Clinical trials: Charcot Marie Tooth Disease
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
Relapsing Multiple Sclerosis
Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis (RMS) (REMODEL-1)
To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis (RMS)
- Trial name
- NCT05147220
- Testing
- Remibrutinib, Teriflunomide
- Where
- 200 sites in United States, Argentina and 32 more
- Volunteers
- 1,000 people
- Sponsor
- Novartis Pharmaceuticals
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
Cardiac Arrest (CA), Intensive Care Unit (ICU) Admission
Kinetics and Prognostic Value of NeuroFilament Light in Post-cardiac Arrest: A Prospective Multicenter Study
The purpose of this study is to assess the prognostic value of absolute value and kinetic of neurofilament light chain (NFL) collected at different time points (at admission, 12h, 24h, 48h, 72h, 96h, and day 7 after ICU…
- Conditions
- Cardiac Arrest (CA), Intensive Care Unit (ICU) Admission, Return of Spontaneous Circulation, Comatose
- Trial name
- CINEFIL (NCT07760636)
- Testing
- Value of neurofilament light chain (NFL) at different time points
- Where
- France
- Volunteers
- 270 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
This is a prospective longitudinal natural history study with a retrospective cross-sectional arm aimed at determining the natural history of sphingosine phosphate lyase insufficiency syndrome (SPLIS), a recently recognized…
- Trial name
- SPLIS-HIS (NCT06669949)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of California, San Francisco
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
Carpal Tunnel Syndrome, Median Nerve Compression
Kinesio Taping for Pain, Median Nerve Morphology, and Quality of Life in Patients With Carpal Tunnel Syndrome
Carpal tunnel syndrome is a common condition caused by compression of the median nerve at the wrist. It can result in pain, numbness, tingling, weakness, and difficulty performing daily activities.
- Conditions
- Carpal Tunnel Syndrome, Median Nerve Compression, Entrapment Neuropathy
- Trial name
- NCT07741708
- Testing
- Kinesio Taping, Nerve and Tendon Gliding Exercises
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Istanbul Gelisim University
Charcot-Marie-Tooth Neuropathy Type 1A
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication.
- Trial name
- NCT03520751
- Testing
- scAAV1.tMCK.NTF3
- Where
- United States
- Sponsor
- Nationwide Children's Hospital
Charcot-Marie-Tooth Disease, Peripheral Neuropathy
NT-3 Levels and Function in Individuals With CMT
This study will assess the serum NT-3 levels in individuals with the diagnosis of peripheral neuropathy or any type of Charcot-Marie-Tooth Neuropathy (CMT) and correlate this with function.
- Conditions
- Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
- Trial name
- NCT05011006
- Testing
- NT3 blood draw
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Zarife Sahenk
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
Down Syndrome
Non-Invasive Brain Stimulation to Improve Language in Down Syndrome.
Down syndrome (DS) is associated with cognitive deficits, caused by alterations in neuroplasticity and synaptic transmission. Non-invasive brain stimulation techniques, such as transcranial direct current stimulation (tDCS), can…
- Trial name
- NCT07044804
- Testing
- Active Group (Active Anodal tDCS to the left IFG or Cathodal tDCS to the left IFG)
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Floriana Costanzo
Meralgia Paresthetica, Lateral Femoral Cutaneous Nerve Entrapment
The Effectiveness of Low-Level Laser Therapy in the Treatment of Meralgia Paresthetica
Patients diagnosed with meralgia paresthetica via electromyography (EMG), who present with neuropathic complaints on the lateral thigh to the Physical Medicine and Rehabilitation outpatient clinics of our hospital, will be…
- Trial name
- NCT07335822
- Testing
- Transcutaneous Electrical Nerve Stimulation (TENS), Low-Level Laser Therapy
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 36 people
- Sponsor
- Istanbul Training and Research Hospital
Traumatic Brain Injury
The Biomarkers in the Hyperbaric Oxygen Brain Injury Treatment Trial (BioHOBIT)
There are no therapeutic agents that have been shown to improve outcomes from severe traumatic brain injury (TBI). Critical barriers to progress in developing treatments for severe TBI are the lack of: 1) monitoring biomarkers…
- Trial name
- NCT04855396
- Where
- 11 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- University of Michigan
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, DIFGAN
Genotype/Phenotype Correlation of MORC2 Mutations
The Microrchidia CW-type zinc finger 2 (MORC2) gene encodes a protein expressed in all tissues and enriched in the brain. It is involved in Charcot-Marie-Tooth disease, with mire than 30 families presenting MORC2 mutations.
- Conditions
- Charcot Marie Tooth Disease, DIFGAN, Developmental Delay (Disorder), Impaired Growth
- Trial name
- PhenoMORC2 (NCT07038239)
- Testing
- Skin biopsy, Blood sample
- Where
- 12 sites in France
- Volunteers
- 45 people
- Sponsor
- Hospices Civils de Lyon
Diabetic Peripheral Neuropathy, Entrapment Neuropathy
Ultrasound-Guided Nerve Hydrodissection for Diabetic Lower Limb Entrapment Neuropathy
The goal of this clinical trial is to learn if a single session of ultrasound-guided nerve hydrodissection can relieve symptoms of diabetic lower limb entrapment neuropathy, a condition caused by compressed nerves in the leg that…
- Trial name
- HUND (NCT07625020)
- Testing
- Ultrasound-guided nerve hydrodissection treatment
- Where
- China
- Volunteers
- 266 people
- Sponsor
- Chongqing Medical University
CHARCOT MARIE TOOTH DISEASE
Evaluation of the Analgesic Efficiency of the Transcutaneous Neurostimulation in the Charcot Syndrome Marie Tooth on the Pains of Lower Limbs
The Charcot syndrome Marie Tooth is the most frequent hereditary neuromuscular diseases: there is no curative treatment at present, on the other hand precautionary measures can be taken (physiotherapy, port of splints…
- Trial name
- NCT01918826
- Testing
- TENS
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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.
Intraoperative Arterial Hypotension
Association of Intraoperative Blood Pressure Excursions Below Cerebral Autoregulatory Boundaries With Organ Injury Following Major Noncardiac Surgery
The aim of study is to investigate the clinical relevance of blood pressure (BP) excursions below cerebral autoregulatory boundaries in major noncardiac surgery.
- Trial name
- AR-NONCARDIAC (NCT05336864)
- Testing
- Non-invasive cerebral oximetry monitoring, additional perioperative blood sampling, clinical & telephone assessments
- Where
- 3 sites in Switzerland
- Volunteers
- 650 people
- Sponsor
- University Hospital, Basel, Switzerland
Charcot-Marie-Tooth Disease Type 1A
Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind…
- Trial name
- NCT06328712
- Testing
- EN001
- Where
- 3 sites in South Korea
- Volunteers
- 27 people
- Sponsor
- ENCell