Clinical trials
Spinal Muscular Atrophy clinical trials
Below are recruiting spinal muscular atrophy clinical trials, each written for real people, not researchers. We’re tracking 61 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT03217578Recruiting
Newborn screening for spinal muscular atrophy in Taiwan
This trial offers testing for spinal muscular atrophy (SMA) to newborns. It aims to identify SMA early so families and doctors can act sooner if results suggest the condition.
Kaohsiung CityAges birth–2 weeks - NCT05747261RecruitingPhase 1/Phase 2
Single-dose gene therapy for children with spinal muscular atrophy
This trial tests a one-time gene therapy given through a vein to add a working SMN gene in children with spinal muscular atrophy (SMA). It mainly looks at safety first, and also checks early signs that treatment helps the disease.
MinskAges Up to 8 months - NCT06169046RecruitingPhase 2
Clenbuterol for spinal and bulbar muscular atrophy
This study tests if clenbuterol (a medication that may help strengthen muscles) can improve symptoms in people with spinal and bulbar muscular atrophy (SBMA, also called Kennedy's disease). It compares clenbuterol to a placebo (a dummy pill) over several months.
Padova, PDAges 18–75 - NCT07805278Recruiting
Pregnancy registry for spinal muscular atrophy
This study is a registry that collects information from families who had a pregnancy where the baby was diagnosed with spinal muscular atrophy (SMA) before birth and received treatment during the pregnancy. The goal is to learn more about these cases to improve care and outcomes.
Memphis, TennesseeAges Any age - NCT07554924RecruitingPhase 1/Phase 2
Gene therapy for newly diagnosed spinal muscular atrophy type I infants
This study tests SKG0201, a new gene therapy designed to treat spinal muscular atrophy type I (SMA type I), a rare genetic condition affecting muscle strength. The trial enrolls very young infants diagnosed with SMA type I to see if the treatment is safe and helps improve muscle function.
Beijing, Beijing MunicipalityAges Up to 6 months - NCT07543003Recruiting
Wearable Monitoring for Young Children With Spinal Muscular Atrophy
This study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.
OxfordAges Up to 4 years - NCT06756633Recruiting
Breathing and movement study for neuromuscular disease
This study looks at breathing, chest and belly movement, and exercise ability in people with Duchenne Muscular Dystrophy or Spinal Muscular Atrophy, as well as healthy people. The goal is to compare these functions to help understand how these diseases affect the body.
Ankara, ÇankayaAges 5–15 - NCT06019637Recruiting
Long-term safety study of Zolgensma for spinal muscular atrophy
This study follows the long-term safety of Zolgensma gene therapy in Brazilian children with spinal muscular atrophy (SMA). It helps doctors understand how safe the treatment is over time.
Curitiba, ParanáAges birth–100 years - NCT07478172Recruiting
Electrical muscle stimulation exercise for neuromuscular disease
This study tests whether whole-body electrical muscle stimulation (using small electrodes on the skin) can help adults with certain neuromuscular diseases exercise and improve muscle strength. It may be an option if you can stand for about 15 minutes at a time and have some muscle strength left.
Columbia, MissouriAges 18 years+ - NCT07467187Recruiting
Home Ventilation with Tracheostomy in Denmark (2016–2025)
This study looks at people in Denmark who need a home ventilator through a tracheostomy. It aims to understand their care and outcomes over several years.
CopenhagenAges Any age - NCT07448610RecruitingPhase 4
Real-world safety and effectiveness of gene therapy for spinal muscular atrophy
This study tracks how well the gene therapy ITVISMA works in everyday medical practice for people with spinal muscular atrophy (SMA). It aims to see if the treatment is safe and effective in a real-world setting.
New York, New YorkAges 2–100 - NCT04174157Recruiting
Spinal muscular atrophy patient registry for OAV-101
This is a registry that collects information from people with spinal muscular atrophy (SMA) who have been treated with OAV-101. It helps researchers understand how patients do over time after this treatment.
