Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)

This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approximately 18 participants from Australia, New Zealand, and other APAC countries are expected to be enrolled, depending on safety review committee (SRC) throughout the course of the study. Participants may be assigned to any of the following: 1. A single 60ug dose of PYC-001 2. Three doses of 10ug PYC-001 at an interval of 8 weeks 3. Three doses of 10ug PYC-001 at an interval of 12 weeks 4. Three doses of 30ug PYC-001 at an interval of 8 weeks 5. Three doses of 30ug PYC-001 at an interval of 12 weeks Following completion of the 4 week safety review of the single 60ug of PYC-001 cohort, and if the 60 μg dose level is deemed safe by the SRC, the following cohorts will also be available: 6. Three doses of 60ug PYC-001 at an interval of 12 weeks

NCT Number	NCT06970106
Lead Sponsor	PYC Therapeutics
Conditions	OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
Enrollment	24 participants
Start Date	2025-09-30
Primary Completion	2029-10 (estimated)
Study Completion	2029-11 (estimated)
Updated on ClinicalTrials.gov	2026-06-29