophy.
* Patient has a severe general medical condition, as determined by the site Principal Investigator.
* Patient has known normal nerve conductions of upper and lower limbs. This will be considered as exclusion criteria because it documents that the child does not have a large-fibre neuropathy. However, patients will not be required to have nerve conduction studies (NCS) or electromyography (EMG).

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/10/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Children's Hospital at 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
Iowa
Country [2] 0 0
United States of America
State/province [2] 0 0
Pennsylvania
Country [3] 0 0
Italy
State/province [3] 0 0
Milan

Funding & Sponsors
Primary sponsor type
Other
Name
Sydney Children's Hospitals Network
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Iowa
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Children's Hospital of Philadelphia
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
University of Pennsylvania
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
University of Rochester
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
National Hospital of Neurology and Neurosurgery
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
Dubowitz Neuromuscular Centre
Country [6] 0 0
Other collaborator category [7] 0 0
Other
Name [7] 0 0
University of Miami
Country [7] 0 0
Other collaborator category [8] 0 0
Other
Name [8] 0 0
Carlo Besta Neurological Institute
Country [8] 0 0
Other collaborator category [9] 0 0
Other
Name [9] 0 0
Johns Hopkins University
Country [9] 0 0
Other collaborator category [10] 0 0
Other
Name [10] 0 0
Vanderbilt University
Country [10] 0 0
Other collaborator category [11] 0 0
Other
Name [11] 0 0
University of Washington
Country [11] 0 0
Other collaborator category [12] 0 0
Other
Name [12] 0 0
Nemours Children's Hospital
Country [12] 0 0
Other collaborator category [13] 0 0
Government body
Name [13] 0 0
National Institutes of Health (NIH)
Country [13] 0 0
Other collaborator category [14] 0 0
Other
Name [14] 0 0
Stanford University
Country [14] 0 0
Other collaborator category [15] 0 0
Other
Name [15] 0 0
Cedars-Sinai Medical Center
Country [15] 0 0
Other collaborator category [16] 0 0
Other
Name [16] 0 0
Harvard/Massachusetts General Hospital
Country [16] 0 0
Other collaborator category [17] 0 0
Other
Name [17] 0 0
University of Michigan
Country [17] 0 0
Other collaborator category [18] 0 0
Other
Name [18] 0 0
University of Minnesota
Country [18] 0 0
Other collaborator category [19] 0 0
Other
Name [19] 0 0
University of Utah
Country [19] 0 0
Other collaborator category [20] 0 0
Other
Name [20] 0 0
University of Connecticut
Country [20] 0 0
Other collaborator category [21] 0 0
Other
Name [21] 0 0
Universiteit Antwerpen
Country [21] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


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/NCT02979145