Gene therapy breakthrough? first human trial launches for rare brain disorder
NCT ID NCT07135050
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PITT HOPKINS SYNDROME are added.
By submitting, you agree to our Terms of use
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Rush University Medical Center
NOT_YET_RECRUITINGChicago, Illinois, 60612, United States
Contact
Contact Email: •••••@•••••
-
Sheba Medical Center
RECRUITINGTel Litwinsky, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact
Contact Email: •••••@•••••