Gene therapy study for Pitt Hopkins syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Pitt Hopkins syndrome with a TCF4 gene mutation found by a genetic test from a certified lab.
- Your symptoms should match Pitt Hopkins syndrome, in the doctor's opinion.
- You must be willing to share your past medical records.
- You cannot have a very large deletion in the TCF4 gene (over 12 million base pairs).
- You cannot have another major genetic condition or brain problem that would make the injection unsafe, like a shunt or signs of high pressure in the brain.
- You must be able to have an MRI and receive anesthesia for the injection.
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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