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Trial record 5 of 301 for:    Recruiting, Not yet recruiting, Available Studies | "Tooth Diseases"

Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers (CMT-TOOLS)

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ClinicalTrials.gov Identifier: NCT02596191
Recruitment Status : Not yet recruiting
First Posted : November 4, 2015
Last Update Posted : November 4, 2015
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Charcot-Marie-Tooth Disease Type 1A Other: Clinical evaluation Other: electrophysiological record Other: Muscle MRI Other: blood samples analysis Not Applicable

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Official Title: Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers
Study Start Date : November 2015
Estimated Primary Completion Date : November 2018
Estimated Study Completion Date : November 2019


Arm Intervention/treatment
Experimental: patients with mild CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score between 1 and 10
Other: Clinical evaluation
Other: electrophysiological record
Other: Muscle MRI
Other: blood samples analysis
Experimental: patients with moderate CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score between 11 and 20
Other: Clinical evaluation
Other: electrophysiological record
Other: Muscle MRI
Other: blood samples analysis
Experimental: patients with severe CMT 1A disease
Charcot-Marie-Tooth Neuropathy Score ≥21
Other: Clinical evaluation
Other: electrophysiological record
Other: Muscle MRI
Other: blood samples analysis
control group
Healthy volunteers
Other: Muscle MRI
Other: blood samples analysis



Primary Outcome Measures :
  1. Change of functional scores at 3 different times [ 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, CMTNDS, CMTNS2, ONLS)



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02596191


Contacts
Contact: Shahram ATTARIAN, MD shahram.attarian@ap-hm.fr

Locations
France
Assistance Publique Hôpitaux de Marseille Not yet recruiting
Marseille, France, 13010
Contact: Shahram Attarian, MD       shahram.attarian@ap-hm.fr   
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
Study Director: Urielle Desalbres Assistance Publique Hôpitaux de Marseille

Responsible Party: Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier: NCT02596191     History of Changes
Other Study ID Numbers: 2014-10
First Posted: November 4, 2015    Key Record Dates
Last Update Posted: November 4, 2015
Last Verified: November 2015

Additional relevant MeSH terms:
Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Stomatognathic Diseases
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn