Clinical trials: PMA - Progressive Muscular Atrophy
Spinal Muscular Atrophy
Antisense Oligonucleotide for Spinal Muscular Atrophy
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.
- Trial name
- NCT05187260
- Testing
- Nusinersen
- Where
- 3 sites in China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis
Digital Assessment of Speech and Fine Motor Control in ALS
This is a single-session, case-control study that incorporates digital tools for assessing speech and motor function in motor neuron disease.
- Conditions
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Trial name
- NCT06315673
- Testing
- Digital Speech and Handwriting Assessment
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Milton S. Hershey Medical Center
ALS
Genomic Translation for Amyotrophic Lateral Sclerosis Care
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms…
- Trial name
- GTAC (NCT02795897)
- Where
- 14 sites in United States, United Kingdom
- Volunteers
- 254 people
- Sponsor
- Columbia University
Neurologic Diseases, Hemiparesis
NuroSleeve Powered Brace & Stimulation System to Restore Arm Function
The purpose of this study is to investigate if a person with weakness or paralysis in one or both arms, can use the NuroSleeve combined powered arm brace (orthosis) and muscle stimulation system to help restore movement in one…
- Conditions
- Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies
- Trial name
- NCT04798378
- Testing
- Neurosleeve
- Where
- 2 sites in United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Thomas Jefferson University
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)
Safety and Tolerability of Anakinra in Combination With Riluzol in Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS) is an adult neurodegenerative disease that is caused by a selective degeneration of the motor nerve cells in the cortex and myelon.
- Trial name
- NCT01277315
- Testing
- Anakinra
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Charite University, Berlin, Germany
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
TRIAL READY (Clinical Trial Readiness)
This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis
- Trial name
- NCT03912987
- Where
- 3 sites in United States
- Volunteers
- 610 people, healthy volunteers welcome
- Sponsor
- University of Miami
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
ALS, PLS
ALS/MND Natural History Study Data Repository
This is a data repository for multi-site multi-protocol clinic-based Natural History Study of ALS and Other Motor Neuron Disorders (MND).
- Conditions
- ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease
- Trial name
- NCT05966038
- Where
- 18 sites in United States, Israel and 1 more
- Volunteers
- 5,000 people
- Sponsor
- Massachusetts General Hospital
Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis
Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS
CAPTURE ALS is a long-term data and biorepository platform that will facilitate future ALS research. CAPTURE ALS will provide the standardized systems and tools necessary to collect, store, and analyze vast amounts of multimodal…
- Conditions
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Degeneration
- Trial name
- CAPTURE ALS (NCT05204017)
- Where
- 4 sites in Canada
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.
- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Trial name
- NCT03067857
- Testing
- Stem Cells
- Volunteers
- 40 people
- Sponsor
- Stem Cells Arabia
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
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
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Amyotrophic Lateral Sclerosis, ALS-Frontotemporal Dementia
Clinical Procedures to Support Research in ALS
The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.
- Conditions
- Amyotrophic Lateral Sclerosis, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
- Trial name
- CAPTURE-ALS (NCT03489278)
- Where
- 11 sites in United States
- Volunteers
- 1,200 people
- Sponsor
- University of Miami
Motor Neuron Disease
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy…
- Trial name
- NCT03196375
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 35 sites in United States
- Volunteers
- 54 people
- Sponsor
- Flex Pharma, Inc.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge.
- Conditions
- Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth, Muscular Dystrophy, Myotonic Dystrophy
- Trial name
- Myokinesis (NCT07321977)
- Testing
- Gait analysis via video capture
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France