Charcot Marie Tooth Disease (CMT)CMT-WALKNCT07432035
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study
An observational study of Charcot Marie Tooth Disease (CMT) and Pes Cavovarus, run by Peking University Third Hospital.
Trial summary
The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking. CMT is an inherited nerve condition that can cause muscle weakness, loss of sensation, and foot deformities. Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery
- What is being tested?
- Functional Surgery plus Rehabilitation and Physical Exercise
- Type of treatment
- Surgery or procedure, Lifestyle or therapy programme
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- 11 Feb 2026to 10 Feb 2028
- How long will I take part?
- 5 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study population comprises adults with Charcot-Marie-Tooth disease (CMT) evaluated and treated at a tertiary referral center specializing in neuromuscular and foot and ankle disorders. Participants were identified from consecutive clinical encounters beginning in February 2017, incorporating both retrospectively and prospectively collected data. Eligible individuals had a genetically or clinically confirmed diagnosis of CMT and presented with foot deformity and gait impairment attributable to peripheral neuropathy. All participants were ambulatory, with or without assistive devices, at the time of enrollment. Management strategies included functional surgical intervention followed by rehabilitation or structured physical therapy alone, reflecting routine clinical practice. Longitudinal outcome data were obtained through standardized assessments conducted at baseline and predefined follow-up time points throughout the study period.
Who may be able to join
- Participants aged 12 years or older at the time of enrollment
- Genetically or clinically confirmed CMT, based on established diagnostic criteria
- Presence of foot deformity and/or gait impairment attributable to CMT, as determined by a treating clinician
- Ability to ambulate at least 10 meters, with or without assistive devices
- Medically eligible for either functional surgical intervention or structured physical therapy, as determined by the treating team
- Willingness and ability to participate in longitudinal follow-up assessments
- Provision of written informed consent
Who cannot join
- Previous major foot or ankle surgery that substantially altered lower-limb biomechanics
- Presence of non-CMT-related neurological disorders affecting gait (eg, stroke, Parkinson disease, multiple sclerosis)
- Severe musculoskeletal conditions unrelated to CMT that limit walking ability (eg, advanced hip or knee osteoarthritis)
- Active lower-limb infection, ulceration, or acute injury at the time of enrollment
- Severe cognitive impairment or psychiatric condition precluding reliable participation
- Medical contraindications to surgery or exercise-based rehabilitation, when relevant
- Inability to complete baseline functional assessments or anticipated inability to complete follow-up
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.
CMT is an inherited nerve condition that can cause muscle weakness, loss of sensation, and foot deformities. These changes often make walking difficult and can reduce independence and quality of life. Treatment options commonly include physical therapy alone or surgery to correct foot alignment followed by rehabilitation. However, it is not clear whether one approach leads to better long-term walking outcomes.
Read more
The main question this study aims to answer is whether individuals who undergo functional foot surgery followed by rehabilitation experience different changes in walking ability over time compared with those who receive structured physical therapy alone.
Researchers will compare walking performance between these two treatment groups over a period of up to two years. Walking ability will be evaluated using standardized walking tests and patient questionnaires.
Participants included in this study are individuals with CMT-related foot deformities that affect walking and who received either surgery followed by rehabilitation or physical therapy alone. Researchers will analyze changes in walking ability over time and determine how many participants achieve meaningful improvement.
The findings from this study may help clinicians and individuals with CMT better understand how different treatment strategies influence walking function over time.
Read the detailed description
Charcot-Marie-Tooth disease (CMT) is a genetically heterogeneous inherited peripheral neuropathy characterized by progressive distal muscle weakness, sensory impairment, and neuromuscular foot deformities. These impairments frequently alter gait mechanics and reduce walking capacity. In the absence of disease-modifying therapies, clinical management focuses on optimizing functional mobility and mitigating secondary musculoskeletal consequences.
Both conservative physical therapy and functional surgical correction followed by rehabilitation are widely used to address gait-limiting deformities in CMT. Physical therapy primarily targets strength, balance, and motor control, whereas surgical intervention aims to improve structural alignment and rebalance muscle-tendon forces. Despite routine implementation of both approaches, comparative evidence regarding their longitudinal effects on objective walking performance remains limited.
This study uses a bidirectional observational cohort design integrating retrospectively ascertained treatment exposure with prospectively collected longitudinal outcomes. Individuals with CMT-related foot deformities affecting walking who were treated between January 2017 and January 2024 were identified from an institutional clinical database. Participants were classified according to treatment strategy: (1) functional surgical correction followed by standardized postoperative rehabilitation, or (2) structured physical therapy alone. Treatment allocation was determined by routine clinical decision-making rather than randomization.
To address confounding inherent in nonrandomized comparisons, propensity score matching was applied to balance baseline demographic and disease-related characteristics between treatment groups. Covariates included factors such as age at presentation, genetic subtype when available, and baseline disease severity. Balance diagnostics were assessed to confirm adequate group comparability following matching.
The primary focus of the study is longitudinal walking performance. Standardized performance-based assessments of habitual walking speed and functional walking capacity were administered at baseline and during follow-up. A multidimensional framework was used to capture impairment-level measures, task-specific walking performance, and patient-reported health status, enabling evaluation of both functional change and broader clinical impact.
Follow-up assessments were conducted at prespecified intervals up to 2 years after treatment initiation. Baseline data were extracted from electronic medical records, and follow-up data were collected prospectively through structured outpatient assessments and standardized remote evaluations when necessary. Consistent assessment procedures were implemented across time points to enhance reproducibility.
