Ignite Creation Date:
2026-03-26 @ 3:17 PM
Ignite Modification Date:
2026-03-31 @ 2:44 AM
Study NCT ID:
NCT07432035
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-02-25
First Post:
2026-02-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D051346', 'term': 'Mobility Limitation'}], 'ancestors': [{'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D012046', 'term': 'Rehabilitation'}, {'id': 'D015444', 'term': 'Exercise'}], 'ancestors': [{'id': 'D000359', 'term': 'Aftercare'}, {'id': 'D003266', 'term': 'Continuity of Patient Care'}, {'id': 'D005791', 'term': 'Patient Care'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D006296', 'term': 'Health Services'}, {'id': 'D005159', 'term': 'Health Care Facilities Workforce and Services'}, {'id': 'D009043', 'term': 'Motor Activity'}, {'id': 'D009068', 'term': 'Movement'}, {'id': 'D009142', 'term': 'Musculoskeletal Physiological Phenomena'}, {'id': 'D055687', 'term': 'Musculoskeletal and Neural Physiological Phenomena'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}, 'targetDuration': '5 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-02-11', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-02', 'completionDateStruct': {'date': '2028-02-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-02-24', 'studyFirstSubmitDate': '2026-02-11', 'studyFirstSubmitQcDate': '2026-02-24', 'lastUpdatePostDateStruct': {'date': '2026-02-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-02-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'the 10-Meter Walk Test (10MWT)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'the 6-Minute Walk Test (6MWT)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'the Walk-12 Questionnaire', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'the Foot and Ankle Disability Index (FADI)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'the American Orthopaedic Foot and Ankle Society (AOFAS) Hindfoot Score', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'the 12-Item Short Form Health Survey (SF-12)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'the Charcot-Marie-Tooth Examination Score (CMTES)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'Lower Limb Muscle Strength (Medical Research Council Scale)', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}, {'measure': 'Ankle Joint Range of Motion', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': 'Ankle dorsiflexion and plantarflexion range of motion were measured using a goniometer and expressed in degrees, with higher values reflecting greater joint mobility.'}, {'measure': 'the Incidence of Disease-Related Clinical Symptoms', 'timeFrame': 'baseline (prior to treatment initiation), 6 months, 1 year, and 2 years after treatment initiation', 'description': '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.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Charcot Marie Tooth disease', 'Walking performance', 'Functional surgery', 'Physical therapy', 'Gait impairment', 'Propensity score matching'], 'conditions': ['Charcot Marie Tooth Disease (CMT)', 'Pes Cavovarus', 'Surgery', 'Exercise Training', 'Neuromuscular Diseases (NMD)', 'Gait Disorders']}, 'descriptionModule': {'briefSummary': '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.\n\nCMT 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.\n\nThe 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.\n\nResearchers 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.\n\nParticipants 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.\n\nThe findings from this study may help clinicians and individuals with CMT better understand how different treatment strategies influence walking function over time.', 'detailedDescription': '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.\n\nBoth 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.\n\nThis 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.\n\nTo 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.\n\nThe 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.\n\nFollow-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.\n\nLongitudinal 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.\n\nQuality assurance procedures included standardized evaluator training, predefined analytic plans, and structured data management processes to minimize bias and enhance methodological rigor.\n\nThrough 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '12 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '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.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants aged 12 years or older at the time of enrollment\n* Genetically or clinically confirmed CMT, based on established diagnostic criteria\n* Presence of foot deformity and/or gait impairment attributable to CMT, as determined by a treating clinician\n* Ability to ambulate at least 10 meters, with or without assistive devices\n* Medically eligible for either functional surgical intervention or structured physical therapy, as determined by the treating team\n* Willingness and ability to participate in longitudinal follow-up assessments\n* Provision of written informed consent\n\nExclusion Criteria:\n\n* Previous major foot or ankle surgery that substantially altered lower-limb biomechanics\n* Presence of non-CMT-related neurological disorders affecting gait (eg, stroke, Parkinson disease, multiple sclerosis)\n* Severe musculoskeletal conditions unrelated to CMT that limit walking ability (eg, advanced hip or knee osteoarthritis)\n* Active lower-limb infection, ulceration, or acute injury at the time of enrollment\n* Severe cognitive impairment or psychiatric condition precluding reliable participation\n* Medical contraindications to surgery or exercise-based rehabilitation, when relevant\n* Inability to complete baseline functional assessments or anticipated inability to complete follow-up'}, 'identificationModule': {'nctId': 'NCT07432035', 'acronym': 'CMT-WALK', 'briefTitle': 'Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study', 'organization': {'class': 'OTHER', 'fullName': 'Peking University Third Hospital'}, 'officialTitle': 'Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study', 'orgStudyIdInfo': {'id': 'PekingUTH WZQ-CMT'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Functional Surgery plus Rehabilitation (FS Group)', 'interventionNames': ['Procedure: Functional Surgery plus Rehabilitation']}, {'label': 'Physical Exercise Alone (PE Group)', 'interventionNames': ['Behavioral: Physical Exercise']}], 'interventions': [{'name': 'Functional Surgery plus Rehabilitation', 'type': 'PROCEDURE', 'description': '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.', 'armGroupLabels': ['Functional Surgery plus Rehabilitation (FS Group)']}, {'name': 'Physical Exercise', 'type': 'BEHAVIORAL', 'description': '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.', 'armGroupLabels': ['Physical Exercise Alone (PE Group)']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Zhuoqi Wei, MD', 'role': 'CONTACT', 'email': '13838384087@163.com', 'phone': '+8613838384087'}, {'name': 'Yang Lv, MD, PhD', 'role': 'CONTACT', 'email': 'lvyang42@126.com', 'phone': '+8618513112060'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Peking University Third Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Zhou Fang', 'investigatorAffiliation': 'Peking University Third Hospital'}}}}