Gene therapy trial hopes to tame rare brain disorder
NCT ID NCT07135050
First seen Jan 09, 2026 · Last updated Apr 30, 2026 · Updated 22 times
Summary
This early-stage trial tests a one-time gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition affecting development. The study will enroll 12 participants to check if the treatment is safe and tolerable. Researchers will monitor side effects and use developmental assessments to see how participants respond.
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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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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact
Contact Email: •••••@•••••
Conditions
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