Phoenix, ArizonaAges Any age - NCT07444450RecruitingPhase 3
Study of salanersen for babies with SMA previously treated with gene therapy
This study tests a new medicine called salanersen in babies who have already received gene therapy for SMA. It aims to see if adding salanersen is safe and effective.
Flower Mound, TexasAges birth–7 months - NCT07444476RecruitingPhase 3
Study of salanersen for spinal muscular atrophy (ages 15-60)
This trial tests a medicine called salanersen (BIIB115) to see how well it improves movement and how safe it is in people with spinal muscular atrophy (SMA). It includes those who have never taken an SMA drug or are currently on risdiplam and willing to switch.
Palo Alto, CaliforniaAges 15–60 - NCT07400198Recruiting
Walking and Bone Health Study for SMA
This study looks at walking and bone health in people with spinal muscular atrophy (SMA). It helps researchers understand how SMA affects your gait (the way you walk) and your bones, which could lead to better treatments.
New York, New YorkAges 8–50 - NCT07332702Recruiting
Spinal muscular atrophy gene study
This study looks at changes in two genes, SMN1 and SMN2, which are linked to spinal muscular atrophy (SMA). It aims to better understand the different versions of these genes in healthy people and those with SMA. Participating involves a blood sample and genetic analysis.
RouenAges 18 years+ - NCT07287982RecruitingPhase 2
ARGX-119 for children with SMA who can walk
This study tests a new medicine called ARGX-119 in children with SMA. It is given through an IV and aims to help kids who can already walk get stronger and stay healthy.
Little Rock, ArkansasAges 5–17 - NCT07286565Recruiting
Remote motor development study for early-diagnosed DMD or SMA
This study remotely tracks motor development in children with Duchenne muscular dystrophy or spinal muscular atrophy who were diagnosed at birth or before symptoms started. It aims to see if early monitoring helps improve long-term care and outcomes.
LiègeAges 4 months+ - NCT07231549Recruiting
SMA study tracking health over time in Spain
This study collects health information from people with spinal muscular atrophy (SMA) over time to learn more about the disease and how to improve care. It may help researchers understand SMA better.
Esplugues de LlobregatAges Any age - NCT07223489Recruiting
Understanding Adult SMA Diagnosis & Treatment Experiences
This trial explores the experiences of adults with spinal muscular atrophy (SMA) in the MedStar Health System, looking at how they are diagnosed and treated, and any differences in care they may face.
Washinton, District of ColumbiaAges 18 years+ - NCT07221669RecruitingPhase 3
Pre-symptomatic salanersen study for babies with SMA
This trial tests if the drug salanersen can help movement and is safe when given to babies diagnosed with spinal muscular atrophy before they show symptoms. It's for very young infants with specific genetic types of SMA.
Chicago, IllinoisAges birth–1 month - NCT04177134Recruiting
French patient registry for spinal muscular atrophy (SMA 5q)
This trial is a patient registry that collects health information from people with spinal muscular atrophy (SMA) caused by changes in the 5q area. It helps researchers understand real-world care and outcomes for these patients across French hospitals.
GarchesAges birth+ - NCT06862596RecruitingPhase 2/Phase 3
Mexiletine for spinal and bulbar muscular atrophy
This trial tests whether mexiletine hydrochloride can help men with spinal and bulbar muscular atrophy (SBMA) by improving muscle strength and function. It may be an option if you have mild to moderate symptoms and meet heart and medication safety checks.
Bunkyō CityAges 18–80 - NCT07070999RecruitingPhase 1/Phase 2
Gene therapy GB221 for infants with SMA type 1
This trial tests a new gene therapy called GB221 for infants with SMA type 1, a rare muscle-weakening disease. It aims to see if this treatment can improve muscle strength and development, and it is for babies who have not had prior gene therapy.
Porto Alegre, Rio Grande do SulAges 2 weeks–1 year
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Spinal Muscular Atrophy trials by city
Studies with a site in or near these metro areas.
Spinal Muscular Atrophy trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for spinal muscular atrophy?
- Yes. Clin2 currently lists 61 recruiting spinal muscular atrophy studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a spinal muscular atrophy trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a spinal muscular atrophy trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.