Spinal Muscular Atrophy Clinical Trials
84 recruiting trials for Spinal Muscular Atrophy. Eligibility criteria explained in plain English.
Recruiting Trials
Clinical trial data sourced from the ClinicalTrials.gov registry, maintained by the National Library of Medicine. Always consult your doctor before considering any clinical trial.
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...
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...
Spinal Muscular Atrophy Neonatal Screening Program
Spinal muscular atrophy (SMA) is a group of disorders caused by the degeneration of the motor neuron cells of the anterior horn of the spinal cord and, in some subtypes, of the...
Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
The clinical trial titled "Investigation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases" aims to evaluate the respiratory...
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international...
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
There is no cure to arrest or delay SBMA progression. It is estimated that \~1000 individuals are affected by SBMA in Italy at any given time (prevalence: 1.5/100000) with an...
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
Gait and Bone Health in SMA
The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that...
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...
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....
Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the...
Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)
The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that...
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve...
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN...
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar...
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...
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous...
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular...
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord, due to the...
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular...
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge. The proliferation of clinical trials has...
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...
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients...
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with...
HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness
This single-arm pilot study will assess the preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in...
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Effects of Whole-body Electrical Muscle Stimulation Exercise on Spinal Motoneuronal Activation in Older Adults
During this pilot study, the investigators will examine the effects of whole-body electrical muscle stimulation exercise (WB-EMS Exercise) on motoneuronal activation in healthy...
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark. This includes indications...
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With...
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously...
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control...
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle...
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine...
Adult SMA Research and Clinical Hub
Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular...
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...
Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement...
Background: People with cerebral palsy, spina bifida, muscular dystrophy, or spinal cord injury often have muscle weakness and problems controlling how their legs move. This can...
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal...
A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded,...
Evaluation of Sarcopenia and Related Factors in Patients Diagnosed With Psoriatic Arthritis
The purpose of the study is identify the prevalence of sarcopenia and its associated factors in patients with psoriatic arthritis. Furthermore, we aimed to investigate the...
Robot-assisted Training
The goal of this clinical trial is to evaluate the long-term effects of isokinetic rehabilitation training in patients with spinal muscular atrophy (SMA). The main question it...
Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to...
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before...
In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This...
Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)
This study will aim to assess the fertility status of men with Spinal Muscular Atrophy (SMA) not on disease-modifying therapies. Participants will: 1. Complete online...
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular...
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...
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of...
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. Furthermore,...
Natural History of SMA
This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed...
A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With...
This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational...
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants...
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant...
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in...
Osteosarcopenia in Axial Spondyloarthritis
Axial spondyloarthritis is a chronic inflammatory disease affecting the spine, sacroiliac joints, entheses, and sometimes peripheral joints with a close link to HLAB27. Typical...
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or...
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer. The trial evaluates safety, tolerability,...
CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
The goal of this observational study is to set up a longitudinal data collection study to perform a long-term follow-up of SMA patients in a real-world setting, and to standardise...
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy. The main questions it...
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.
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous...
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the...
Showing 50 of 84 trials.Search all Spinal Muscular Atrophy trials
Explore Other Conditions
Frequently Asked Questions
There are currently 84 clinical trials for Spinal Muscular Atrophy, with 84 actively recruiting participants. These include trials across all phases from early-stage Phase 1 to late-stage Phase 3.
To join a clinical trial for Spinal Muscular Atrophy, review the eligibility criteria on the trial detail pages, then talk to your doctor about whether a trial is right for you. Your doctor can help you evaluate the potential benefits and risks.
Phase 3 trials are large-scale studies that test whether a treatment is effective and monitor side effects. There are 6 Phase 3 trials for Spinal Muscular Atrophy, representing treatments closest to potential FDA approval.
Clinical trials follow strict safety protocols overseen by Institutional Review Boards (IRBs) and the FDA. Participants are monitored closely and can withdraw at any time. Always discuss risks and benefits with your healthcare provider before enrolling.
Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice, always talk to your doctor about clinical trial participation.
For this entity, the underlying data on this page comes from the NIH ClinicalTrials.gov registry. The breakdown above is the federal record; the paragraphs below add the per-entity context that makes the headline numbers usable for a real decision rather than just a data lookup.
Every number on this page links back to the NIH ClinicalTrials.gov registry; the methodology page describes the inputs, refresh cadence, and known limitations of the underlying data product.
Practical use of this page is in combination with the comparison and ranking pages elsewhere on the site, which surface the same data for this entity’s peers within active and historical clinical trials. A single-entity reading without peer context can be misleading when an entity is an outlier on one axis but typical on another.