Clinical trials: Neuron Disease, Motor
Spinal Muscular Atrophy (SMA)
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
- Trial name
- SPECTRUM (NCT05335876)
- Testing
- onasemnogene abeparvovec
- Where
- 32 sites in United States, Australia and 17 more
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis, ALS
A Phase IIb, Multi-Center, Multinational, Double-Blind, Placebo-Controlled Study, With an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in Subjects With ALS
69 subjects with ALS will be enrolled in the study and randomized at a 2:1 ratio to receive the study drug or placebo tablets. Randomization sequences will be in random block sizes and stratified for ENCALS risk category [high…
- Trial name
- PARADIGM (NCT05357950)
- Testing
- PrimeC
- Where
- 4 sites in Canada, Israel and 1 more
- Volunteers
- 69 people
- Sponsor
- NeuroSense Therapeutics Ltd.
Neurologic Disorder, Paralysis
COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
The Synchron motor neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex.
- Conditions
- Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- NCT05035823
- Testing
- Motor Neuroprosthesis (MNP)
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Synchron Medical, Inc.
Spinal Muscular Atrophy (SMA)
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor…
- Trial name
- NCT04174157
- Testing
- Prospective observational registry, Zolgensma
- Where
- 99 sites in United States, Greece and 9 more
- Volunteers
- 700 people
- Sponsor
- Novartis Pharmaceuticals
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
Healthy Volunteers
A Study to Assess the Safety, Tolerability and PK of NPT520-34 in Healthy Subjects
To evaluate the PK, safety and tolerability of orally administered NPT520-34 in healthy subjects at single and multiple doses that may be therapeutically relevant.
- Trial name
- NCT03954600
- Testing
- NPT520-34 (125 mg)
- Where
- United States
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Neuropore Therapies Inc.
Amyotrophic Lateral Sclerosis, ALS
Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis
The purpose of this pilot study is to assess the safety and tolerability of multiple doses of MaaT033 in ALS patients and to analyze the gut microbiota composition and evolution before considering a larger randomized controlled…
- Trial name
- IASO (NCT05889572)
- Testing
- MaaT033
- Where
- 2 sites in France
- Volunteers
- 15 people
- Sponsor
- MaaT Pharma
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
Spinal Muscular Atrophy (SMA)
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
- Trial name
- NCT07265232
- Testing
- vesemnogene lantuparvovec
- Where
- Indonesia
- Sponsor
- Lantu Biopharma
Amyotrophic Lateral Sclerosis
Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis
The purpose of this study is to determine the safety and tolerability of intramuscular injections of VM202 at different injection sites in people with amyotrophic lateral sclerosis.
- Trial name
- NCT02039401
- Testing
- VM202
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Helixmith Co., Ltd.
ALS (Amyotrophic Lateral Sclerosis)
Augmented Reality BCI Longitudinal Study for Persons With Late Stage ALS
The goal of this study is refine the usability of a BCI based communication platform. The study will take place in the greater Los Angeles area and will enroll up to 10 participants with late stage ALS.
- Trial name
- NCT06810219
- Testing
- Cognixion ONE
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Cognixion
Muscular Atrophy, Spinal
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP).
- Trial name
- PIERRE-PK (NCT06555419)
- Testing
- Nusinersen, ThecaFlex DRx System
- Where
- 21 sites in United States, France and 5 more
- Volunteers
- 78 people
- Sponsor
- Biogen
Spinal Cord Injury, Safety
Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial
Purpose: This clinical trial is studying an investigational cell therapy called XS228-a lab-made stem cell product designed to help repair damaged nerves in the spinal cord.
- Conditions
- Spinal Cord Injury, Safety, Efficacy, Clinical Trial
- Trial name
- NCT06974968
- Testing
- Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells
- Where
- China
- Volunteers
- 60 people
- Sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
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
Spinal Muscular Atrophy
AveXis Managed Access Program Cohort for Access to AVXS-101
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
- Trial name
- NCT03955679
- Testing
- AVXS-101
- Where
- United States
- Sponsor
- United BioSource, LLC
Amyotrophic Lateral Sclerosis
Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.
The study will evaluate the safety, tolerability and therapeutic effects (preliminary efficacy) of injection of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), as a possible…
- Trial name
- NCT01051882
- Testing
- MSC-NTF cells transplantation (IM), MSC-NTF cells transplantation (IT)
- Where
- Israel
- Volunteers
- 12 people
- Sponsor
- Brainstorm-Cell Therapeutics
Amyotrophic Lateral Sclerosis
CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS
Approximately 21 subjects with amyotrophic lateral sclerosis (ALS) will be randomized (6 to 1) to receive by mouth seven morning doses of CC100 or placebo for 7 days.
- Trial name
- CC100B (NCT03049046)
- Testing
- CC100
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Chemigen, LLC
Amyotrophic Lateral Sclerosis, Cytochrome P-450 Enzyme System
A Study of LY3873862 in Participants With Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS).
The main purpose of this study in participants with Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS) is to test the safety of different doses and to determine the amount of LY3873862 that gets into the bloodstream and how…
- Trial name
- NCT07883590
- Testing
- LY3873862, CYP450 Drug Cocktail
- Where
- 4 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Eli Lilly and Company
Muscular Atrophy, Spinal
Adult Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
- Trial name
- NCT05618379
- Where
- 12 sites in China
- Volunteers
- 200 people
- Sponsor
- Biogen