Clinical trials: Spinal Muscular Atrophy
Motor Disorders
Patients With ALS and Other Motor Disorders Will be Treated With Umbilical or Placental Mesenchymal Cell Exosome Solution
Patients with ALS and other Motor Discorders will be treated with mesenchymal cell exosome solution.
- Trial name
- NCT07105371
- Testing
- Exosome Secretome
- Where
- 2 sites in United States, Greece
- Volunteers
- 70 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Spinal Muscular Atrophy
A Registered Cohort Study on SMA
Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord.
- Trial name
- NCT04010604
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
ALS (Amyotrophic Lateral Sclerosis), Postpoliomyelitis Syndrome
Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis
Motor neuron disease (MND) or ALS is a nervous system disease. ALS leads to a loss of movement ability that eventually leads to death. At the moment, there is no known treatment for ALS.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), Postpoliomyelitis Syndrome, Spinal Muscular Atrophy
- Trial name
- MotorMarker (NCT05663008)
- Testing
- 128 electrode electroencephalography (EEG), Bipolar surface electromyography (sEMG), High-density electromyography (HD-EMG)
- Where
- Ireland
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University of Dublin, Trinity College
Post-polio Syndrome
Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% Dual Inactivation and Filtration (DIF) in Participants With Post-polio Syndrome
This was a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in participants with post-polio syndrome (PPS).
- Trial name
- FORCE (NCT02176863)
- Testing
- Flebogamma® 5% DIF
- Where
- 21 sites in United States, Canada and 7 more
- Volunteers
- 191 people
- Sponsor
- Instituto Grifols, S.A.
Muscular Atrophy, Spinal
Pediatric Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q…
- Trial name
- NCT05042921
- Where
- 25 sites in China
- Volunteers
- 600 people
- Sponsor
- Biogen
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
Muscular Atrophy, Spinal
A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- ONWARD (NCT04729907)
- Testing
- Nusinersen
- Where
- 39 sites in United States, Brazil and 14 more
- Volunteers
- 115 people
- Sponsor
- Biogen
ALS, SCI - Spinal Cord Injury
ECoG BMI for Motor and Speech Control
Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.
- Conditions
- ALS, SCI - Spinal Cord Injury, Stroke, Multiple Sclerosis
- Trial name
- BRAVO (NCT03698149)
- Testing
- PMT/Blackrock Combination Device
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Karunesh Ganguly
Cystinosis, Cystinosis, Nephropathic
Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy
Newborn screening in Germany is a voluntary program. Cystinosis and spinal muscular atrophy (SMA) are rare autosomal recessive diseases. They are inherited in an autosomal recessive manner, i.e.
- Conditions
- Cystinosis, Cystinosis, Nephropathic, Spinal Muscular Atrophy
- Trial name
- GENESIS1 (NCT06027385)
- Testing
- molecular-based screening
- Where
- 4 sites in Germany
- Volunteers
- 300,000 people
- Sponsor
- Cystinose Stiftung
Natural History of Type 1 Spinal Muscular Atrophy (SMA)
Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
Spinal muscular atrophy (SMA) is an autosomal recessive disorder characterized by degeneration of motor neurons in the spinal cord and caused by mutations of the survival motor neuron 1 (SMN1) gene.
- Trial name
- NCT02466529
- Volunteers
- 111 people
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
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
Post Polio Syndrome, PPS
Efficacy and Safety of Xepol (Human Immunoglobulin) in Subjects With Post-Polio Syndrome (PPS)
The primary objective was to assess the effect of Xepol compared to placebo on physical health and on muscle strength in subjects with post-polio syndrome.The secondary objective was to assess the effect of Xepol compared to…
- Trial name
- NCT00160082
- Testing
- Xepol
- Where
- 4 sites in Sweden
- Volunteers
- 124 people
- Sponsor
- Calliditas Therapeutics AB
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
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy
Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women
Spinal muscular atrophy (SMA) prenatal carrier screening is recommended by American College of Medical Genetics (ACMG) and American College of Obstetrics and Gynecology (ACOG).
- Trial name
- NCT04859179
- Where
- Thailand
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Spinal Muscular Atrophy (SMA)
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
- Trial name
- SPECTRUM (NCT05335876)
- Testing
- onasemnogene abeparvovec
- Where
- 32 sites in United States, Australia and 17 more
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal, Ultrasound
Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen
Intrathecal administration of Nusinersen, an antisense oligonucleotide capable of increasing Survival Motor Neuron protein production, has been tested in Spinal Muscular Atrophy (SMA) to improve motor function and survival.
- Trial name
- NCT04674618
- Testing
- US-assisted nusinersen administration
- Where
- Italy
- Volunteers
- 58 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Cerebrovascular Accident, Post-polio Syndrome
Nomad P-KAFO Study
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that has affected their ability to walk.
- Conditions
- Cerebrovascular Accident, Post-polio Syndrome, Spinal Cord Injuries, Multiple Sclerosis
- Trial name
- NCT05644522
- Testing
- Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Shirley Ryan AbilityLab
Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1)
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD).
- Conditions
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- NCT04907162
- Testing
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- LMU Klinikum