Clinical trials: CMT (Charcot Marie Tooth Disease)
Multiple Sclerosis
A 12 Week Randomized Open Label Parallel Group Multicenter Study to Evaluate Bioequivalence of 20 mg Subcutaneous Ofatumumab Injected by Pre-filled Syringe or Autoinjector in Adult RMS Patients
The primary purpose of this study is to demonstrate pharmacokinetic bioequivalence of ofatumumab injected by Pre-filled Syringe (PFS) versus Auto-Injector (AI) devices and thereby establish a bridge between the ongoing Phase 3…
- Trial name
- NCT03560739
- Testing
- ofatumumab with PRF, ofatumumab with AI
- Where
- 41 sites in United States, Austria and 7 more
- Volunteers
- 284 people
- Sponsor
- Novartis Pharmaceuticals
Entrapment Neuropathy
Study Evaluating Treatment of Forefoot Pain Related to Nerve Entrapment Using the Cryo-Touch III Device
A proof of concept study to evaluate the feasibility of safe and effective treatment through optimization of the Cryo-Touch III Device for temporary relief of pain.
- Trial name
- NCT01753778
- Testing
- Cryo-Touch III Device
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pacira Pharmaceuticals, Inc
Bladder Outlet Obstruction, Multiple Sclerosis
GentleCath™ for Men Intermittent Catheter With FeelClean™ Technology
Male self catherterisng observational study.
- Conditions
- Bladder Outlet Obstruction, Multiple Sclerosis, Cauda Equina Syndrome, Enlarged Prostate With Lower Urinary Tract Symptoms
- Trial name
- NCT05470751
- Testing
- Intermittent self-catheterisation
- Where
- 16 sites in United States, France and 1 more
- Volunteers
- 72 people
- Sponsor
- ConvaTec Inc.
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory…
- Trial name
- CMT4J (NCT03810508)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Neurogene Inc.
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 Type 4J
Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)
First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.
- Trial name
- NCT07447557
- Testing
- ELP-02
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Elpida Therapeutics SPC
Amyotrophic Lateral Sclerosis
Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
This registry study aims to collect data on the provision of assistive devices, medicines, and other healthcare measures, such as ventilation therapy and nutrition support, in patients with Amyotrophic lateral sclerosis (ALS)…
- Trial name
- NCT05852418
- Where
- 16 sites in Germany
- Volunteers
- 25,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Charcot-Marie-Tooth Disease
The Impact of Charcot-Marie-Tooth Disease in the Real World
An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of…
- Trial name
- NCT03782883
- Where
- United Kingdom
- Volunteers
- 3,321 people
- Sponsor
- Vitaccess Ltd
Scapholunate Advanced Collapse (SLAC), Scapholunate Crystalline Advanced Collapse (SCAC)
The KinematX Midcarpal Total Wrist Arthroplasty Registry
The goal of this observational study is to learn about functional and patient reported outcomes in patient undergoing total wrist replacement with the KinematX total wrist replacement study.
- Conditions
- Scapholunate Advanced Collapse (SLAC), Scapholunate Crystalline Advanced Collapse (SCAC), Scaphoid, Trapezium, and Trapezoid Advanced Collapse (STTAC), Carpal Tunnel Syndrome (CTS)
- Trial name
- NCT05719935
- Testing
- KinematX total wrist arthroplasty system
- Where
- 4 sites in United States
- Volunteers
- 50 people
- Sponsor
- Extremity Medical
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
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
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
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
Charcot Marie Tooth Disease, Type 1
The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease.
A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1)
- Trial name
- NCT05947578
- Testing
- CLZ-2002
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- Cellatoz Therapeutics, Inc
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
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
Peripheral Neuropathy, Neuro-Degenerative Disease
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J.
- Conditions
- Peripheral Neuropathy, Neuro-Degenerative Disease, Neuromuscular Diseases
- Trial name
- CMT4J (NCT06151600)
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Elpida Therapeutics SPC
Charcot-Marie-Tooth Disease
Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls
The primary aim of this study is to discover whether patients with CMT type 1 and 2 have neuromuscular junction transmission deficits.
- Trial name
- ESTABLISH (NCT04980807)
- Where
- 2 sites in United States, Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- NMD Pharma A/S
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