Clinical trials: Motor Neuron Diseases
Amyotrophic Lateral Sclerosis, ALS
Open Label Extension Study of AMX0035 in Patients With ALS
This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500…
- Trial name
- CENTAUR-OLE (NCT03488524)
- Testing
- AMX0035
- Where
- 24 sites in United States
- Volunteers
- 90 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis
The Effect of RNS60 on ALS Biomarkers
Amyotrophic Lateral Sclerosis (ALS) is a rare lethal neurodegenerative disease involving inflammation. Riluzole, the only drug for ALS, improves median survival by 3 months. This prompts new treatments of ALS.
- Trial name
- RNS60 (NCT03456882)
- Testing
- RNS60
- Where
- 23 sites in United States, Italy
- Volunteers
- 147 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Amyotrophic Lateral Sclerosis
Clinical Trial of Glatiramer Acetate in Amyotrophic Lateral Sclerosis (ALS)
Teva is developing 40 mg/ml Glatiramer Acetate (GA) Injection , administered once daily under the skin, for the treatment of ALS.
- Trial name
- NCT00326625
- Testing
- 40 mg glatiramer acetate
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 366 people
- Sponsor
- Teva Pharmaceutical Industries, Ltd.
Amyotrophic Lateral Sclerosis
Phase 3 Extension Study of Dexpramipexole in ALS
The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.
- Trial name
- ENVISION (NCT01622088)
- Testing
- Dexpramipexole
- Where
- 80 sites in United States, Australia and 9 more
- Volunteers
- 616 people
- Sponsor
- Knopp Biosciences
Amyotrophic Lateral Sclerosis
HEALEY ALS Platform Trial - Regimen G DNL343
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
- Trial name
- NCT05842941
- Testing
- DNL343
- Where
- United States
- Volunteers
- 249 people
- Sponsor
- Merit E. Cudkowicz, MD
Amyotrophic Lateral Sclerosis
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)
This was a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants, 18 to 80…
- Trial name
- HIMALAYA (NCT05237284)
- Testing
- SAR443820
- Where
- 63 sites in United States, Belgium and 11 more
- Volunteers
- 305 people
- Sponsor
- Sanofi
Amyotrophic Lateral Sclerosis
Electrical Impedance Myography as an Outcome Measure in Amyotrophic Lateral Sclerosis Clinical Trials
Trials evaluating new therapies for stopping or slowing the progression of ALS depend critically upon the use of outcome measures to assess whether a potential treatment is effective.
- Trial name
- NCT00620698
- Where
- 8 sites in United States
- Volunteers
- 89 people
- Sponsor
- Beth Israel Deaconess Medical Center
ALS (Amyotrophic Lateral Sclerosis)
Safety and Feasibility of the EyeControl Device
The EyeControl device is an eye movement-based communication device in the form of wearable glasses with connected infrared cam-era that tracks the pupil and translates blinks and movements into commands.This study is aimed to…
- Trial name
- NCT02891629
- Testing
- EyeControl device
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Eyefree Assisting Communication Ltd
Amyotrophic Lateral Sclerosis (ALS), Neuromuscular Diseases
Acute Intermittent Hypoxia and Breathing in Neuromuscular Disease
This project seeks to investigate the effects of a single acute intermittent hypoxia (AIH) session on respiratory and non-respiratory motor function and EMG (electromyography) activity on patients with ALS (amyotrophic lateral…
- Trial name
- AIH-ALS (NCT03645031)
- Testing
- Acute Intermittent Hypoxia
- Where
- 2 sites in United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- University of Florida
Amyotrophic Lateral Sclerosis
Multiple Doses of AT-1501-A201 in Adults With ALS
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40 ligand (CD40L).
- Trial name
- NCT04322149
- Testing
- AT-1501
- Where
- 13 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Anelixis Therapeutics, LLC
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS)
This is a multi-center, 20-week study of inosine treatment. Study Objectives and Endpoints The primary objective of the study is to determine the safety and tolerability of oral administration of inosine (administered daily)…
- Trial name
- SURE-ALS2 (NCT03168711)
- Testing
- Inosine
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Massachusetts General Hospital
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases, Neurodegenerative Diseases
- Trial name
- CENTAUR (NCT03127514)
- Testing
- AMX0035
- Where
- 25 sites in United States
- Volunteers
- 137 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
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
Spinal Muscular Atrophy
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants.
- Trial name
- STOPSMA (NCT00528268)
- Testing
- Sodium phenylbutyrate
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Utah
Amyotrophic Lateral Sclerosis
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT05039268
- Testing
- 3K3A-APC Protein
- Where
- Australia
- Volunteers
- 16 people
- Sponsor
- Macquarie University, Australia
Spinal Muscular Atrophy
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically…
- Trial name
- NURTURE (NCT02386553)
- Testing
- Nusinersen
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 25 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis, Frontal Temporal Dementia
Therapy in Amyotrophic Lateral Sclerosis (TAME)
The purpose of this study is to determine if memantine at up to 20 mg twice a day when used in conjunction with riluzole, can slow down the disease progression of patients with ALS including potentially improving their…
- Trial name
- TAME (NCT02118727)
- Testing
- Memantine
- Where
- 13 sites in United States
- Volunteers
- 89 people
- Sponsor
- University of Kansas Medical Center
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