Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy
Status:
RECRUITING
Trial end date:
2027-01-01
Target enrollment:
Participant gender:
Summary
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 21 participants from across UK and Australia 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 g 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 8 weeks
7. Three doses of 60ug PYC-001 at an interval of 12 weeks