Clinical trials: CMT1A
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
Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy, Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
- Trial name
- NCT02967679
- Testing
- MD1003
- Where
- France
- Volunteers
- 15 people
- Sponsor
- MedDay Pharmaceuticals SA
Charcot-Marie-Tooth Disease, Type IA
Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)
CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures.
- Trial name
- NCT03386266
- Where
- Germany
- Volunteers
- 156 people
- Sponsor
- University Medical Center Goettingen
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
Charcot-Marie-Tooth Type 1A Neuropathy
Correlation Between Clinical and Electrophysiological Phenotypes in a Population of Patients With Neuropathy Charcot-Marie-Tooth Disease Type 1A
This exploratory cross-sectional study proposes, firstly, to objectify in a population of Charcot-Marie-Tooth disease type 1A (CMT 1A)if there is a correlation between the recording of electrical parameters and upper limb muscle…
- Trial name
- CCA1 (NCT01750710)
- Where
- France
- Volunteers
- 37 people
- Sponsor
- University Hospital, Clermont-Ferrand
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathies
Ascorbic Acid Treatment in CMT1A Trial (AATIC)
Charcot-Marie-Tooth type IA (CMT1A) is the most prevalent hereditary peripheral neuropathy. Demyelination of peripheral nerves is the hallmark of CMT1A.
- Trial name
- AATIC (NCT00271635)
- Testing
- ascorbic acid
- Where
- Netherlands
- Volunteers
- 13 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2
This project includes two projects. One is looking for new genes that cause Charcot Marie Tooth disease (CMT). The other is looking for genes that do not cause CMT, but may modify the symptoms a person has.
- Trial name
- INC-6602 (NCT01193088)
- Where
- 24 sites in United States, Australia and 4 more
- Volunteers
- 1,050 people, healthy volunteers welcome
- Sponsor
- University of Iowa
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 IA, Peripheral Neuropathy Hereditary
Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment
Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common hereditary peripheral neuropathy, affecting approximately 26,000 patients in France.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA, Peripheral Neuropathy Hereditary, Motor Activity, Walking, Difficulty
- Trial name
- CMT1A-HOME (NCT07591779)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Limoges
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
Charcot-Marie-Tooth Disease
Change in MUNIX in Patients With CMT1A Undergoing a Home Ankle Strengthening Program Versus Standard of Care
Here the investigators aim to show that a focused lower extremity resistance strength training program in patients with Charcot-Marie-Tooth disease (CMT) results in increased motor strength of ankle plantar- and dorsi-flexion.
- Trial name
- NCT03715283
- Testing
- Home Ankle Strengthing Program, Standard of Care
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Charcot-Marie-Tooth Type 1A
A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A
This is a randomized, double-blind, placebo-controlled and multicenter 3 phase trial evaluating the therapeutic effect and safety of CMT1A by PXT3003.
- Trial name
- NCT05092841
- Testing
- PXT3003
- Where
- 25 sites in China
- Volunteers
- 176 people
- Sponsor
- Tasly GeneNet Pharmaceuticals Co., Ltd
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
Charcot-Marie-Tooth Disease
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be…
- Trial name
- PREMIER (NCT04762758)
- Testing
- (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 350 people
- Sponsor
- Pharnext S.C.A.
Charcot-Marie-Tooth Disease, Type Ia
High Dose Ascorbic Acid Treatment of CMT1A
This study will look at the impact of ascorbic acid (Vitamin C) on the progression of disease in people with CMT1A as compared to volunteers receiving a placebo.
- Trial name
- NCT00484510
- Testing
- Ascorbic acid (Vitamin C)
- Where
- 3 sites in United States
- Volunteers
- 110 people
- Sponsor
- Wayne State University
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
Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
- Conditions
- Charcot-Marie-Tooth Disease, Hereditary Neuropathy With Liability to Pressure Palsies, Genetic Disorders
- Trial name
- NCT01401257
- Testing
- PXT3003 Low dose, PXT3003 Intermediate Dose, PXT3003 High Dose
- Where
- 6 sites in France
- Volunteers
- 80 people
- Sponsor
- Pharnext S.C.A.
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)
Charcot-Marie-Tooth neuropathy type 1A (CMT1A) is one of the most common inherited neurological disorders. The study will evaluate the efficacy and safety of an innovative rehabilitation protocol constituted by exercises at the…
- Trial name
- TreSPE (NCT01289704)
- Testing
- TreSPE, SPE
- Where
- 4 sites in Italy
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- University of Genova
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 Type 1A
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A).
- Trial name
- PLEO-CMT (NCT02579759)
- Testing
- PXT3003 dose 1, PXT3003 dose 2
- Where
- 30 sites in United States, Belgium and 6 more
- Volunteers
- 323 people
- Sponsor
- Pharnext S.C.A.