Clinical Trial Evaluating the Impact of an Intensive Rehabilitation Program Combined With Tendon Vibratory Stimulation on Functional Balance in Individuals With Charcot-Marie-Tooth Disease Type 1A (EQUIVIB-CMT)

Charcot-Marie-Tooth (CMT) disease is caused by a genetic abnormality involving the PMP22 gene, resulting in demyelination of the peripheral nerves. Demyelination leads to sensorimotor impairment and causes progressive muscle weakness and tendon contractures, initially affecting the lower limbs. Consequently, individuals with CMT experience balance impairments and gait disturbances, including ankle instability, foot drop, and postural instability, which lead to frequent falls and reduced quality of life. Currently, there is no disease-modifying treatment for CMT.

Several rehabilitation approaches have been proposed, including endurance training and muscle strengthening programs, to improve independence in activities of daily living. However, rehabilitation practices for individuals with CMT remain poorly standardized, and there is still a lack of clearly defined rehabilitation protocols, despite broad agreement among healthcare professionals regarding their potential benefits.

More recently, noninvasive focal tendon vibration has been investigated in several neurological disorders to improve sensory function, balance, and motor performance. Previous studies suggest that mechanical vibratory stimulation applied to the quadriceps tendon may induce sustained improvements in postural control and lower-limb muscle strength.

The present study aims to evaluate a rehabilitation program combined with tendon vibratory stimulation. The objective is to compare the effectiveness of a short-term (2-week), intensive multidisciplinary rehabilitation program focused on balance with different types of focal tendon vibration, in order to better address balance impairments and their associated complications in individuals with Charcot-Marie-Tooth disease.

Study Overview

• Status: Recruiting
• Conditions: Charcot-Marie-Tooth Disease Type 1A
• Intervention / Treatment: Other: Multidisciplinary rehabilitation program

Detailed Description

Charcot-Marie-Tooth (CMT) disease is the most common inherited peripheral neuropathy, with a prevalence ranging from 3.1 to 82.3 per 100,000 individuals. It is caused by genetic abnormalities affecting peripheral nerves. The most common form, accounting for approximately 60% of CMT cases, is Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A is characterized by a demyelinating neuropathy, autosomal dominant inheritance, and a mutation involving the PMP22 gene.

The first clinical signs of the disease typically appear before the age of 20 years and consist of a length-dependent sensorimotor impairment with distal predominance and diffuse areflexia. The disease primarily affects the lower limbs; upper limb involvement is variable and may develop after several years of disease progression. These impairments result in progressive distal muscle weakness associated with muscle atrophy, tendon contractures leading to pes cavus and claw toe deformities, and mild-to-moderate distal sensory impairment. These manifestations lead to major functional complaints among individuals with CMT, including balance disorders, gait disturbances related to ankle instability, foot clearance difficulties during the swing phase, and postural instability, resulting in frequent falls and impaired quality of life.

Currently, there is no disease-modifying treatment for CMT.

Assistive devices and lower-limb orthotic management may be prescribed. Surgical treatment may be considered in cases of severe and disabling musculoskeletal deformities.

Several rehabilitation approaches have been proposed, including endurance training and strengthening programs to improve independence in activities of daily living, as well as aerobic training programs aimed at improving functional capacity, aerobic capacity, muscle strength, and fatigue in individuals with CMT. The French National Diagnostic and Care Protocol (PNDS) for hereditary motor and sensory neuropathies associated with CMT highlights the lack of standardized rehabilitation practices and clearly defined protocols, despite a consensus among healthcare professionals regarding their potential benefits. Indeed, the literature includes only one randomized controlled trial, published in 2006, with a small sample size (n = 16), demonstrating improvements in balance, assessed using the Berg Balance Scale (BBS), following a two-week dynamic training program combining passive stretching, muscle strengthening, and standing balance exercises.

In addition, non-invasive focal tendon vibration has been proposed in several neurological disorders to improve sensory function, balance, and motor performance, particularly in individuals with diabetic peripheral neuropathy. Some studies suggest that mechanical vibratory stimulation applied to the quadriceps tendon may induce sustained improvements in postural control and lower-limb strength in women over 60 years of age.

In individuals with CMT, a pilot study involving 14 participants with CMT1A demonstrated improved balance performance on the Berg Balance Scale following three consecutive days of musculoskeletal vibration therapy applied to the quadriceps and triceps surae muscles.

In this context, we selected the Vibramoov device, a neurorehabilitation device that has already demonstrated clinical benefits in comparable studies involving other neurological disorders. Vibramoov delivers vibrations applied at the musculotendinous junction, thereby mechanically stimulating muscle spindles and reproducing the sensory signals associated with natural movement. This approach, already validated in other clinical settings, represents a promising strategy to improve balance and gait in individuals with CMT by integrating it into an intensive multidisciplinary rehabilitation program.

Therefore, it appears relevant to investigate a specific intensive multidisciplinary rehabilitation program combined with mechanical vibratory stimulation to improve functional balance and gait abilities in individuals with CMT.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Laurent Bensoussan, MD; Phone Number: +33 4 91 74 42 00; Email: laurent.bensoussan@ugecam.assurance-maladie.fr
• Study Contact Backup: Name: Clotilde Pheulpin, MD

Study Locations

• France
  • Marseille, France, 13009
    • Recruiting
    • IUR Valmante Sud
    • Contact: Laurent Bensoussan, MD; Phone Number: +33 4 91 74 42 00; Email: laurent.bensoussan@ugecam.assurance-maladie.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Individuals with genetically confirmed Charcot-Marie-Tooth disease type 1A (CMT1A), characterized by PMP22 gene duplication on chromosome 17 (17p11.2).
• Age between 18 and 65 years.
• Overall Neuropathy Limitations Scale (ONLS) score between 2 and 3/7 for the lower limbs.
• Affiliation with a social security/health insurance system.
• Written informed consent voluntarily provided after receiving information regarding the study objectives, procedures, and potential risks.

