New gene therapy hopes to change lives of children with rare brain disorder
NCT ID NCT07135050
First seen Jan 09, 2026 · Last updated May 24, 2026 · Updated 25 times
Summary
This early-stage trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins Syndrome, a rare genetic condition that affects development. The study will include 12 children and adults who will receive a single injection of the therapy into the fluid around the brain. Researchers will closely monitor safety and any changes in health or development over time.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rush University Medical Center
NOT_YET_RECRUITINGChicago, Illinois, 60612, United States
Contact
Contact Email: •••••@•••••
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Sheba Medical Center
RECRUITINGTel Litwinsky, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact
Contact Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.