Clinical trials
Spinal Muscular Atrophy Type I clinical trials
Below are recruiting spinal muscular atrophy type i clinical trials, each written for real people, not researchers. We’re tracking 23 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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+ - 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 - NCT05824169RecruitingPhase 1/Phase 2
Gene therapy for babies with severe spinal muscular atrophy
This early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
BeijingAges birth–6 months - NCT06363357Recruiting
Shoulder brace study for arm weakness from nerve or muscle diseases
This study tests a fabric shoulder brace that moves like a muscle. It may help people with nerve or muscle diseases improve arm movements like reaching and lifting.
Seoul, Jongno-guAges 10 years+ - NCT06421831RecruitingPhase 1/Phase 2
Gene therapy trial for spinal muscular atrophy type 3
This trial is testing a one-time gene therapy drug for people with SMA type 3. The goal is to see if it can safely improve muscle function and strength.
Beijing, Beijing MunicipalityAges 2 years+ - NCT06839469Recruiting
Walking study for rare muscle and nerve conditions
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
Palo Alto, CaliforniaAges 5 years+ - 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 - NCT06971094RecruitingPhase 3
Gene therapy for type 2 spinal muscular atrophy
This trial tests a new gene therapy called GC101 given as a one-time injection into the spinal fluid for people with type 2 spinal muscular atrophy (SMA). It may help improve motor function.
BeijingAges 2–12 - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Phoenix, ArizonaAges Up to 2 years - 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 - 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 - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - 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 - 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+ - NCT06888661Enrolling by invitationEarly Phase 1
Testing EXG001-307 for spinal muscular atrophy in infants
This trial tests a new medicine called EXG001-307 for babies with spinal muscular atrophy (SMA) types 1 and 2. The goal is to see if it is safe and helps with muscle strength and breathing.
Shanghai, Shanghai MunicipalityAges 1 day–2 years - NCT06772402Enrolling by invitation
Gene therapy for children with type 2 SMA
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
Hangzhou, ZhejiangAges 2–12 - NCT06588803Enrolling by invitation
Long-term follow-up after EXG001-307 for type 1 SMA
This study follows up on patients with type 1 spinal muscular atrophy (SMA) who already received the EXG001-307 gene therapy in a previous trial. The goal is to monitor their long-term health and safety over time.
Shanghai, Shanghai MunicipalityAges 1 month+ - NCT05614531Enrolling by invitationPhase 1/Phase 2
Safety and benefit study of EXG001-307 for baby spinal muscular atrophy
This Phase 1/2 study tests whether EXG001-307 is safe and may improve health in babies with spinal muscular atrophy (SMA) type 1. It mainly looks at safety first, and whether the treatment can help the body work better over time.
Shanghai, Shanghai MunicipalityAges 1 day–6 months - NCT06322654Recruiting
Comparing two types of robotic rehab for spinal muscular atrophy
This study compares two robot-assisted rehabilitation programs to see which one helps children and young adults with spinal muscular atrophy (SMA) improve their movement and function better. You may join if you have SMA (types 1-4), are up to 21 years old, and are already receiving medication for SMA.
PoznanAges Up to 21 years - NCT04010604Recruiting
Study of spinal muscular atrophy in patients and carriers
This is a long-term observation study that follows people affected by spinal muscular atrophy (SMA), people who carry the SMA gene but have no symptoms, and families of people with SMA. It helps researchers better understand SMA and related genetic status, which can support future treatment planning.
Fuzhou, FujianAges 1 week–70 years - NCT06955897Recruiting
Study of Fatigue in SMA Patients on Nusinersen
This study looks at how tired you feel during physical activities while being treated with nusinersen (Spinraza) for spinal muscular atrophy (SMA). The goal is to understand how well the treatment helps with daily energy levels.
New York, New YorkAges 12 years+ - NCT07488338Recruiting
Movement and hand training for children with SMA
This study tests whether a combined program of home-based activities and hand training can help children with spinal muscular atrophy improve their movement and daily function. It is for children who can sit or walk with support and have been on stable medication for at least 6 months.
New York, New YorkAges 5–17
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Common questions
- Are there clinical trials for spinal muscular atrophy type i?
- Yes. Clin2 currently lists 23 recruiting spinal muscular atrophy type i 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 type i 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 type i 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.