Clinical trials: Motor Neurone Disease
Amyotrophic Lateral Sclerosis (ALS)
Study of COYA 302 for the Treatment of ALS
The ALSTARS trial will be conducted across 20-25 sites in the US and Canada, and will evaluate the safety and efficacy of an investigational treatment called COYA 302 for adults with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- ALSTARS (NCT07161999)
- Testing
- COYA 302
- Where
- 25 sites in United States, Canada
- Volunteers
- 120 people
- Sponsor
- Coya Therapeutics
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
ALS - Amyotrophic Lateral Sclerosis
Controlling Amyotrophic Lateral Sclerosis Motor Neuron Excitability Study
Following completion of the ALS Early Feasibility Study of the MyoRegulator® device for treatment of ALS (NCT06165172), the CALM study will further assess the feasibility of the MyoRegulator® device to treat ALS in an expanded…
- Trial name
- CALM (NCT06649955)
- Testing
- Multi-site direct current stimulation (DCS)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- PathMaker Neurosystems Inc.
Amyotrophic Lateral Sclerosis
A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis
This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of…
- Trial name
- NCT05006352
- Testing
- DNL343
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 29 people
- Sponsor
- Denali Therapeutics Inc.
Amyotrophic Lateral Sclerosis (ALS)
Extension Study of Participants From SPG302-ALS-001
This study will evaluate the long-term safety and efficacy of participants enrolled in SPG302-ALS-101 with Amyotrophic Lateral Sclerosis (ALS)
- Trial name
- NCT06903286
- Testing
- SPG302
- Where
- 3 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Spinogenix
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
Dexpramipexole Japanese PK Study
This is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects.
- Trial name
- NCT01424163
- Testing
- Single dose reduced, Single dose standard, Multiple Dose
- Where
- United Kingdom
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Knopp Biosciences
Neuromuscular Disorders, Neurodevelopmental Disorders
Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural Environments
Neurodevelopmental disorders often lead to abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as an inability to stand and walk.
- Trial name
- NCT06765070
- Testing
- EXPLORER
- Where
- 6 sites in Spain
- Volunteers
- 15 people
- Sponsor
- MarsiBionics
Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Clinical Characteristics, Natural History, Health Care Measures, and Genetic Screening in Patients With ALS
Patients with sporadic ALS (sALS), which refers to those without a family history of ALS, are typically not subjected to genetic investigations as part of their standard care. Therefore, their mutation status is often unknown.
- Trial name
- ID-ALS (NCT05852405)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis &Amp; Other Neuromuscular Disorders
Amyotrophic Lateral Sclerosis: A Multicenter Retrospective Observational Registry
This retrospective observational study will analyze de-identified clinical data from patients with amyotrophic lateral sclerosis (ALS) collected at multiple centers over 7 years.
- Trial name
- NCT07143656
- Testing
- Imaging and Clinical Reports
- Where
- Poland
- Volunteers
- 36 people
- Sponsor
- Biocells Medical
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Spinal Muscular Atrophy
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
This will be a Phase I, 2-part, open-label, non-randomized study to investigate the safety, tolerability, and pharmacokinetics (PK) of a multiple-dosing regimen of risdiplam (Part 1) and the effect of risdiplam on the PK of…
- Trial name
- NCT03988907
- Testing
- Risdiplam, Midazolam
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis (ALS)
Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS
This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn™) in…
- Trial name
- NurOwn (NCT02017912)
- Testing
- Nurown MSC-NTF cells
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Brainstorm-Cell Therapeutics
Amyotrophic Lateral Sclerosis
Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of…
- Trial name
- EPI-589 (NCT02460679)
- Testing
- EPI-589
- Where
- 3 sites in United States
- Volunteers
- 19 people
- Sponsor
- PTC Therapeutics
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
Amyotrophic Lateral Sclerosis, Healthy Volunteer
A Study to Assess the Effect of Erythromycin on the Test Medicine (SAR443820) When Given Orally as Tablets to Healthy Adult Male and Female Participants (Part A); and the Effect of Itraconazole on the Test Medicine (SAR443820) When Given Orally as Capsules to Healthy Adult Male Participants (Part B)
This is a Phase 1, cross-over, 2-part study for pharmacokinetic (PK) assessment of SAR443820 when co-administered with cytochrome P450 3A4 (CYP3A4) inhibitors (erythromycin ethyl succinate (EES) in Part A and possibly…
- Trial name
- NCT05797753
- Testing
- SAR443820, Erythromycin ethyl succinate, Itraconazole
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Sanofi
Amyotrophic Lateral Sclerosis
Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
- Trial name
- Phoenix (NCT05021536)
- Testing
- AMX0035
- Where
- 69 sites in United States, Belgium and 10 more
- Volunteers
- 664 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis (ALS)
Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS)
The primary objective of this study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip once a day in patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24…
- Trial name
- NCT00330681
- Testing
- MCI-186
- Volunteers
- 206 people
- Sponsor
- Shionogi
Amyotrophic Lateral Sclerosis
CARE Canadian ALS Riluzole Evaluation
Evaluate the efficacy of riluzole 50-mg bid defined by comparing the percentage of riluzole-treated subjects who experienced death, permanently assisted ventilation (PAV) or tracheostomy, to a group of recent historical controls…
- Trial name
- NCT00542412
- Testing
- Riluzole
- Where
- Canada
- Volunteers
- 414 people
- Sponsor
- Sanofi
Amyotrophic Lateral Sclerosis (ALS)
A Study for Patients Who Completed VITALITY-ALS (CY 4031)
The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).
- Trial name
- VIGOR-ALS (NCT02936635)
- Testing
- tirasemtiv
- Where
- 67 sites in United States, Belgium and 9 more
- Volunteers
- 280 people
- Sponsor
- Cytokinetics