Clinical trials: Neuron Disease, Motor
Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD)
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy…
- Trial name
- Active-NBS BE (NCT07286565)
- Testing
- MAIJU, Syde, Questionnaires
- Where
- Belgium
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Diseases, Spinal Muscular Atrophy
New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
This study aims to refine the capability of MSOT to characterise muscle tissue and to determine non-invasive, quantitative biomarkers for the disease assessment in patients with spinal muscular atrophy (SMA) using Multispectral…
- Trial name
- MSOT_SMA (NCT04115475)
- Testing
- Multispectral Optoacoustic Tomography (MSOT)
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Amyotrophic Lateral Sclerosis
A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS
This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 μg sNN0029/day in patients with ALS who previously participated in study sNN0029-003
- Trial name
- NCT02269436
- Testing
- sNN0029 infusion solution
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 11 people
- Sponsor
- Newron Sweden AB
Healthy, Amyotrophic Lateral Sclerosis
A P300 Brain Computer Interface to Operate Power Wheelchair Tilt
The investigators want to develop a brain-computer interface (BCI) that will eventually allow people who are completely paralyzed to independently control the tilt feature on their power wheelchairs.
- Trial name
- NCT01123148
- Testing
- Using a BCI to control wheelchair tilt
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
The purpose of this study is to learn about rates of patient-reported disease progression in patients with motor neuron diseases (amyotrophic lateral sclerosis, progressive muscular atrophy, primary lateral sclerosis, hereditary…
- Conditions
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02852278
- Testing
- Web-based Survey
- Volunteers
- 410 people
- Sponsor
- University of South Florida
Amyotrophic Lateral Sclerosis (ALS)
Expanded Controlled Study of Safety and Efficacy of MCI-186 in Patients With Amyotrophic Lateral Sclerosis (ALS)
This is a long-term, double-blind, placebo-controlled study of MCI-186 to treat ALS. This study is the long-term extension of Study NCT00330681; Study NCT00330681 is a Phase 3, randomized, double-blind, placebo control, parallel…
- Trial name
- NCT00424463
- Testing
- MCI-186
- Where
- Japan
- Volunteers
- 181 people
- Sponsor
- Shionogi
Amyotrophic Lateral Sclerosis
Muscle Training of Patients With Amyotrophic Lateral Sclerosis (ALS)
Patients with neuropathic diseases are experiencing increasing muscle weakness, loss of muscle strength and functional abilities during their illness.
- Trial name
- ALS-project (NCT01504009)
- Testing
- Muscle training
- Where
- Denmark
- Volunteers
- 6 people
- Sponsor
- University of Southern Denmark
Amyotrophic Lateral Sclerosis
Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.
- Trial name
- NCT03836716
- Testing
- Arimoclomol
- Where
- 23 sites in United States, Belgium and 9 more
- Volunteers
- 120 people
- Sponsor
- ZevraDenmark
Spinal Muscular Atrophy
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.
- Trial name
- NCT06178393
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Dystrophies, Myopathies
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases.
- Conditions
- Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
- Trial name
- NCT06666816
- Testing
- inapplicable
- Where
- 2 sites in Italy
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Development and Test of a Headset for BCI Until Obtaining an Efficient and Comfortable System That Can be Used in Daily Practice by ALS People
"Brain-computer interfaces (BCIs) are computer-based systems that acquire brain signals, analyze them, and translate them into commands that are relayed to an output device to carry out a desired action.
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Spinal Cord Diseases, Alternative Communication Devices
- Trial name
- TECH-ICOPA (NCT04380649)
- Testing
- New headset prototype
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Parkinson Disease, Amyotrophic Lateral Sclerosis (ALS)
Discovery and Validation of Protein Structural Complexes in Circulating Biofluids As Novel Biomarkers for Early Diagnosis, Prognosis and Therapeutic Management of Patients Affected by Neurodegenerative Disorders
Neurodegenerative disorders (NDDs), such as Parkinson¿s disease (PD), Alzheimer¿s disease (AD), Frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS) are characterized by aggregation and intracellular accumulation…
- Conditions
- Parkinson Disease, Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), Alzheimer's Disease (AD)
- Trial name
- NCT06803784
- Testing
- Genetic: whole genome sequencing, whole exome sequencing
- Where
- Italy
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Neuromed IRCCS
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Spinal Muscular Atrophy (SMA), Neuromuscular Disease
Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
This study aims to investigate the relationship between ultrasonographic muscle thickness measurement and echogenicity assessment with functional scales in children with spinal muscular atrophy (SMA).
- Trial name
- NCT06599606
- Testing
- Ultrasonographic Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Istanbul University - Cerrahpasa
Amyotrophic Lateral Sclerosis, Multiple Sclerosis
Human Amniotic Mesenchymal Cell Secretome for Neurodegeneration and Neuroinflammation
Neurodegenerative diseases are debilitating conditions characterized by chronic inflammation, leading to dysfunction of both the non-neuronal cellular components of the central nervous system and peripheral blood immune cells.
- Trial name
- CONTRASTOME (NCT06551649)
- Testing
- Venous blood draw and skin biopsy
- Where
- 2 sites in Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Amyotrophic Lateral Sclerosis
Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis
The purpose of this study was to evaluate the safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis as compared to Placebo.
- Trial name
- REViVALS-1A (NCT04632225)
- Testing
- Engensis
- Where
- 5 sites in United States, South Korea
- Volunteers
- 18 people
- Sponsor
- Helixmith Co., Ltd.
Amyotrophic Lateral Sclerosis
A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.
- Trial name
- NCT06215755
- Testing
- VRG50635
- Where
- 7 sites in Belgium, Canada and 2 more
- Volunteers
- 54 people
- Sponsor
- Verge Genomics
Spinal Muscular Atrophy (SMA)
Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder caused by mutations in the SMN1 gene, leading to motor neuron degeneration and progressive muscle atrophy.
- Trial name
- MOSAiC-SMA (NCT07883655)
- Where
- 3 sites in Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
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