Single-dose gene therapy for children with spinal muscular atrophy
Part of Brain & nervous system clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child must be under 240 days old when the parent/guardian signs consent
- Child must have confirmed 5q-SMA with specific SMN1 and SMN2 gene results (2 or 3 SMN2 copies)
- If symptoms are present, they must have started very early (by 180 days after birth)
- Parent/guardian must be able to understand study information and follow study procedures
- Child must not need long daily breathing support (16+ hours/day) or a tracheostomy
- Child must have low enough pre-existing AAV9 antibodies (tested at screening)
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 new gene therapy called Vesemnogene Lantuparvovec for people with spinal muscular atrophy (SMA). It is designed for patients in low- and middle-income countries who cannot access or did not respond to other treatments. The goal is to see if the therapy is safe and effective in real-world conditions.
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.
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.
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.
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.
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