Exclusion Criteria:

• Comorbidities causing peripheral neuropathy (e.g., diabetes, renal failure, medication-induced neuropathy).
• Balance or gait disorders due to another cause.
• Individuals without a permanent residence.
• Individuals deprived of liberty by judicial or administrative decision or under legal guardianship.
• Individuals unable to understand the study objectives and procedures or unable to provide informed consent.
• Individuals unable to complete all study procedures.
• Previous use of the Vibramoov device.
• Pregnant women or women planning to become pregnant during the study period.
• Concurrent participation in another research study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: With mechanical vibratory stimulation	Other: Multidisciplinary rehabilitation program; 2-week intensive multidisciplinary rehabilitation program including physical therapy, occupational therapy, and adapted physical activity sessions, followed by balance assessments and evaluation of neuropathy status.
Sham Comparator: Without mechanical vibratory stimulation	Other: Multidisciplinary rehabilitation program; 2-week intensive multidisciplinary rehabilitation program including physical therapy, occupational therapy, and adapted physical activity sessions, followed by balance assessments and evaluation of neuropathy status.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Berg Balance Scale	The primary outcome measure of this study will be the assessment of balance using the Berg Balance Scale (BBS). The BBS is a 14-item scale in which each item is scored from 0 to 4 points and evaluates static and dynamic balance as well as fall risk. This scale has been validated in individuals with neurological disorders and has been used to assess balance impairments in individuals with Charcot-Marie-Tooth disease. The BBS has demonstrated good reliability and responsiveness to change. A change of 4 to 7 points, depending on the baseline score, has been identified as a clinically meaningful indicator of improvement in balance.	Baseline, Week 3 , Week 7, Week 15

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
CMT Neuropathy Score	This validated score provides a reliable measure of overall neuropathy severity.	Baseline, Week 3 , Week 7, Week 15
Posturography	Assessment of balance and proprioception by recording center of pressure sway on a force platform.	Baseline, Week 3, Week 7, Week 15
Timed Up and Go (TUG) test	This test is used to identify individuals at risk of falls and to assess functional mobility. Participants are timed while standing up from a chair without armrests, walking 3 meters, turning around, walking back to the chair, and sitting down.	Baseline, Week 3, Week 7, Week 15
Knee joint position sense assessment using electronic goniometers	Participants will be seated with their knees flexed at 90°. The examiner will passively move the knee to a target angle. After returning to the starting position, participants, with their eyes closed, will be asked to actively reproduce the target knee position.	Baseline, Week 3, Week 7, Week 15
Gait assessment	6-Minute Walk Test (6MWT): Assessment of walking endurance. Walking distance (meters) and number of rest stops will be recorded.	Baseline, Week 3, Week 7, Week 15
Walking speed	10-Meter Walk Test (10MWT): Assessment of self-selected walking speed by measuring the time (seconds) required to walk 10 meters.	Baseline, Week3, Week 7, Week 15
Borg Rate of Perceived Extorsion (RPE) scale	Measure of perceived exertion (Borg Rate of Perceived Extorsion (RPE) scale from 1 - "at rest" to 10 - "maximal") during during walk speed assessment.	Baseline, Week 3, Week 7, Week 15
Muscle strength assessment	Muscle strength will be assessed using the Medical Research Council (MRC) scale (0 - "no movement" to 5 - "normal force") for the tibialis anterior, extensor hallucis longus, extensor digitorum longus, triceps surae, peroneal, quadriceps, and hamstring muscles.	Baseline, Week 3, Week 7, Week 15
Patient-Reported Outcome: R-ODS questionnaire	The Rasch-built Overall Disability Scale (R-ODS) with 24 items is a questionnaire that reflects how neuropathy affects the patient's daily and social activities. Scale 0 - "impossible" to 48 - "without difficulties".	Baseline, Week 3, Week 7, Week 15
Patient-Reported Outcome: FSS Scale	The Fatigue Severity Scale (FSS) is a short questionnaire with 9 items that requires the patient to rate their level of fatigue for each item from 0 - "strongly diasagree" to 7 - "strongly agree".	Baseline, Week 3, Week 7, Week 15
Patient-Reported Outcome: QoL NMD v1.0	The Quality of Life in Neuromuscular Disease (QoL NMD v1.0) is a questionnaire with 26 items that assesses the quality of life in patients with neuromuscular disease. The final score is between 0 -"Poor" and 75 - "Excellent".	Baseline, Week 3, Week 7, Week 15
Patient-reported Outcome: NRS Scale	The Numeric Rating Scale (NRS) assesses pain intensity using a 0 - 10 ranking scale with 0 representing "no pain" and 10 "unbearable pain"	Baseline, Week 3, Week 7, Week 15

Collaborators and Investigators

• Sponsor: UGECAM PACA et Corse

Study record dates

Study Major Dates

• Study Start (Actual): April 28, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: July 21, 2026
• First Submitted That Met QC Criteria: July 21, 2026
• First Posted (Actual): July 24, 2026

Study Record Updates

• Last Update Posted (Actual): August 3, 2026
• Last Update Submitted That Met QC Criteria: July 30, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Balance; Vibration; Charcot-Marie-Tooth Disease
• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neurodegenerative Diseases; Congenital Abnormalities; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Polyneuropathies; Hereditary Sensory and Motor Neuropathy; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Charcot-Marie-Tooth Disease
• Other Study ID Numbers: 2024-A02577-40 (Other Identifier: ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No