Gene therapy study for babies with fused skull bones
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This study tests a new treatment called ASO (antisense oligonucleotide) therapy on tissue samples from babies with syndromic craniosynostosis—a condition where skull bones fuse too early due to specific genetic changes. Researchers hope this approach could eventually help prevent or reduce the severity of this condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is between 0 and 5 years old
- Your child has been diagnosed with syndromic craniosynostosis caused by specific genetic mutations (in FGFR1, FGFR2, FGFR3, TWIST1, TCF12, EFNB1, ERF, MSX2, or ALX4)
- Your child has already had or will have skull surgery, and tissue samples from that surgery can be used for research
- You and your child's other parent (or legal guardians) agree in writing to participate
- Your child's genetic diagnosis must be confirmed and clearly identified—not 'possibly' or 'unknown'
- There must be enough good-quality tissue available from surgery for the researchers to work with
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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