Clinical trials: Motor Neurone Disease
Amyotrophic Lateral Sclerosis, ALS
Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS.
- Trial name
- NCT04259255
- Testing
- Edaravone (Radicava®/Radicava ORS®)
- Where
- 20 sites in United States, Canada
- Volunteers
- 74 people
- Sponsor
- Shionogi
Amyotrophic Lateral Sclerosis
Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)
About 213 people with ALS will participate in this study. There will be locations in North and South America. During the first part, participants will be randomly assigned to a group (like by flipping a coin).
- Trial name
- NCT03068754
- Testing
- Acthar
- Where
- 69 sites in United States, Argentina and 5 more
- Volunteers
- 143 people
- Sponsor
- Mallinckrodt
Amyotrophic Lateral Sclerosis
Brain-Computer Interface Communication Device for People With ALS
This study compares a wearable brain-computer interface (BCI) communication device with the eye-tracking communication devices that people with ALS already use.
- Trial name
- NCT07871071
- Testing
- Cognixion ONE AR-BCI configuration, Cognixion ONE screen-based BCI configuration, Existing eye-gaze speech generating device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Cognixion
Healthy
A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN
The purpose of this study is to assess the safety, tolerability and pharmacokinetics (i.e. how study drug is taken up by the body) of TBN in healthy participants.
- Trial name
- NCT04667013
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Guangzhou Magpie Pharmaceuticals Co., Ltd.
Spinal Muscular Atrophy
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
Spinal Cord Injury, Safety
Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial
Purpose: This clinical trial is studying an investigational cell therapy called XS228-a lab-made stem cell product designed to help repair damaged nerves in the spinal cord.
- Conditions
- Spinal Cord Injury, Safety, Efficacy, Clinical Trial
- Trial name
- NCT06974968
- Testing
- Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells
- Where
- China
- Volunteers
- 60 people
- Sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Amyotrophic Lateral Sclerosis (ALS)
Noninvasive Assessment of Neuromuscular Disease Using Electrical Impedance Myography
The purpose of this protocol is to test a new Electrical Impedance Myography (EIM) device and study its reliability and ability to differentiate ALS patients from healthy controls.
- Trial name
- NCT01803386
- Testing
- EIM Measurements
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Amyotrophic Lateral Sclerosis, ALS
Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis
This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).
- Trial name
- NCT05163886
- Testing
- LAM-002A
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- IRulya Therapeutics Inc.
Amyotrophic Lateral Sclerosis (ALS)
A Long-Term Study in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to investigate the safety and efficacy of long-term E0302 administration in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT00445172
- Testing
- E0302 (mecobalamin)
- Where
- 42 sites in Japan
- Volunteers
- 135 people
- Sponsor
- Eisai Co., Ltd.
Healthy Volunteers
A Study of VRG50635 in Healthy Volunteers
The primary objectives of this study are to investigate the safety and tolerability of VRG50635 and to determine how VRG50635 is absorbed by the body.
- Trial name
- NCT06286475
- Testing
- VRG50635
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Verge Genomics
ALS (Amyotrophic Lateral Sclerosis)
Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel…
- Trial name
- TCIM/ELA (NCT02286011)
- Testing
- MNC (Mononuclear cells), Saline
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- Red de Terapia Celular
Alzheimer's Disease, Multiple Sclerosis
A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery
The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies.
- Conditions
- Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease
- Trial name
- NCT01592552
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Amyotrophic Lateral Sclerosis (ALS)
Safety, Tolerability, and Pharmacokinetics of FHND1002 Granules in Healthy Adults
The goal of this clinical trial is to evaluate the safety, tolerability, and pharmacokinetic (PK) profile of FHND1002 granules in healthy adult volunteers.
- Trial name
- FHND1002-Ⅰ (NCT06782958)
- Testing
- 50mg FHND1002, 100mg FHND1002, 150 mg FHND1002
- Where
- China
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.
Amyotrophic Lateral Sclerosis
A Study to Evaluate, Safety, Tolerability, and PK of AP-101 in Participants With ALS
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
- Trial name
- NCT05039099
- Testing
- AP-101
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 73 people
- Sponsor
- AL-S Pharma
Amyotrophic Lateral Sclerosis
A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study was to assess the effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.
- Trial name
- FORTITUDE-ALS (NCT03160898)
- Testing
- Reldesemtiv
- Where
- 64 sites in United States, Australia and 4 more
- Volunteers
- 458 people
- Sponsor
- Cytokinetics
Amyotrophic Lateral Sclerosis
To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers
Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts. After single dose administration, followed by an independent Data and Safety Monitoring Board (DSMB) meeting…
- Trial name
- NCT03651349
- Testing
- HK-001
- Where
- Taiwan
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Everfront Biotech Co., Ltd.
Asthma, Heart Failure
The General Breathing Record Study
This study will test the use of a new handheld device (called the N-Tidal C), that measures a person's tidal breath carbon dioxide, in diagnosing the cause of someone's breathlessness.
- Conditions
- Asthma, Heart Failure, Pneumonia, Motor Neuron Disease
- Trial name
- GBRS (NCT03356288)
- Where
- United Kingdom
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- TidalSense
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
Amyotrophic Lateral Sclerosis
A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)
The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.
- Trial name
- NCT04516096
- Testing
- AMX0035
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis, ALS
Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)
This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive…
- Conditions
- Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease, Lou Gehrig's Disease
- Trial name
- NCT03537807
- Testing
- Riluzole
- Sponsor
- Biohaven Pharmaceuticals, Inc.