Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study

NCT07432035 | Not Yet Recruiting DMC

• 1 other identifier: PekingUTH WZQ-CMT
• Study Type: observational
• Target: 200
• Locations: 0 countries
• Sites: N/A

Brief 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. 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. 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.

Conditions

Charcot Marie Tooth Disease (CMT); Pes Cavovarus; Surgery; Exercise Training; Neuromuscular Diseases (NMD); Gait Disorders

Keywords

Charcot Marie Tooth disease; Walking performance; Functional surgery; Physical therapy; Gait impairment; Propensity score matching

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation

Secondary Outcomes (8)

• the Walk-12 Questionnaire

  baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation

• the Foot and Ankle Disability Index (FADI)

  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

  baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation

• the 12-Item Short Form Health Survey (SF-12)

  baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation

• the Charcot-Marie-Tooth Examination Score (CMTES)

  baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation

Study Arms (2)

Functional Surgery plus Rehabilitation (FS Group)

Procedure: Functional Surgery plus Rehabilitation

Physical Exercise Alone (PE Group)

Behavioral: Physical Exercise

Interventions

Functional Surgery plus Rehabilitation PROCEDURE

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.

Functional Surgery plus Rehabilitation (FS Group)

Physical Exercise BEHAVIORAL

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.

Physical Exercise Alone (PE Group)

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Peking University Third Hospital: lead

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease; Neuromuscular Diseases; Mobility Limitation

Interventions

Rehabilitation; Exercise

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Aftercare; Continuity of Patient Care; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services; Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Central Study Contacts

Zhuoqi Wei, MD

CONTACT

+8613838384087; 13838384087@163.com

Yang Lv, MD, PhD

CONTACT

+8618513112060; lvyang42@126.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 25, 2026
• Study Start: February 11, 2026
• Primary Completion (Estimated): February 10, 2027
• Study Completion (Estimated): February 10, 2028
• Last Updated: February 25, 2026

Record last verified: 2026-02