- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02596191
Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers (CMT-TOOLS)
July 19, 2023 updated by: Assistance Publique Hopitaux De Marseille
This is a 2-year follow-up study of a cohort of 60 CMT1A patients.
The objective is to identify markers allowing to better understand the phenotypic variability observed on patients with CMT1A, to identify predictive markers of the disease's progression and to provide validated measurement tools that can be used as outcome measures in future clinical trials.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
80
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Shahram Attarian, MD
- Email: shahram.attarian@ap-hm.fr
Study Locations
-
-
-
Marseille, France, 13010
- Recruiting
- Assistance Publique Hopitaux de Marseille
-
Contact:
- Shahram Attarian, MD
- Email: shahram.attarian@ap-hm.fr
-
Montpellier, France, 34285
- Recruiting
- CHU Gui de Chauliac, CHU MONTPELLIER
-
Contact:
- William Camu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 70 years (Adult, Older Adult)
Accepts Healthy Volunteers
Yes
Description
Inclusion Criteria:
- Patients with CMT 1A disease
- Diagnosis of CMT 1A confirmed by genotyping (duplication of the 17p11.2 region)
Exclusion Criteria:
- Patients suffering from co-morbidity at the origin of peripheral neuropathy (diabetes, hypothyroidism, renal insufficiency, drugs...) or muscle, articular, rheumatological disease
- With HIV or cancer
- With a significant progressive disease in the previous month
- With a contra-indication for MRI
- With a dislocation, fracture, or recent surgery (less than 6 months before inclusion)
- with alcohol or psychoactive substances abuse
- Treated by an anti-inflammatory drug over the past four weeks
- Pregnant or breastfeeding women
- Homeless patients
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: patients with mild CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score between 1 and 10
|
|
Experimental: patients with moderate CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score between 11 and 20
|
|
Experimental: patients with severe CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score ≥21
|
|
Other: control group
Healthy volunteers
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change of functional scores
Time Frame: 3 months, 12 months and 24 months
|
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNDS)
|
3 months, 12 months and 24 months
|
Change of functional scores
Time Frame: 3 months, 12 months and 24 months
|
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNS)
|
3 months, 12 months and 24 months
|
Change of functional scores
Time Frame: 3 months, 12 months and 24 months
|
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNS2)
|
3 months, 12 months and 24 months
|
Change of functional scores at
Time Frame: 3 months, 12 months and 24 months
|
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (ONLS)
|
3 months, 12 months and 24 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Walkin test
Time Frame: 12 months and 24 months
|
Use of connected soles for walking test (Digitsole)
|
12 months and 24 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Study Director: Urielle DESALBRES, Assistance Publique Hopitaux de Marseille
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 6, 2016
Primary Completion (Estimated)
June 6, 2026
Study Completion (Estimated)
December 6, 2027
Study Registration Dates
First Submitted
October 30, 2015
First Submitted That Met QC Criteria
November 3, 2015
First Posted (Estimated)
November 4, 2015
Study Record Updates
Last Update Posted (Actual)
July 20, 2023
Last Update Submitted That Met QC Criteria
July 19, 2023
Last Verified
July 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Stomatognathic Diseases
- Neurodegenerative Diseases
- Peripheral Nervous System Diseases
- Heredodegenerative Disorders, Nervous System
- Nervous System Malformations
- Polyneuropathies
- Disease
- Tooth Diseases
- Nerve Compression Syndromes
- Charcot-Marie-Tooth Disease
- Hereditary Sensory and Motor Neuropathy
Other Study ID Numbers
- 2014-10
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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