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NCT06860672 · ClinicalTrials.gov record · last posted 2026-07-30

Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation

StatusTerminated
PhaseEarly Phase 1
SponsorYongguo Yu
Started2025-02-19
View on ClinicalTrials.gov ↗
Why the trial stopped
The single participant died on 31 March 2025 from thrombotic microangiopathy, a serious adverse event assessed as definitely related to the study drug. Planned follow-up was not completed, and the study was terminated.
from the ClinicalTrials.gov record
To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene

Protocol amendment history 3 changes detected by DataLookout

2026-07-31
Trial terminated
2026-07-31
Trial terminated: The single participant died on 31 March 2025 from thrombotic microangiopathy, a serious adverse event assessed as defin...
2026-07-31
Completion moved earlier: 2025-09 → 2025-03-31
Trial Details
NCT Number NCT06860672
Lead Sponsor Yongguo Yu
Conditions Developmental Delay Disorder, Intellectual Disability, Rare Diseases
Enrollment 1 participants
Start Date 2025-02-19
Primary Completion 2025-03-31 (estimated)
Study Completion 2025-03-31 (estimated)
Updated on ClinicalTrials.gov 2026-07-30