Clinical trials: Charcot Marie Tooth Disease
Multiple Sclerosis, Neurofilament Light Chain
To Study the Pathophysiological Features of Multiple Sclerosis
Multiple sclerosis (MS) is the most common chronic inflammatory disease of the central nervous system1, whose demyelination is the pathological hallmark.
- Conditions
- Multiple Sclerosis, Neurofilament Light Chain, Glial Fibrillary Acidic Protein
- Trial name
- NCT04242056
- Testing
- 18F-PM-PBB3
- Volunteers
- 38 people
- Sponsor
- Chang Gung Memorial Hospital
Charcot-Marie-Tooth Disease Type 1A
Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers
This is a 2-year follow-up study of a cohort of 60 CMT1A patients. The objective is to identify markers allowing to better understand the phenotypic variability observed on patients with CMT1A, to identify predictive markers of…
- Trial name
- CMT-TOOLS (NCT02596191)
- Testing
- Clinical evaluation, electrophysiological record, Muscle MRI
- Where
- 2 sites in France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
MS (Multiple Sclerosis), Multiple Sclerosis (MS) - Relapsing-remitting
Comparison of Efficacy and Safety in Patients Switching From MabThera® to Rixathon® in Relapsing-Remitting Multiple Sclerosis
This observational study investigates if switching from MabThera® to Rixathon® is associated with changes in disease activity and/or safety in people with multiple sclerosis (MS).
- Conditions
- MS (Multiple Sclerosis), Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (Relapsing Remitting)
- Trial name
- NCT07235644
- Where
- Sweden
- Volunteers
- 184 people
- Sponsor
- Uppsala University
Charcot-Marie-Tooth Disease Type 1A
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
Charcot-Marie-Tooth (CMT) disease is caused by a genetic abnormality involving the PMP22 gene, resulting in demyelination of the peripheral nerves.
- Trial name
- EQUIVIB-CMT (NCT07726043)
- Testing
- Multidisciplinary rehabilitation program
- Where
- France
- Volunteers
- 30 people
- Sponsor
- UGECAM PACA et Corse
Appendicitis
Are Post-operative Antibiotics Indicated in Simple Appendicitis?
Hypothesis: A single dose of prophylactic antibiotics is as effective as a three dose regime in preventing post-operative complications in paediatric patients with simple appendicitis.
- Trial name
- NCT01420367
- Testing
- metronidazole and cephalzolin
- Where
- Australia
- Volunteers
- 300 people
- Sponsor
- Monash University
Relapsing Multiple Sclerosis
Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis (RMS) (REMODEL-2)
To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis (RMS)
- Trial name
- NCT05156281
- Testing
- Remibrutinib, Teriflunomide
- Where
- 223 sites in United States, Argentina and 25 more
- Volunteers
- 1,007 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
The purpose of this study is to learn about rates of patient-reported disease progression in patients with motor neuron diseases (amyotrophic lateral sclerosis, progressive muscular atrophy, primary lateral sclerosis, hereditary…
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02852278
- Testing
- Web-based Survey
- Volunteers
- 410 people
- Sponsor
- University of South Florida
Charcot-Marie-Tooth Disease, Type 1
Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E
Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disease that causes causes muscle atrophy, muscle weakness, sensory loss, balance disorder, gait disorder, blindness, hearing disorder, breathing disorder, vocal…
- Trial name
- NCT06218134
- Testing
- human Wharton's jelly-derived mesenchymal stem cells
- Where
- South Korea
- Volunteers
- 3 people
- Sponsor
- Samsung Medical Center
Charcot-Marie-Tooth Type 1A
Pilot Study to Evaluate Fatigue and Fatigability Profiles and Their Impact on Walking and Balance in Patients With Charcot-Marie-Tooth Type 1A Disease Using a Global Approach in Comparison to Healthy Volunteers
Charcot-Marie-Tooth disease type 1A is the most common form of inherited neuropathy that causes peripheral nerve damage in all four limbs.
