🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

Find Clinical Trials for Your Rare Disease

1+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Clinical trial records are synced from ClinicalTrials.gov.

Showing 1 of 1 recruiting trials for “Pitt-Hopkins syndrome”

Phase 1, PHASE2RecruitingNCT07135050 ↗

Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

🏥 Mahzi Therapeutics📍 3 sites📅 Started Dec 2025View details ↗

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