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
- 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+ - NCT06396325Recruiting
Active video game trial for arm strength in kids with SMA
This trial tests whether playing active video games (exergaming) with your arms can help children and teens with spinal muscular atrophy improve their arm strength and function. If you join, you'll play specially designed games for a period of time, and researchers will see if it makes a difference.
Calgary, AlbertaAges 8–18 - NCT06321965Recruiting
Study of SMA in Children on SMN-Boosting Therapies
This study is looking for children and teens with spinal muscular atrophy (SMA) who are already on a treatment like Spinraza, Evrysdi, or Zolgensma. The goal is to understand different forms of SMA and how the treatment affects them over time.
Bron, RhoneAges birth–16 years - 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 - 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 - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Little Rock, ArkansasAges 3 months–2 years - NCT05866419Recruiting
Study of a port and catheter system for spinal muscular atrophy
This study tests a new device called the ThecaFlex DRx System, which is a small port and catheter placed under the skin to deliver Spinraza directly into spinal fluid—instead of needing repeated spinal taps. The device aims to make receiving medication easier and safer for people with SMA.
Phoenix, ArizonaAges 3 years+ - NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Little Rock, ArkansasAges 3 months–2 years - 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 - NCT05789758Recruiting
Study of Spinraza in pregnancy for 5q spinal muscular atrophy
This study looks at how Spinraza (nusinersen) taken during or before pregnancy affects people with 5q spinal muscular atrophy (SMA) and the health of their babies. It mainly fits those who were pregnant and received Spinraza around the time of conception or during pregnancy.
Palo Alto, CaliforniaAges 18–54 - 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 - NCT04944940Recruiting
Study tests blood, scans, and genes in SBMA and healthy men
This study looks for early “biomarkers” (measurable signs) in the body using blood tests, imaging scans, and sometimes other procedures in men with genetically confirmed spinal and bulbar muscular atrophy (SBMA). The results may help future drug studies by showing better ways to track disease changes over time.
Bethesda, MarylandAges 18–120 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - 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+ - NCT07321977Recruiting
Walking test with a portable device for neuromuscular disease
This study tests a portable device that analyzes walking without markers. It aims to see if it can help assess walking in people with neuromuscular diseases and healthy volunteers, which could lead to simpler check-ups.
ParisAges 18–65 - 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 - NCT07521930Recruiting
Brain implant to restore communication and movement control
This trial tests a surgically implanted brain device designed to help people with severe paralysis or movement disorders regain the ability to communicate and control their limbs. The device reads signals from your brain and translates them into commands that could help you interact with computers or assistive technology.
Baltimore, MarylandAges 18–80 - NCT07072676Enrolling by invitation
Can a walker help prevent falls in neuromuscular disease?
This study tests if using assistive walking devices, after a short training period, can lower the risk of falls in people with neuromuscular diseases. It aims to help you stay steady and safe on your feet.
Bad FeilnbachAges 65–65
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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.