Condition

Spinal Muscular Atrophy (SMA) clinical trials and research

• 15Clinical trials
• 2New in 30 days
• 20Countries

Newest Spinal Muscular Atrophy (SMA) clinical trials

All recruiting trials (15)

• Newborn SMA Screening Service Evaluation

  This project tests newborn SMA screening to see if it works well and is acceptable to families in the UK.

  Recruiting at 2 sites University of Oxford · London and 2 other locations · NCT07888868

• Biomarker search in SMA using advanced multi-omics

  Researchers are trying to see if new biomarkers can help diagnose and monitor spinal muscular atrophy in patients and newborns.

  Recruiting at 3 sites Istituto Giannina Gaslini · Genova and 2 other locations · NCT07883655

• Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA

  This study tests whether a portable knee-training robot with a compliant vs. rigid stiffness setting improves knee strength in children with spinal muscular atrophy, using each child’s other leg as a comparison.

  Recruiting Peking University Third Hospital · Beijing · NCT07822711

• Wearable monitoring of motor development in babies and young children with spinal muscular atrophy detected at birth

  This project will try wearing small motion sensors at home to see if they can track how babies and young children diagnosed with spinal muscular atrophy at birth move and develop, compared with healthy children.

  Recruiting University of Oxford · Oxford · NCT07543003

• HABIT-ILE plus Functional Strength Training for Children with SMA

  This study will try an intensive HABIT-ILE program combined with functional strength training for children aged 5–17 with SMA who are on stable disease-modifying therapy to see if it improves motor skills.

  Recruiting Teachers College, Columbia University · New York, New York · NCT07488338

• Long-read genetic analysis for Spinal Muscular Atrophy (LOREASI)

  This project tries a long-read DNA technique on adults with known SMN1 copy-number patterns to find hidden '2+0' carrier genotypes that standard tests miss.

  Recruiting University Hospital, Rouen · Rouen · NCT07332702

• Portable markerless video system to measure walking in adults with neuromuscular conditions or healthy volunteers.

  This project will test a portable video device that measures walking patterns to see if it can track gait changes in adults with neuromuscular diseases and in healthy volunteers.

  Recruiting Institut de Myologie, France · Paris · NCT07321977

• ARGX-119 IV for ambulant children aged 5 to under 18 with spinal muscular atrophy

  This trial will test whether IV ARGX-119, given with standard SMA therapies, is safe and can reduce muscle weakness and tiredness in children aged 5 to under 18.

  Recruiting at 18 sites Phase 2 argenx · Little Rock, Arkansas and 27 other locations · NCT07287982

• Remote monitoring of motor development in newborn-identified children with SMA or DMD

  This project will try wearable sensors at home to see if they can track motor development in infants and toddlers diagnosed with spinal muscular atrophy or Duchenne muscular dystrophy through newborn or family screening.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège · NCT07286565

• Diagnostic journey and treatment disparities for adults with spinal muscular atrophy at MedStar Health

  This project will see how adults with spinal muscular atrophy in the MedStar Health system experienced diagnosis, access to approved SMA therapies, and barriers to care.

  Recruiting Medstar Health Research Institute · Washinton, District of Columbia · NCT07223489

• Data collection for adults with Spinal Muscular Atrophy

  This study is collecting information from adults with Spinal Muscular Atrophy to see how current treatments are working and to improve care for patients.

  Recruiting at 18 sites Newcastle-upon-Tyne Hospitals NHS Trust · Birmingham and 17 other locations · NCT06978985

• Using magnetic stimulation to improve muscle function in spinal muscular atrophy

  This study is testing whether a type of brain stimulation can help improve muscle function in people with spinal muscular atrophy without them having to do any physical exercise.

  Recruiting Charitable Foundation Children with Spinal Muscular Atrophy · Kharkiv, Kharkiv Oblast · NCT06977269

• Observing walking patterns in patients with neuromuscular diseases

  This study is testing how walking patterns change in people with neuromuscular diseases and whether wearable sensors can help track their daily activity and fatigue levels.

  Recruiting IRCCS Eugenio Medea · Bosisio Parini, Lecco and 1 other locations · NCT06666816

• Investigating a new gene therapy for children with spinal muscular atrophy

  This study is testing a new gene therapy called ANB-004 to see if it can help young children with spinal muscular atrophy feel better and stay safe.

  Recruiting at 7 sites Phase 1/2 Biocad · Minsk and 6 other locations · NCT05747261

• Registry for patients with spinal muscular atrophy

  This study is tracking patients with spinal muscular atrophy over 15 years to see how well the new treatment OAV-101 (Zolgensma) works and how the disease progresses in real life.

  Recruiting at 72 sites Novartis · Phoenix, Arizona and 98 other locations · NCT04174157

Related topics

Quick answers

How many Spinal Muscular Atrophy (SMA) clinical trials are recruiting right now?

15, as of November 12, 2026. 2 of them were first registered in the last 30 days. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 20 countries. The most sites are in United States (4 trials), United Kingdom (4 trials), Italy (2 trials), Russia (2 trials), France (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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