ARGX-119 for children with SMA who can walk
Part of Brain & nervous system clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 5 and 17 years old
- You have a confirmed genetic diagnosis of spinal muscular atrophy (SMA)
- You are currently taking nusinersen or risdiplam, or have had gene therapy (Zolgensma) in the past
- You can walk at least 50 meters (about half a football field) without a walker or cane
- You do not need a breathing machine during the day (using one only at night or naps is okay)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
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.
This study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.
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.
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.
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.
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