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New treatment option for Pitt Hopkins Syndrome
Official title Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
ClinicalTrials.gov ID: NCT07135050
What this study is testing
What is MZ-1866?
MZ-1866 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for pitt hopkins syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The goal of this clinical trial is to learn if MZ-1866 is a safe and tolerable treatment for children and adults with Pitt Hopkins Syndrome. To evaluate the safety of MZ-1866, the following will be evaluated: frequency and severity of adverse events physical exam, laboratory results and electrocardiogram findings Participants will: receive a single dose of MZ-1866 by intracerebroventricular injection be seen by the study physician and site staff periodically to assess changes to their health status be periodically evaluated using neurodevelopmental tools Caregivers will: be interviewed periodically about the health status and development of the participant keep diaries and complete periodic questionnaires regarding participant symptoms
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 25
You may be able to join if
- The participant has a TCF4 gene mutation confirmed as "pathogenic" or "likely pathogenic" by whole exome sequencing, whole genome sequencing, gene...
- Clinical phenotype consistent with Pitt Hopkins Syndrome, in the opinion of the investigator
- The participant, or the participant's parent or legal guardian, is willing to provide access to prior medical records for the collection of...
You likely can't join if
- A deletion that includes the TCF4 gene that is over 12 Mbp in size
- Another genetic mutation or clinical comorbidity not associated with Pitt Hopkins Syndrome that could potentially confound interpretation of the...
- A central nervous system structural or vascular abnormality that is a contraindication to the ICV administration procedure, including but not limited...
- Not able to receive prophylactic corticosteroids due to a medical contraindication or participant has a history of a condition that could worsen with...
- Not able to undergo MRI procedures
- Cannot be anesthetized for the ICV injection
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Aurora, Colorado, United States
- Chicago, Illinois, United States
- Tel Litwinsky, Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Aurora, Colorado, United States; Chicago, Illinois, United States; Tel Litwinsky, Israel. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.