Clinical trials: CMT 1A, active
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 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 Type 1A
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A using multi-omics analysis.
- Trial name
- CMT-MODS (NCT07049588)
- Testing
- Quantitative neuromuscular MRI, Skin biopsy, Clinical scores
- Where
- France
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
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
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
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
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 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
Charcot-Marie-Tooth Neuropathy Type 1A, Hereditary Transthyretin Amyloid Neuropathy
Longitudinal Quantitative Neuromuscular MRI in Neuropathic Patients
The pathophysiological process common to neuropathies is fatty replacement of muscle tissue, more commonly known as intramuscular fat fraction (F.F).
- Conditions
- Charcot-Marie-Tooth Neuropathy Type 1A, Hereditary Transthyretin Amyloid Neuropathy, Acquired Demyelinating Neuropathy
- Trial name
- IMAGINERV (NCT06845644)
- Testing
- Quantitative neuromuscular MRI, Neuromuscular ultrasound
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
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