Clinical trials: Charcot Marie Tooth Disease (CMT)
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
Nephrectomy
HYbrid RObotic Surgery in Urology
The HYbrid RObotic Surgery in urology (HYROS) study is an early feasibility clinical investigation of a new medical device under the commercial name of Bitrack System.
- Trial name
- HYROS (NCT05864040)
- Testing
- Bitrack System-assisted laparoscopic radical/simple nephrectomy
- Where
- Spain
- Volunteers
- 3 people
- Sponsor
- Rob Surgical Systems S.L.
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
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Study of Electrical Impedance Myography (EIM) in ALS
This trial is studying Electrical Impedance Myography (EIM) for measuring muscle health. The trial is studying people with Amyotrophic Lateral Sclerosis (ALS), other neuromuscular diseases, and healthy volunteers to see if the…
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
- Trial name
- NCT02011204
- Testing
- Electrical impedance myography (EIM)
- Where
- 6 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Skulpt, 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
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
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.
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
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
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, 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
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
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.
Relapsing Remitting Multiple Sclerosis (RRMS)
ELIOS - Investigational Biomarkers to Track Disease Modification in Active RRMS
The exploratory ELIOS study aims to assess the value of novel investigational Eye Movement Biomarkers (EMBs) in tracking disease-related changes among a real-world cohort of Canadian patients with active RRMS, within the context…
- Trial name
- ELIOS (NCT06733922)
- Testing
- ETNATM-ProgMS
- Where
- 14 sites in Canada
- Volunteers
- 224 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Charcot-Marie-Tooth Disease
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to…
- Trial name
- NCT03124459
- Testing
- ACE-083
- Where
- 16 sites in United States
- Volunteers
- 63 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX…
- Trial name
- NCT03943290
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 62 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH