Clinical trials: Motor Neuron Disease
Amyotrophic Lateral Sclerosis (ALS)
Remotely Supervised tDCS for Slowing ALS Disease Progression
Most ALS care is centered on patient support and symptom management, making rehabilitation an integral aspect for slowing disease progression, prolonging life span, and increasing quality of life.
- Trial name
- NCT04866771
- Testing
- Transcranial Direct Current Stimulation (tDCS)
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Illinois Chicago
Spinal Muscular Atrophy
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age.
- Trial name
- NCT00227266
- Testing
- Valproic Acid and Levocarnitine
- Where
- 6 sites in United States, Canada
- Volunteers
- 94 people
- Sponsor
- University of Utah
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
Amyotrophic Lateral Sclerosis
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS). The ALSpire Study consists of two parts: Part 1: 6-month placebo-controlled study.
- Trial name
- ALSpire (NCT04494256)
- Testing
- BIIB105
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 99 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Clinical Trial of Ezogabine (Retigabine) in ALS Subjects
This study evaluates the effect of retigabine (600 mg/day, 900 mg/day, or placebo) on motor neuron activity in people with Amyotrophic Lateral Sclerosis (ALS). The total study duration is approximately 14 weeks.
- Trial name
- NCT02450552
- Testing
- Ezogabine
- Where
- 12 sites in United States
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Brian Wainger
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
The Use of Airway Clearance Devices in ALS
The investigator is examining the use of one airway clearance medical device compared to the use of two airway clearance medical devices together in patients with amyotrophic lateral sclerosis (ALS).
- Trial name
- NCT02682030
- Testing
- High Frequency Chest Compression Device (HFCC), Cough Assist
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Cedars-Sinai Medical Center
Amyotrophic Lateral Sclerosis
Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
The purposes of the study are to determine the energy balance and evaluate the nutritional status of patients with ALS, and to investigate the use of NIPPV as respiratory support to treat patients with ALS.
- Trial name
- NCT00116558
- Testing
- Early NIPPV, Standard NIPPV
- Where
- 12 sites in United States
- Volunteers
- 153 people
- Sponsor
- Edward Kasaraskis
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis ALS
ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments
GenieUs developed an analysis platform that will be tested to separate study participants with ALS into four categories based on blood work.
- Trial name
- NCT06429059
- Testing
- Astaxanthin, Protandim, Melatonin
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Duke University
Muscular Atrophy, Spinal
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- RESPOND (NCT04488133)
- Testing
- Nusinersen
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 46 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
C9orf72 Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia
Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD
The primary objective is to assess the safety and tolerability of Metformin in subjects with C9orf72 amyotrophic lateral sclerosis administered for 24 weeks.
- Trial name
- NCT04220021
- Testing
- Metformin
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Florida
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, Lou Gehrig's Disease
Clinical Trial of High Dose CoQ10 in ALS
The purpose of this study is to determine the efficacy and preferred dose of CoQ10 in individuals with ALS for a possible future phase III study.
- Trial name
- NCT00243932
- Testing
- coenzyme Q10
- Where
- 19 sites in United States
- Volunteers
- 185 people
- Sponsor
- Columbia University
Amyotrophic Lateral Sclerosis
Study of Safety and Proof of the Mechanism of BLZ945 in ALS Patients
It was an open label study to evaluate safety, tolerability and brain microglia response in participants with Amyotrophic Lateral Sclerosis (ALS) following multiple doses of BLZ945.
- Trial name
- NCT04066244
- Testing
- BLZ945
- Where
- 4 sites in United States, Finland and 1 more
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
Gilenya in Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to determine whether Gilenya, also known as fingolimod, is safe and tolerable in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT01786174
- Testing
- Gilenya
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Massachusetts General Hospital
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
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.
ALS (Amyotrophic Lateral Sclerosis)
A Clinical Demonstration of EEG Brain-computer Interface for ALS Patients
The goal of this VA demonstration project is to show that the Brain-computer interface (BCI) technology is a clinically practical and important new communication and control option that can improve the lives of veterans with…
- Trial name
- ALS (NCT00786032)
- Testing
- BCI Device
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- US Department of Veterans Affairs