Gene therapy trial for FOXG1 syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy called FRF-001 for people with FOXG1 syndrome. It aims to see if the treatment is safe and can help improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed FOXG1 gene mutation that is likely causing your symptoms.
- You or your parent/guardian must be part of the FOXG1 Research Foundation's natural history study.
- You must be able to attend all study visits and follow the study plan.
- You cannot have another genetic condition or a FOXG1 gene duplication or deletion outside the FOXG1 gene.
- You must not have had gene therapy, cell therapy, or any experimental treatment for FOXG1 syndrome before.
- You must be able to safely have an MRI, a lumbar puncture (spinal tap), and the medicines needed for these procedures.
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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