Clinical trials: Motor Neuron Disease
Amyotrophic Lateral Sclerosis
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.
- Trial name
- NCT05053035
- Testing
- AL001
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Alector Inc.
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
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
A Pilot Study of Inosine in Amyotrophic Lateral Sclerosis (ALS)
This is a single center, open label, 12-week study of inosine treatment. Inosine treatment leads to an increase in the levels of urate (uric acid) in the blood.
- Trial name
- NCT02288091
- Testing
- Inosine
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Massachusetts General Hospital
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
Parkinson Disease, Amyotrophic Lateral Sclerosis
Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with neurodegenerative diseases (Parkinson's…
- Trial name
- PROBE (NCT05542576)
- Testing
- AMDX2011P
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Amydis Inc.
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
ALS (Amyotrophic Lateral Sclerosis)
T-regulatory Cells in ALS
This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same…
- Trial name
- Tregs in ALS (NCT04055623)
- Testing
- Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- The Methodist Hospital Research Institute
ALS, Amyotrophic Lateral Sclerosis
A Trial of Tocilizumab in ALS Subjects
This research study is being done to find out if tocilizumab, also known as Actemra™, can help with Amyotrophic Lateral Sclerosis (ALS).
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Motor Neuron Disease
- Trial name
- TCZALS-001 (NCT02469896)
- Testing
- Tocilizumab
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Barrow Neurological Institute
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
ALS (Amyotrophic Lateral Sclerosis)
ALS Reversals - Lunasin Regimen
This is a 12-month, widely inclusive, largely virtual, single-center, open-label pilot trial utilizing a historical control group.
- Trial name
- NCT02709330
- Testing
- Lunasin Regimen, Historical control
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Richard Bedlack, M.D., Ph.D.
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.
- Trial name
- CL211 (NCT00931944)
- Testing
- KNS-760704
- Where
- 19 sites in United States
- Volunteers
- 74 people
- Sponsor
- Knopp Biosciences
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Amyotrophic Lateral Sclerosis, Respiratory Muscle Weakness
ALS and Airway Clearance (ALSAC) Therapy
Patients will receive one of three respiratory therapy interventions for airway clearance assistance: 1) High frequency chest wall oscillation (HFCWO) and mechanical insufflation/exsufflation (MIE), 2) HFCWO or 3) MIE.
- Trial name
- ALSAC (NCT04947436)
- Testing
- High Frequency Chest Wall Oscillation, Mechanical insufflation/exsufflation
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Muscular Atrophy, Spinal
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
- Trial name
- Rainbowfish (NCT03779334)
- Testing
- Risdiplam
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 26 people
- Sponsor
- Hoffmann-La Roche
Obstructive Sleep Apnea, Amyotrophic Lateral Sclerosis
Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx
This is a pilot study to examine the diagnostic utility of a novel transmembrane surface sensor, and compare signals obtained with the transmembrane sensor to conventional needle EMG signals from healthy volunteers to those with…
- Conditions
- Obstructive Sleep Apnea, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Healthy
- Trial name
- NCT03986671
- Testing
- Transmembrane EMG Oropharynx Probe
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Powell Mansfield Inc.
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
This is a 24-month, Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)
- Trial name
- NCT04579666
- Testing
- Pegcetacoplan (APL-2)
- Where
- 60 sites in United States, Australia and 12 more
- Volunteers
- 249 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
Arimoclomol in Amyotropic Lateral Sclerosis
A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)
- Trial name
- NCT03491462
- Testing
- Arimoclomol
- Where
- 29 sites in United States, Belgium and 10 more
- Volunteers
- 245 people
- Sponsor
- ZevraDenmark
Amyotrophic Lateral Sclerosis
Cervical Electrical Stimulation for ALS
Veterans are at higher risk than non-Veterans of falling ill with amyotrophic lateral sclerosis (ALS). ALS causes degeneration of motor neurons in both the brain and the spinal cord.
- Trial name
- NCT03411863
- Testing
- CES at rest, CES plus active hand or wrist movements
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- VA Office of Research and Development