Clinical trials: Motor Neuron Diseases
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
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
Amyotrophic Lateral Sclerosis (ALS)
Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral…
- Trial name
- NCT04577404
- Testing
- MT-1186
- Where
- 44 sites in United States, Canada and 4 more
- Volunteers
- 124 people
- Sponsor
- Shionogi
Amyotrophic Lateral Sclerosis
Dexpramipexole SAD/MAD Study
This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
- Trial name
- NCT01449578
- Testing
- Dexpramipexole
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Knopp Biosciences
Amyotrophic Lateral Sclerosis
RAPA-501 Therapy of ALS Expanded Access Protocol
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).
- Trial name
- NCT06169176
- Testing
- RAPA-501 Autologous T stem cells
- Where
- 10 sites in United States
- Sponsor
- Rapa Therapeutics LLC
Amyotrophic Lateral Sclerosis
Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT01730716
- Testing
- Human spinal cord stem cell implantation
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neuralstem Inc.
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
Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
- Trial name
- NCT02988297
- Testing
- RNS60
- Sponsor
- Revalesio Corporation
Amyotrophic Lateral Sclerosis
Phase 3 Study of Dexpramipexole in ALS
The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- EMPOWER (NCT01281189)
- Testing
- Dexpramipexole
- Where
- 82 sites in United States, Australia and 9 more
- Volunteers
- 942 people
- Sponsor
- Knopp Biosciences
Amyotrophic Lateral Sclerosis
Creation of a Bank of Fibroblast From Patients With Amyotrophic Lateral Sclerosis: Pilot Study
Patients with ALS will be included in the reference center for ALS in hospital La Pitié Salpêtrière, Paris. The study proposes to investigate the pathophysiology of ALS by setting up a fibroblast bank for studying molecular…
- Trial name
- ALSCELL (NCT01639391)
- Testing
- skin biopsy
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Institut Pasteur
Amyotrophic Lateral Sclerosis
Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)
This was a 2-part study of dexpramipexole in patients with ALS. Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels…
- Trial name
- CL201 (NCT00647296)
- Testing
- Dexpramipexole 50 mg/day, Dexpramipexole 150 mg/day, Dexpramipexole 300 mg/day
- Where
- 21 sites in United States
- Volunteers
- 194 people
- Sponsor
- Knopp Biosciences
Amyotrophic Lateral Sclerosis
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration…
- Trial name
- ALS (NCT07287397)
- Testing
- VTx-002, Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 36 people
- Sponsor
- Vector Y Therapeutics
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
A Long-term Extension Study of TCH346 and Placebo Administered Once Daily in Patients With Amyotrophic Lateral Sclerosis(ALS)
This is a study to evaluate the safety and clinical effects of 4 oral doses of TCH346 compared to placebo in patients with mild or mild to moderate stages of ALS.
- Trial name
- NCT00230074
- Testing
- TCH346
- Where
- United States
- Volunteers
- 350 people
- Sponsor
- Novartis
Amyotrophic Lateral Sclerosis (ALS)
A Follow up Study to Protocol 101/2 - Continued Treatment by IPL344 IV
This is a prospective, open-label, follow up study to protocol 101/2 - continued treatment by IPL344 IV administered once a day in up to 15 participants with ALS.
- Trial name
- NCT03755167
- Testing
- IPL344
- Where
- 2 sites in Israel
- Volunteers
- 15 people
- Sponsor
- Immunity Pharma Ltd.
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Tazbentetol in ALS Participants
The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.
- Trial name
- NCT07325591
- Testing
- Tazbentetol
- Volunteers
- 430 people
- Sponsor
- Spinogenix
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.
ALS (Amyotrophic Lateral Sclerosis)
Intermediate-size Patient Population Expanded Access Protocol
The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial.
- Trial name
- NCT07088159
- Testing
- Tazbentetol
- Where
- 16 sites in United States
- Sponsor
- Spinogenix
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