- Trial name
- FatMobCMT1A (NCT07066683)
- Testing
- CLINICAL ASSESSMENT OF BODY FUNCTION (PROM AND PHYSICAL EXAMINATION) and ACTIVITIES AND PARTICIPATION
- Where
- France
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Carpal Tunnel Syndrome, Compression Neuropathy, Carpal Tunnel
Diagnosis of Carpal Tunnel Syndrome: Effectiveness of Diagnostic Tests. Clinical Trial of Accuracy
There is no golden universal standard for the diagnosis of Carpal Tunnel Syndrome (CTS). In this scenario, for a comparison of the effectiveness of the principal diagnostic tests CTS should determine how they affect the…
- Conditions
- Carpal Tunnel Syndrome, Compression Neuropathy, Carpal Tunnel, Median Neuropathy, Carpal Tunnel
- Trial name
- DCTS (NCT02553811)
- Testing
- accuracy diagnostic
- Where
- Brazil
- Volunteers
- 115 people
- Sponsor
- Federal University of São Paulo
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia
Natural History Study of Patients with HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations
- Conditions
- Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease
- Trial name
- NCT05848271
- Testing
- Patient Registry, Dry blood spots sampling
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of California, San Diego
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
Charcot-Marie-Tooth Disease, Healthy
Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Charcot-Marie-Tooth disease research and drug development by using cells from the body (such as skin or blood cells) to make…
- Trial name
- NCT06203093
- Testing
- Biological Sample Collection
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- New York Stem Cell Foundation Research Institute
Hydrocephalus, Normal Pressure, Biochemical Lesions Head Region
Role of Brain Specific Biomarkers in Hydrocephalus
Normal pressure hydrocephalus (NPH) is a neurodegenerative disease of unclear etiology characterized by a clinical trias named after the neurosurgeon Hakim.
- Conditions
- Hydrocephalus, Normal Pressure, Biochemical Lesions Head Region, Brain Damage
- Trial name
- NCT06083233
- Testing
- Lumbar puncture, External lumbar drainage, Ventriculo-peritoneal shunt placement
- Where
- Czechia
- Volunteers
- 32 people
- Sponsor
- University Hospital Hradec Kralove
Charcot-Marie-Tooth Disease
Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.
- Trial name
- SYNAPSE-CMT (NCT06482437)
- Testing
- NMD670
- Where
- 19 sites in United States, Belgium and 3 more
- Volunteers
- 81 people
- Sponsor
- NMD Pharma A/S
Charcot-Marie-Tooth Disease, Type 1A
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
- Trial name
- NCT07140614
- Testing
- EDK060, dose A, EDK060, dose B, EDK060, dose C
- Where
- 4 sites in Canada
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Carpal Tunnel Syndrome Bilateral
Superb Microvascular Imaging of Median Nerve in Carpal Tunnel Syndrome: A Novel Technique in Diagnostic Ultrasound
The aim of the study was to determine whether there is a correlation between the increased intraneural flow as measured using Doppler ultrasound (US) and superb microvascular imaging (SMI) methods and the severity of CTS as…
- Trial name
- NCT03213847
- Testing
- Superb microvascular imaging (SMI) (Toshiba)
- Where
- Turkey (Türkiye)
- Volunteers
- 80 people
- Sponsor
- Uşak University
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.
Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study
This cohort study (participants with CMT and control participants) has two parts (Part 1: CMT1A cohort; Part2: CMT1B, CMT2A and CMTX1 cohort) and is proposed to take place over 3 years across three sites.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease, Type X
- Trial name
- NCT03550300
- Where
- United Kingdom
- Volunteers
- 130 people
- Sponsor
- University College, London
Charcot-Marie-Tooth Type 1A Neuropathy
Fall Risk Assessment in a Population of Charcot-Marie-Tooth Disease Type 1A (CMT 1A) by Timed Up and Go Test
The main objective of this study is to explore the relationship between the onset of fall and the time taken to complete the Timed Up and Go test (TUG) in this CMT1A patient population.
- Trial name
- DeteCTCMT (NCT05142059)
- Testing
- Exploratory physiopathology study, including non-invasive functional explorations carried out in patients with Charcot-Marie-Tooth disease type 1A
- Where
- France
- Volunteers
- 42 people
- Sponsor
- University Hospital, Clermont-Ferrand