Clinical trials: Spinal Muscular Atrophy
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy, SMA
UK SMA Patient Registry
Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults.
- Trial name
- NCT04292574
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 800 people
- Sponsor
- Newcastle University
Spinal Muscular Atrophy, Spine Deformity
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
- Conditions
- Spinal Muscular Atrophy, Spine Deformity, Scoliosis
- Trial name
- PIERRE (NCT05866419)
- Testing
- ThecaFlex DRx System
- Where
- 21 sites in United States, Germany and 3 more
- Volunteers
- 90 people
- Sponsor
- Alcyone Therapeutics, Inc
Spinal and Bulbar Muscular Atrophy, Kennedys Disease
Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)
Background: SBMA is an inherited chronic disease. It affects males in mid to late adulthood. It causes slowly progressive weakness of muscles and hand tremors. Researchers want to learn more about the effects of SBMA.
- Conditions
- Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
- Trial name
- NCT04944940
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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
Muscular Atrophy, Spinal
Adult 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) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
- Trial name
- NCT05618379
- Where
- 12 sites in China
- Volunteers
- 200 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Equine-Assisted Physiotherapy for Spinal Muscular Atrophy
This prospective interventional study evaluates the immediate and sustained effects of intensive equine-assisted physiotherapy in children with spinal muscular atrophy (SMA).
- Trial name
- HIPOSMA (NCT05341453)
- Testing
- Individual physiotherapy (SMA-SOC-N), Equine-Assisted Physiotherapy Based on Neuroproprioceptive Facilitation and Inhibition, Therapeutic grooming
- Where
- Czechia
- Volunteers
- 21 people
- Sponsor
- Charles University, Czech Republic
Spinal Muscular Atrophy
Spinal Muscular Atrophy and School Transitions
A qualitative questionnaire-based study to explore the experiences of children and young people with Spinal Muscular Atrophy (SMA) and their families regarding school transition and educational participation.
- Trial name
- NCT07720518
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
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
Spinal Muscular Atrophy
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA.
- Trial name
- NCT06532474
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- St. Jude Children's Research 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
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
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 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
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Phenotype, Genotype & Biomarkers in ALS and Related Disorders
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02327845
- Where
- 15 sites in United States, Germany and 1 more
- Volunteers
- 708 people, healthy volunteers welcome
- Sponsor
- University of Miami
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
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
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
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma