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 63 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - NCT06147414Recruiting
Blood test for genetic disorders in pregnancy
This study tests a safer blood test to check for certain genetic disorders during pregnancy, instead of an invasive procedure like amniocentesis. It's for pregnant women who are at risk of passing on a known genetic condition.
ParisAges 18 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 - 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 - 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+ - 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 - 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+ - NCT06532474Recruiting
Muscle function study in SMA patients on SMN therapies
This study looks at how muscles work in children and young adults with spinal muscular atrophy (SMA) who are taking or starting SMN-directed therapies (like Evrysdi, Spinraza, or Zolgensma). It aims to understand the body's response to these treatments, which could help improve future care.
Memphis, TennesseeAges 5–20 - NCT06555419RecruitingPhase 1
How Nusinersen works with ThecaFlex DRx system
This study looks at how a medicine called Nusinersen (Spinraza) works in the body when given through a new device called ThecaFlex DRx. It is for people with spinal muscular atrophy who are already on a steady dose of Nusinersen and are part of the main PIERRE study.
Orange, CaliforniaAges 3 years+ - NCT06562283Recruiting
Reproducibility of a fatigue test for spinal muscular atrophy
This study checks how consistent a fatigue test is for people with spinal muscular atrophy (SMA). It helps doctors better understand fatigue in SMA and may improve future care.
Saint-Etienne, FranceAges 6 years+ - NCT06666816Recruiting
Watching changes in walking for muscle and nerve diseases
This study watches how people with certain muscle or nerve diseases walk over time. It may help doctors understand changes in walking patterns and is open to adults who can walk and have a genetic diagnosis of a neuromuscular condition.
Bosisio Parini, LeccoAges 18–75
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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 63 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.