Safety and benefit study of EXG001-307 for baby spinal muscular atrophy
Part of Brain & nervous system, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby must have SMA type 1 confirmed by specific SMN1 gene changes and have 2 copies of SMN2
- Your baby must be 180 days old or younger on the day they receive the study medicine
- Your baby’s symptoms must match SMA type 1 (low muscle tone, delayed movement, weak head control, rounded shoulders, very loose joints)
- A parent/guardian must understand the study, agree to it, and sign consent
- You must be willing to follow the usual feeding and breathing-support plans (like tube feeding and noninvasive breathing help) if your study team recommends them
- Your baby must not have certain major breathing problems (for example, needing invasive ventilation/tracheotomy or heavy daily noninvasive support)
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 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.
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 study follows up on patients with type 1 spinal muscular atrophy (SMA) who already received the EXG001-307 gene therapy in a previous trial. The goal is to monitor their long-term health and safety over time.
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.
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.
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.
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