Clinical trials: Motor Neuron Diseases
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Spinal Muscular Atrophy
Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients
The "SMOB" project intends to contribute to fill the gap with reliable and operational outcome measures for type III and IV SMA.
- Trial name
- SMOB (NCT04690998)
- Testing
- Blood Samples, MRI
- Where
- 11 sites in France
- Volunteers
- 104 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
ALS - Amyotrophic Lateral Sclerosis
Controlling Amyotrophic Lateral Sclerosis Motor Neuron Excitability Study
Following completion of the ALS Early Feasibility Study of the MyoRegulator® device for treatment of ALS (NCT06165172), the CALM study will further assess the feasibility of the MyoRegulator® device to treat ALS in an expanded…
- Trial name
- CALM (NCT06649955)
- Testing
- Multi-site direct current stimulation (DCS)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- PathMaker Neurosystems Inc.
Spinal Muscular Atrophy, Duchenne Muscular Dystrophy
Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices
The addition of SMA and DMD muscle diseases to newborn screening and premarital carrier screening has been controversial. In this study, researchers aim to measure the awareness level of SMA and DMD muscle diseases of individuals…
- Conditions
- Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Healthy Individuals
- Trial name
- NCT05110885
- Testing
- Questionnaire
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Bahçeşehir University
Cerebral Cortex Dysfunction
Brain Function in Primary Lateral Sclerosis and Amyotrophic Lateral Sclerosis
This study will test whether primary lateral sclerosis (PLS) and amyotrophic lateral sclerosis (ALS) affect parts of the brain responsible for thinking, planning, memory and emotion.
- Trial name
- NCT00334516
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
PARKINSON DISEASE (Disorder), Parkinson's Disease (PD)
Effects of Vagal Nerve Stimulation on Leg Muscle Activity and Posture in Parkinson's Disease
This study is designed to better understand the mechanisms contributing to impaired activation of leg muscles in people with Parkinson's disease (PD) and to test if stimulation of a nerve at the neck can improve muscle…
- Trial name
- NCT07226284
- Testing
- Vagal nerve stimulation, No intervention
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Amyotrophic Lateral Sclerosis (ALS)
Hypercaloric PEG Nutrition in ALS to Sustain Energy Homeostasis
Weight loss is a known negative prognostic factor in amyotrophic lateral sclerosis (ALS). Over the last years, various interventional studies targeting the energy deficit in ALS yielded promising results; however,it is still…
- Trial name
- PEGASUS (NCT06877143)
- Testing
- 100% of individual calory requirement, 120% of individual calory requirement
- Where
- Germany
- Volunteers
- 76 people
- Sponsor
- University of Ulm
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis
Effects of Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis
Specific aims: Aim 1. To determine the incidence of hypoventilation in Amyotrophic Lateral Sclerosis (ALS) patients. Aim 2. To identify the clinical characteristics and risk factors associated . Aim 3.
- Trial name
- NCT00958048
- Testing
- non-invasive ventilation
- Where
- Taiwan
- Volunteers
- 70 people
- Sponsor
- National Taiwan University Hospital
Pure Autonomic Failure, Amyotrophic Lateral Sclerosis
Anti-Cholinergic Receptors Antibodies, Autonomic Profile and Dysautonomia Symptoms in PAF, ALS and POTS (DISAUT-AB)
Anti alfa-3 and alfa-7 ganglionic cholinergic receptors (anti-AChRs) antibodies (Abs) plasma removal by plasmapheresis (1,2) acutely improved dysautonomia symptoms in case reports with Pure Autonomic Failure (PAF) (3).
