Clinical trials: CMT - Charcot-Marie-Tooth Disease
Foot Drop, Charcot-Marie-Tooth Disease Type 1A
Muscle MRI Fat Fraction Before Surgery to Predict Strength Recovery After Foot-Drop Surgery in Charcot-Marie-Tooth Disease
The purpose of this prospective observational cohort study is to determine whether the amount of fat inside the donor muscles before surgery, measured with a magnetic resonance imaging (MRI) technique called Dixon fat fraction…
- Conditions
- Foot Drop, Charcot-Marie-Tooth Disease Type 1A, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease (CMT)
- Trial name
- CMT-FF-TPT (NCT07894536)
- Testing
- Tibialis posterior tendon transfer, Individualized adjunct procedures for cavovarus deformity correction, Ankle-foot orthosis (AFO)
- Volunteers
- 100 people
- Sponsor
- Peking University Third Hospital
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth
Natural History Study for Charcot Marie Tooth Disease
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden…
- Conditions
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
- Trial name
- NCT05902351
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Hereditary Neuropathy Foundation
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
- Trial name
- NCT07223632
- Testing
- VCA-894A
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Vanda Pharmaceuticals
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)
Confirmatory Study of Govorestat in CMT-SORD
This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will assess the effect of govorestat compared to placebo in patients with…
- Trial name
- NCT07191912
- Testing
- Govorestat
- Where
- 13 sites in United States, Australia and 5 more
- Sponsor
- Applied Therapeutics, Inc.
CMT - Charcot-Marie-Tooth Disease, CMT1A
AUTONOMOUS DISORDERS IN CMT
Hereditary neuropathies are a phenotypically and genetically heterogeneous group of disorders. One of the most common forms is Charcot-Marie-Tooth neuropathy (CMT), which can be further divided into demyelinating (CMT1) and…
- Conditions
- CMT - Charcot-Marie-Tooth Disease, CMT1A, CMT (Charcot Marie Tooth Disease)
- Trial name
- CMT-autonom (NCT07570446)
- Where
- Germany
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
CMT, CMT (Charcot Marie Tooth Disease)
TREMOR IN CHARCOT-MARIE-TOOTH
Tremor is a symptom that has already been described in many case reports and case series concerning patients with Charcot-Marie-Tooth (CMT) disease.
- Conditions
- CMT, CMT (Charcot Marie Tooth Disease), Charcot Marie Tooth Disease (CMT), Charcot Marie Tooth Disease
- Trial name
- CMT-TREM (NCT07570459)
- Where
- Germany
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
CMT, CMT (Charcot Marie Tooth Disease)
A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)
The most common inherited neuropathy is Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a duplication of the gene expressing PMP22.
- Conditions
- CMT, CMT (Charcot Marie Tooth Disease), CMT - Charcot-Marie-Tooth Disease, CMT1A
- Trial name
- CMT-MODs (NCT07476365)
- Where
- Germany
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
CMT1A, CMT (Charcot Marie Tooth Disease)
Longitudinal Biomarkers With Selected Outcome Measures In CMT
The goal of this study is to better understand the progression of CMT1A and identify risk factors influencing disease course. CMT1A, the most common hereditary peripheral neuropathy, shows high variability in individual…
- Conditions
- CMT1A, CMT (Charcot Marie Tooth Disease), CMT 1A, CMT - Charcot-Marie-Tooth Disease
- Trial name
- CMT-BIO-Extend (NCT06794489)
- Where
- Germany
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
Charcot Marie Tooth Disease (CMT), Pes Cavovarus
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort 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.
- Conditions
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery, Exercise Training
- Trial name
- CMT-WALK (NCT07432035)
- Testing
- Functional Surgery plus Rehabilitation, Physical Exercise
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Cerebral Palsy (CP), Stroke
High Intensity Exercise and Improving Physical Activity Among People With Neurologic Dysfunction
The Hawks in Motion (HIM) High Intensity Exercise program is designed to implement the American Physical Therapy Clinical Practice Guidelines and American College of Sports Medicine recommendations for exercise for people with…
- Conditions
- Cerebral Palsy (CP), Stroke, Spinal Cord Injury, Traumatic Brain Injury
- Trial name
- NCT06945835
- Testing
- Participate in high intensity exercise of at least 20 minutes in 60 minutes session of RPE greater than 6.
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Hartford
Leukodystrophy, White Matter Disease
The Myelin Disorders Biorepository Project
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects.
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome
- Trial name
- MDBP (NCT03047369)
- Where
- 23 sites in United States
- Volunteers
- 12,000 people
- Sponsor
- Children's Hospital of Philadelphia
Polyneuropathy, Charcot Marie Tooth Disease (CMT)
Effects of Resistance Exercises in Hereditary Sensory-Motor Neuropathy (Charcot-Marie-Tooth Disease)
The goal of this clinical trial is to compare the effects of an 8-week resistance exercise training program on skeletal muscle quality, functional capacity, and quality of life in young individuals aged 15 to 29 years, with and…
- Trial name
- PNP (NCT07152197)
- Testing
- Progressive Resistance Exercise Training
- Where
- 2 sites in Chile
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Universidad de La Frontera
Charcot Marie Tooth Disease (CMT)
CMT Gait, Mobility, Balance - AOFAS Grant
The overall objective of the proposed research is to begin to better understand the potential benefits and limitations of ankle -foot orthosis (AFO) use in the context of mobility and balance during gait for individuals with…
- Trial name
- NCT07188415
- Testing
- Ankle-foot Orthoses
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Bopha Chrea
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Charcot-Marie-Tooth Disease (CMT)
Research of SORD-CMT Natural History and Epalrestat Treatment
Charcot-Marie-Tooth disease (CMT) is a group of the most common hereditary peripheral neuropathy with high clinical and genetic heterogeneity.
- Trial name
- SORD-CMT (NCT05777226)
- Testing
- Epalrestat
- Volunteers
- 30 people
- Sponsor
- The Third Xiangya Hospital of Central South University
CMT - Charcot-Marie-Tooth Disease, Peripheral Neuropathy
Effect of Orthoses and Underfoot Vibration on Balance in Neuropathy
A cross-sectional study which will look at the effects of AFO's and vibrating insoles on balance performance in people with CMT disease.
- Conditions
- CMT - Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Hereditary Peripheral Neuropathy, CMT1A
- Trial name
- NCT03278093
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- St George's, University of London
CIDP (Chronic Inflammatory Demyelinating Polyradiculoneuropathy), CMT (Charcot Marie Tooth Disease)
Diffusion Tensor Imaging in Chronic Inflammatory Demyelinating Polyneuropathy (PIDC)
The main purpose of this study is to assess the clinical feasibility of diffusion tensor imaging (DTI) for the diagnosis of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
- Trial name
- PIDC (NCT03460951)
- Testing
- cervical MRI
- Where
- France
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand