Brain-Injected gene therapy trial offers hope for rare genetic disorder
NCT ID NCT07135050
Summary
This early-stage study is testing whether a new gene therapy called MZ-1866 is safe for people with Pitt Hopkins Syndrome, a rare genetic disorder that affects brain development. Twelve children and adults will receive a single injection of the therapy directly into the fluid around their brain. Researchers will monitor participants' health and track developmental changes over time to see if the treatment shows promise.
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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
Explore the condition pages connected to this study.