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Gene therapy breakthrough? first human trial launches for rare brain disorder

NCT ID NCT07135050

Disease control Sponsor: Mahzi Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Jul 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 25 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * The participant has a TCF4 gene mutation confirmed as "pathogenic" or "likely pathogenic" by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray, performed at an accredited laboratory * 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 demographics and diagnostic and treatment history Exclusion Criteria: * 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 study data * A central nervous system structural or vascular abnormality that is a contraindication to the ICV administration procedure, including but not limited to: signs or symptoms of increased intracranial pressure, history of a space-occupying lesion, or presence of a ventricular shunt that would preclude ICV procedures or safety assessments, or increase risk to the participant * Not able to receive prophylactic corticosteroids due to a medical contraindication or participant has a history of a condition that could worsen with corticosteroid therapy as assessed and determined by the Investigator * Not able to undergo MRI procedures * Cannot be anesthetized for the ICV injection

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Conditions

The condition(s) this trial relates to.

Pitt-Hopkins syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pitt hopkins syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    4 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Rush University Medical Center

    RECRUITING

    Chicago, Illinois, 60612, United States

  • Sheba Medical Center

    RECRUITING

    Tel Litwinsky, Israel

  • UCSF Benioff Children's Hospital Oakland

    RECRUITING

    Oakland, California, 94609, United States

More trials for these conditions

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