Clinical trials: Charcot-Marie-Tooth Disease
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
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
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, Type IA
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A
- Trial name
- NCT05333406
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- ENCell
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.
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, 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
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
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.
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
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.
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.
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 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
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
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
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
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
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