Clinical Trial

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

Active, Not Recruiting Phase 1
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Summary
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
Protocol Amendment History 7 amendments
This ClinicalTrials.gov record has been amended 7 times since 2024-06-11; most recent amendment 2026-01-19.
Status change: Recruiting → Active, Not Recruiting 2026-01-19
Status change: Not Yet Recruiting → Recruiting 2024-11-05
Trial Details
NCT Number NCT06461286
Lead Sponsor PYC Therapeutics
Conditions OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
Enrollment 18 participants
Start Date 2024-10-31
Primary Completion 2026-08 (estimated)
Study Completion 2026-08 (estimated)
Updated on ClinicalTrials.gov 2026-01-21