- Conditions
- Pure Autonomic Failure, Amyotrophic Lateral Sclerosis, Postural Orthostatic Tachycardia Syndrome, Anti-Cholinergic Receptors Antibodies
- Trial name
- DISAUT-AB (NCT04875949)
- Testing
- Immunoabsorption/plasmapheresis procedure
- Where
- Italy
- Volunteers
- 75 people
- Sponsor
- Istituto Clinico Humanitas
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Amyotrophic Lateral Sclerosis
Efficacy and Tolerability of Beta Hydroxybutyrate Ester in Patients With Amyotrophic Lateral Sclerosis (ALS)
Weight loss is a known negative prognostic factor in amyotrophic lateral sclerosis (ALS). One potential mechanism of weight loss in ALS is a disturbance of the mitochondrial complex I which causes an energy deficit in affected…
- Trial name
- KETO-ALS (NCT04820478)
- Testing
- Beta Hydroxybutyrate Ester (KetoneAid KE4)
- Where
- Germany
- Volunteers
- 81 people
- Sponsor
- University of Ulm
Locked-in Syndrome, Neuromuscular Diseases
Investigation on the Cortical Communication System
The goal of this clinical trial is to demonstrate communication through a brain implant in people in locked-in state, i.e. people with severe paralysis and communication problems.
- Conditions
- Locked-in Syndrome, Neuromuscular Diseases, Brainstem Stroke, Traumatic Brain Injury
- Trial name
- CortiCom (NCT06207591)
- Testing
- ECoG (electrocorticography) sensing
- Where
- Netherlands
- Volunteers
- 2 people
- Sponsor
- UMC Utrecht
Neurodegenerative Diseases, Parkinson Disease
Neurodegenerative Diseases Progression Markers (MARKERS-NDD)
MARKERS-NDD is a prospective, observational, longitudinal study, which aims to collect data from patients affected by neurodegenerative diseases (NDD) followed longitudinally for routine examinations performed as part of normal…
- Conditions
- Neurodegenerative Diseases, Parkinson Disease, Synucleinopathies, Lewy Body Disease
- Trial name
- MARKERS-NDD (NCT06596746)
- Testing
- Observation
- Where
- Italy
- Volunteers
- 600 people
- Sponsor
- Casa di Cura San Raffaele Cassino
Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid
Compassionate Use of Ceftriaxone in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative…
- Conditions
- Amyotrophic Lateral Sclerosis, Cerebrospinal Fluid, Neurodegenerative Disease, Motor Neuron Disease
- Trial name
- NCT00718393
- Volunteers
- 1 people
- Sponsor
- Drexel University College of Medicine
Amyotrophic Lateral Sclerosis (ALS), Motor Neurone Disease (MND)
Metabolism and Nutrition in MND
Motor neurone disease (MND) is a progressive neurological condition that can affect movement, speech, swallowing and breathing. Weight loss and malnutrition are common in people with MND and may be caused by factors including…
- Trial name
- MET-MND (NCT07851714)
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Amyotrophic Lateral Sclerosis
An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS
This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.
- Trial name
- NCT05299658
- Testing
- CNMAu8
- Where
- 2 sites in Australia
- Volunteers
- 40 people
- Sponsor
- Clene Nanomedicine
Amyotrophic Lateral Sclerosis (ALS), Emotional Distress
Resiliency Intervention for Patients With ALS and Their Care-Partners
The purpose of this study is to pilot a resiliency and coping intervention for persons recently diagnosed with Amyotrophic Lateral Sclerosis (ALS) and their primary informal caregivers.
- Trial name
- RT-ALS (NCT06968468)
- Testing
- Resilient Together ALS
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Spinal Muscular Atrophy Type I, Scoliosis
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation.
- Conditions
- Spinal Muscular Atrophy Type I, Scoliosis, Chest Deformities, Spinal Orthosis
- Trial name
- NCT05878418
- Testing
- Thoracolumbosacral Spinal Orthosis (TLSO), Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Medipol University
Alzheimer's Disease, Amyotrophic Lateral Sclerosis
Laughter Therapy Effects on Mood, Stress and Self-efficacy in People With Neurological Diseases.
This is a prospective investigation of the effects of Laughter therapy (LT) on perceived stress, self-efficacy, mood and other wellness measures in people with the following neurological conditions: Alzheimer's disease…
- Conditions
- Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Brain Injury, Huntington's Disease
- Trial name
- NCT02750982
- Testing
- Laughter Therapy
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Brown, Theodore R., M.D., MPH
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