Longitudinal analyses were performed using repeated-measures modeling to evaluate within- and between-group changes over time, with particular emphasis on group-by-time interactions. Multivariable regression analyses were conducted to explore associations between treatment strategy and changes in walking performance while adjusting for relevant clinical covariates. Clinically meaningful improvement was evaluated using established minimal clinically important difference thresholds for walking-related outcomes. Missing data were handled using multiple imputation techniques.
Quality assurance procedures included standardized evaluator training, predefined analytic plans, and structured data management processes to minimize bias and enhance methodological rigor.
Through this longitudinal and performance-centered design, the study aims to clarify the functional consequences of surgical and conservative management strategies for gait-limiting CMT-related foot deformities and to better characterize the trajectory and clinical relevance of walking outcomes over time.
- Conditions
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery, Exercise Training, Neuromuscular Diseases (NMD), Gait Disorders
- Being tested
- Functional Surgery plus Rehabilitation, Physical Exercise
- Keywords
- Charcot Marie Tooth disease, Walking performance, Functional surgery, Physical therapy, Gait impairment, Propensity score matching
- Medical subject headings
- Charcot-Marie-Tooth Disease, Neuromuscular Diseases, Mobility Limitation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Functional Surgery plus RehabilitationProcedure or surgery
FS group underwent individualized surgical correction aimed at improving foot alignment, restoring muscle-tendon balance, and optimizing walking mechanics. Surgical procedures included soft tissue release, tendon transfers to augment weakened muscles, and calcaneal osteotomy for hindfoot malalignment. Arthrodesis was reserved for rigid deformities or painful osteoarthritis not amenable to realignment. All surgically treated participants followed a standardized postoperative rehabilitation program, consisting of an initial 2-week period of immobilization with non-weight-bearing, followed by partial weight-bearing with a removable orthosis from weeks 2 to 6, progression to full weight-bearing between weeks 6 and 12, and a structured rehabilitation phase from weeks 12 to 24 emphasizing strengthening, balance training, and task-specific gait retraining. Afterward, participants continued rehabilitation using the same protocol applied to the physical therapy group.
Physical ExerciseBehavioural
Participants in the PE group received structured physical therapy without surgical intervention. Programs were individualized based on disease severity and functional capacity to improve walking ability, balance, and lower-limb coordination. Therapy included aerobic exercise, ankle mobility exercises, lower-limb stretching, and balance training, all performed within the available range of motion. After an initial supervised phase, participants transitioned to a home-based, self-supervised program performed approximately twice weekly for up to 12 months. Aerobic training was conducted at moderate intensity using a stationary bicycle or seated alternatives when needed. Exercises were modified as necessary to avoid pain or excessive fatigue.
| Study group | Role | What they receive |
|---|---|---|
| Functional Surgery plus Rehabilitation (FS Group) | Not described Receives: Procedure: Functional Surgery plus Rehabilitation | |
| Physical Exercise Alone (PE Group) | Not described Receives: Behavioral: Physical Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
the 10-Meter Walk Test (10MWT)
Habitual walking speed was determined using the 10-Meter Walk Test (10MWT) and expressed in meters per second (m/s), with higher values reflecting faster walking speed.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the 6-Minute Walk Test (6MWT)
Functional walking capacity was assessed using the 6-Minute Walk Test (6MWT) and expressed in meters walked during 6 minutes, with higher values reflecting greater walking endurance.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
Secondary outcomes (8)
the Walk-12 Questionnaire
Walking-related disability was assessed using the 12-item Walk-12 questionnaire and expressed as total score according to standardized scoring procedures, with higher scores reflecting greater perceived walking limitation.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the Foot and Ankle Disability Index (FADI)
Foot and ankle-specific functional status was assessed using the Foot and Ankle Disability Index (FADI) and expressed as total score according to standardized scoring procedures, with higher scores reflecting better function.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the American Orthopaedic Foot and Ankle Society (AOFAS) Hindfoot Score
Hindfoot function was assessed using the American Orthopaedic Foot and Ankle Society (AOFAS) Hindfoot Score and expressed in points on a 0-100 scale, with higher scores reflecting better clinical function.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the 12-Item Short Form Health Survey (SF-12)
Health-related quality of life was assessed using the 12-Item Short Form Health Survey (SF-12), including Physical Component Summary (PCS) and Mental Component Summary (MCS) scores, expressed in standardized score units, with higher scores reflecting better health status.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the Charcot-Marie-Tooth Examination Score (CMTES)
Disease severity was assessed using the Charcot-Marie-Tooth Examination Score (CMTES) and expressed as total score according to standardized scoring criteria, with higher scores reflecting greater disease severity.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
Lower Limb Muscle Strength (Medical Research Council Scale)
Lower limb muscle strength was assessed using the Medical Research Council (MRC) grading scale and expressed as muscle strength grade, with higher grades reflecting greater muscle strength.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
Ankle Joint Range of Motion
Ankle dorsiflexion and plantarflexion range of motion were measured using a goniometer and expressed in degrees, with higher values reflecting greater joint mobility.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
the Incidence of Disease-Related Clinical Symptoms
The occurrence of disease-related clinical symptoms, including ankle sprain, plantar callus formation, foot pain, and limping, was assessed by structured patient report and clinical record review. The outcome was expressed as the number of participants experiencing each symptom during the follow-up period. Higher values reflect greater symptom burden.
Measured: baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2026
- Study start11 Feb 2026 (estimated)
- Primary completion10 Feb 2027 (estimated)
- Study completion10 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot Marie Tooth Disease (CMT)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Peking University Third Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhou Fang, Peking University Third Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07432035
- Sponsor's protocol ID
- PekingUTH WZQ-CMT
- Acronym
- CMT-WALK
- Status last verified
- Feb 2026
- First submitted
- 11 Feb 2026
- Last updated
- 25 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided