Clinical trials: Charcot-Marie-Tooth Disease, Type Ia
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.
Chronic Inflammatory Demyelinating Polyneuropathy, Charcot-Marie-Tooth Disease, Type IA
Quantification of Nerve Stiffness in Neuropathies
This project aims to understand how nerve mechanical properties are altered in patients with rare peripheral neuropathies . Stiffness of various peripheral nerves will be measured using ultrasound shear wave elastography.
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Charcot-Marie-Tooth Disease, Type IA, Anti-MAG Neuropathy
- Trial name
- Nerf_SSI (NCT03397303)
- Testing
- elastography measurements
- Where
- France
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
CMT
The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients
- Trial name
- NCT05361031
- Testing
- Engensis (VM202)
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Helixmith Co., Ltd.
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, 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
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
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 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 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
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
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 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
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
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