Gene therapy breakthrough? first human trial launches for rare brain disorder
NCT ID NCT07135050
First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 2 times
Summary
This study tests a new gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition that affects development and breathing. The therapy is given as a single injection into the fluid around the brain. The main goal is to see if it is safe and tolerable, with 12 participants being closely monitored for side effects and changes in health.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- MZ-1866 (a gene therapy delivered via injection into the brain's fluid spaces)
- What this could lead to
- If successful, this could point toward a treatment that controls or reduces the symptoms of Pitt Hopkins Syndrome, a rare genetic disorder.
- What could go wrong
- This is a very early, first-in-human trial with only 12 participants, so safety and effectiveness are not yet known. Gene therapies can have unpredictable side effects, and the approach may not work as hoped.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
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Sheba Medical Center
RECRUITINGTel Litwinsky, Israel
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
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