Gene therapy GB221 for infants with SMA type 1
Part of Brain & nervous system, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby must have a confirmed genetic diagnosis of SMA type 1 (two bad copies of the SMN1 gene) and no more than 3 copies of the backup SMN2 gene.
- If your baby is already showing symptoms of SMA, they must be between 2 weeks and under 12 months old, with symptoms starting before 6 months.
- If your baby is not yet showing symptoms, they must be between 2 weeks and under 5 months old, with no more than 2 copies of SMN2.
- Your baby cannot have had any prior gene therapy, stem cell or organ transplant.
- Your baby cannot have a current active infection (like HIV, hepatitis B or C) or have used immune-weakening drugs in the last 3 months.
- Your baby cannot require a breathing tube or have low oxygen levels 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.
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