Clinical trials: Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria.
- Trial name
- NCT07862959
- Testing
- Ulefnersen
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Cerebral Palsy (CP)
Effects of Motor Imagery and Action Observation on Motor Function in Unilateral Cerebral Palsy
The aim of this study is to evaluate the effects of action observation therapy and motor imagery methods, provided in addition to a conventional physiotherapy and rehabilitation program, on gross motor function, upper and lower…
- Trial name
- NCT07612371
- Testing
- motor imagery, Action observation therapy, Conventional physiotherapy group
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people
- Sponsor
- Bahçeşehir University
Muscular Atrophy, Spinal
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy. Up to 21 mL blood will be drawn from eligible patients at a single visit.
- Trial name
- NCT01910168
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis
A Physiotherapy Intervention Study in Patients With Amyotrophic Lateral Sclerosis (ALS)
This study investigates whether an individualized physiotherapy program, tailored to each patient's specific motor deficits, can better support physical function compared with usual care physiotherapy in people with ALS.
- Trial name
- Train ALS (NCT07341984)
- Testing
- Individualized Physiotherapy
- Where
- Switzerland
- Volunteers
- 38 people
- Sponsor
- Charlotte Vogt
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
Motor Neuron Disease
Brain Function in Primary Lateral Sclerosis
This study will examine whether the motor cortex (the part of the brain that controls movement) works properly in patients with primary lateral sclerosis (PLS), a disorder in which voluntary movements are very slow.
- Trial name
- NCT00071435
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia
Phenotype, Genotype and Biomarkers 2
The purpose of this study is to learn more about amyotrophic lateral sclerosis (ALS) and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic…
- Conditions
- Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Trial name
- PGB2 (NCT04875416)
- Where
- 4 sites in United States, South Africa
- Volunteers
- 217 people, healthy volunteers welcome
- Sponsor
- University of Miami
Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis Web Based Patient Care Database: ALSConnection.Org
The purpose is to collect data for ALS research. The data will be used to learn more about the origin of ALS and to improve quality of care for people with ALS.
- Trial name
- NCT00302198
- Testing
- ALS Registry
- Where
- United States
- Volunteers
- 15,000 people
- Sponsor
- Forbes Norris MDA/ALS Research Center
Amyotrophic Lateral Sclerosis
A Registered Cohort Study on Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a kind of motor neuron degeneration disorder without effective therapy. This registered cohort study will provide further insights into the clinical course of ALS, and investigate…
- Trial name
- NCT04008329
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Spinal Muscular Atrophy
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
This is an open label phase I/II clinical trial to assess safety, tolerability and potential effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a potential effect on SMN levels in vitro.
- Trial name
- NCT00374075
- Testing
- Valproic Acid
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Utah
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
Demyelinating Disease, Healthy
Magnetic Stimulation of the Human Nervous System
Transcranial Magnetic Stimulation (TMS) is a non-invasive technique to gather information about brain function. It is very useful when studying the areas of the brain related to motor activity (motor cortex, corticospinal tract…
- Conditions
- Demyelinating Disease, Healthy, Lysosomal Storage Disease, Motor Neuron Disease
- Trial name
- NCT00001780
- Where
- United States
- Volunteers
- 450 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy, Neuromuscular Disorders
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them.
- Trial name
- NCT02235090
- Testing
- Direct current stimulation of cervical spinal cord
- Where
- Ukraine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Complete Locked-in State
Brain Computer Interface Complete locked-in State Communication
Electroencephalography (EEG) and/or near-infrared spectroscopy (NIRS) based Brain computer interface for communication in patients without any means of communication.
- Trial name
- NCT02980380
- Testing
- Non-invasive brain computer interface
- Where
- Germany
- Volunteers
- 15 people
- Sponsor
- University Hospital Tuebingen
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Deglutition Disorder, Motor Neuron Disease
Ultrasound and Videofluoroscopy for Diagnosing Swallowing Disorders
This study will identify clinical signs and symptoms critical for diagnosing swallowing disorders and will characterize swallowing problems in various patient populations, such as patients with Parkinson's disease, stroke…
- Trial name
- NCT00001220
- Where
- United States
- Volunteers
- 750 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
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
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.