Clinical trials: Neuron Disease, Motor
Spinal Cord Injuries, Amyotrophic Lateral Sclerosis
Diaphragmatic Pacer Placement: Anesthetic Management (DP)
The diaphragmatic pacemaker (DP) has proven its utility in replacing mechanical ventilation (MV) in patients with chronic spinal cord injury (SCI) and Amyotrophic Lateral Sclerosis (ALS), by improving the patients quality of life…
- Conditions
- Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Respiratory Paralysis
- Trial name
- DP (NCT02942953)
- Testing
- Diaphragmatic Pacer
- Volunteers
- 16 people
- Sponsor
- Institut Guttmann
Amyotrophic Lateral Sclerosis
The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS)
There is currently no effective treatment in ALS. Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis.
- Trial name
- NCT04140136
- Testing
- Tocotrienols
- Where
- Malaysia
- Volunteers
- 20 people
- Sponsor
- University of Malaya
ALS, Amyotrophic Lateral Sclerosis
A Study to Explore the Role of Gut Flora in ALS
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records regarding ALS
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- Trial name
- NCT04150809
- Testing
- No intervention
- Where
- United States
- Sponsor
- ProgenaBiome
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Hereditary Spastic Paraplegia, Neurodegenerative Diseases
Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)
The purpose of the HSP Sequencing Initiative is to better understand the role of genetics in hereditary spastic paraplegia (HSP) and related disorders.
- Conditions
- Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle
- Trial name
- NCT05354622
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Muscular Atrophy, Spinal
Newborn Screening for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare, treatable, genetic disease that typically occurs in infancy and early childhood. SMA progressively, and irreversibly, destroys motor neurons in the brainstem and spinal cord, which control…
- Trial name
- SMA-NBS (NCT05481164)
- Where
- 9 sites in United Kingdom
- Volunteers
- 33,568 people
- Sponsor
- University of Oxford
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
Spinal Muscular Atrophy (SMA) is an autosomal recessive disease of motor neurons. In the early 1980s, Werdnig from Vienna University and Hoffman from Heidelberg University described this disorder.
- Trial name
- NCT02855112
- Testing
- Adipose derived mesenchymal stem cell
- Where
- Iran
- Volunteers
- 10 people
- Sponsor
- Tehran University of Medical Sciences
Amyotrophic Lateral Sclerosis
Geometrical Facial Deformation According to Posture in Amyotrophic Lateral Sclerosis
The measurement of the facial deformation according to the body posture will be conducted by 3D scanning for each posture after marking the landmarks on the face.
- Trial name
- NCT04798183
- Testing
- 3-dimensional scan
- Where
- South Korea
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Pusan National University Yangsan Hospital
Spinal Muscular Atrophy, Developmental Delay
Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder causing progressive muscle weakness and atrophy.
- Trial name
- NCT07547189
- Testing
- Online Early Intervention Program for Infants with SMA Type 1
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Medipol University
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Study of ALS Reversals 2: Genetic Analyses
The purpose of this study is to try to understand why reversals of amyotrophic lateral sclerosis (ALS) and primary muscular atrophy (PMA) take place.
- Trial name
- StAR2 (NCT03464903)
- Where
- United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Duke University
Spinal Muscular Atrophy (SMA)
Adult SMA Research and Clinical Hub
Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA).
- Trial name
- REACH (NCT06978985)
- Testing
- Risdiplam, Nusinersen Injectable Product
- Where
- 18 sites in United Kingdom
- Volunteers
- 600 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
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
Measuring Levels of SMN in Blood Samples of SMA Patients
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN.
- Trial name
- NCT00061607
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and…
- Trial name
- NCT00568802
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is caused by the homozygous loss of the Survival Motor Neuron (SMN) 1 gene, which leads to degeneration of spinal alpha-motor neurons and muscle atrophy.
- Trial name
- MAP_THE_SMA-01 (NCT05769465)
- Testing
- disease modifying treatments
- Where
- Italy
- Volunteers
- 247 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Muscular Atrophy, Spinal
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through…
- Trial name
- STELLAR-1 (NCT07221669)
- Testing
- Salanersen
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Implementation of FEES in Spinal Muscle Atrophy
The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores.
- Trial name
- DYS-SMA (NCT04773470)
- Testing
- Flexible Endoscopic Evaluation of Swallowing
- Where
- Germany
- Volunteers
- 79 people
- Sponsor
- University of Giessen