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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: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Sep 2025

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At a glance

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

Most Recent Events

  • 01 Sep 2025 New trial record

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