MZ-1866 for Pitt Hopkins Syndrome

Overseen By Emily Radomile

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Mahzi Therapeutics

Disqualifiers: Large TCF4 deletion, CNS abnormalities, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety of a new treatment, MZ-1866, for individuals with Pitt Hopkins Syndrome, a rare genetic disorder affecting development and behavior. Participants will receive a single dose of the treatment via an injection into the brain. Researchers will closely monitor participants' health and development over time. Individuals with a confirmed mutation in the TCF4 gene and symptoms of Pitt Hopkins Syndrome may qualify. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that MZ-1866 is likely to be safe for humans?

Research shows that MZ-1866 is being explored as a possible treatment for Pitt Hopkins Syndrome. Understanding the safety of this treatment for people is crucial. As a Phase 1/2 study, it remains in the early stages of testing and has not been widely tried in people yet.

The main goal is to determine if MZ-1866 is safe and well-tolerated. The study closely monitors any side effects or health changes. Participants receive one dose injected into the fluid-filled spaces of the brain and undergo regular check-ups by doctors.

Due to the early stage of testing, limited information about safety is available. However, the study tracks any unexpected medical issues to understand potential risks. Anyone interested in joining should consider this and discuss any concerns with a healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the current therapies for Pitt Hopkins Syndrome, which primarily focus on managing symptoms and improving quality of life, MZ-1866 offers a novel approach by directly targeting the underlying genetic causes of the condition. Researchers are excited about MZ-1866 because it has the potential to correct the genetic mutations associated with Pitt Hopkins Syndrome, something current treatments can't do. This unique mechanism of action could mean significant improvements in cognitive and developmental outcomes for patients, offering hope for a more effective treatment option.

What evidence suggests that MZ-1866 might be an effective treatment for Pitt Hopkins Syndrome?

Research has shown that MZ-1866 is designed to treat Pitt Hopkins Syndrome by targeting its root cause. This condition stems from a problem with the TCF4 gene, and MZ-1866 aims to provide extra copies of this gene. Animal studies suggest that delivering MZ-1866 directly into the central nervous system might improve symptoms by replacing the missing TCF4. Although research in humans is ongoing, these early findings are promising. Participants in this trial will receive MZ-1866, with Cohort 1 including ages 12-25 and Cohort 2 including ages 2-11. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for children and adults with Pitt Hopkins Syndrome, a rare genetic disorder. Participants must have a clinical diagnosis of the syndrome, confirmed 'pathogenic' or 'likely pathogenic' TCF4 gene mutation, and be willing to provide medical records. It's not specified who can't join.

Inclusion Criteria

My condition aligns with Pitt Hopkins Syndrome and I agree to share my medical history.

My tests show a harmful TCF4 gene mutation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of MZ-1866 by intracerebroventricular injection

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including periodic evaluations using neurodevelopmental tools

104 weeks

Periodic visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• MZ-1866

Trial Overview The study tests MZ-1866, an AAV-9 gene therapy given once via injection into the brain's ventricles. The trial aims to assess its safety by monitoring adverse events, physical exams, lab results, ECGs, health changes over time, and neurodevelopmental progress.

How Is the Trial Designed?

Group I: Cohort 2 (MZ-1866) Experimental Treatment 1 Intervention

Participants aged 2-11 years

Group II: Cohort 1 (MZ-1866) Experimental Treatment 1 Intervention

Participants ages 12-25 years

Who Is Running the Clinical Trial?

Mahzi Therapeutics

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07135050

Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy ... 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 ...

2. pitthopkins.org pitthopkins.org/mahzi/

Mahzi Gene Therapy Clinical Trial MZ-1866 is intended address the root cause of Pitt Hopkins Syndrome by providing additional copies of properly functioning TCF4 genes. While it is not known ...

3. cirm.ca.gov cirm.ca.gov/our-progress/awards/development-gene-therapy-treatment-pitt-hopkins-syndrome-phs-translating-animal-proof-concept-support-pre-ind-meeting/

Development of a Gene Therapy for the Treatment of Pitt ... A single delivery of Mz-1866 into the central nervous system may transduce neurons to replace the deficiency of TCF4 and ultimately improve the clinical ...

4. patlynk.com patlynk.com/trial/NCT07135050

MZ-1866 Gene Therapy for Pitt Hopkins Syndrome This clinical trial is focused on testing a new treatment called MZ-1866, specifically designed for children and adults diagnosed with Pitt Hopkins Syndrome ...

5. synapse.patsnap.com synapse.patsnap.com/drug/8f9be21f76bd4cb8a4f53cdef5d98639

MZ-1866 - Drug Targets, Indications, Patents Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by ...

6. adisinsight.springer.com adisinsight.springer.com/trials/700385458

Phase 1/2 First-in-Human Study to Evaluate the Safety ... 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.