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Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

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Trial Profile

Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Sep 2026

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At a glance

  • Drugs MZ 1866 (Primary)
  • Indications Pitt-Hopkins syndrome
  • Focus Adverse reactions; First in man
  • Acronyms UNITE
  • Sponsors MAHZI Therapeutics

Most Recent Events

  • 25 Aug 2026 According to MAHZI Therapeutics media release, this study is more than 50% enrolled. This study has enrolled 7 of 12 planned participants and enrollment is projected to be completed by the end of this year.
  • 25 Feb 2026 According to MAHZI Therapeutics media release, company that the first patient has been dosed in its Phase 1/2 UNITE study evaluating MZ-1866, an investigational gene therapy for the treatment of Pitt Hopkins syndrome.
  • 18 Feb 2026 Status changed from not yet recruiting to recruiting.

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