Clinical trials
Muscular Atrophy clinical trials
Below are recruiting muscular atrophy clinical trials, each written for real people, not researchers. We’re tracking 68 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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+ - 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+ - 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 - NCT06977269Recruiting
Brain stimulation safety study for spinal muscular atrophy
This trial tests whether a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) is safe and tolerable for people with spinal muscular atrophy (SMA). It may help improve muscle function by activating low motor neurons.
Kharkiv, Kharkiv OblastAges 12 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 - 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 - 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 - 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 - 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+ - 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+ - 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - NCT06555419RecruitingPhase 1
Nusinersen dosing study via flexible spinal catheter
This trial investigates how the drug nusinersen (Spinraza) enters the body when given through a small, flexible tube placed in the lower back (ThecaFlex DRx system). It aims to learn how the drug is processed in adults and children with spinal muscular atrophy, which could help make dosing more convenient.
Phoenix, ArizonaAges 3 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 - 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 - 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 - 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
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Common questions
- Are there clinical trials for muscular atrophy?
- Yes. Clin2 currently lists 68 recruiting 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 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 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.