Safety of Single and Repeat Dose of PYC-001 Eye Injections in People with ADOA
Research type
Research Study
Full title
A Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
IRAS ID
1012458
Contact name
Sreenivasu Mudumba
Contact email
Sponsor organisation
PYC Therapeutics, LLC
Clinicaltrials.gov Identifier
Research summary
Autosomal dominant optic atrophy (ADOA) is an inherited eye condition that causes vision to get worse over time, with mild to moderate loss of sight and damage to the part of the eye that helps you see. Most people with ADOA have a mutation in their OPA1 gene. Usually, people have two working copies of this gene; however, in people with OPA1 mutation driven ADOA, only one of these copies works properly. This leads the body to make less OPA1 protein, which leads to the optic nerve breaking down (“optic atrophy”), which leads to vision loss. There are currently no approved standard therapies to treat ADOA.
In this trial, the drug PYC-001 is being studied to see if it is safe and to understand the best dose and treatment schedule for treating ADOA. The long-term plan is to see if PYC-001 will help the body make the desired amount of OPA1 protein in the eye and will help prevent further vision loss in people with ADOA.
This is an open-label trial, meaning that everyone participating in the trial will receive PYC 001. All participants will be 18 years or older and have an ADOA diagnosis. Participants in the trial will receive either a single dose of PYC-001 or multiple doses of PYC 001 in the eye. Each participant will be in the trial for ~12-15 months. Participants will be monitored for any side effects, and eye tests will be performed to understand any changes to participant vision and eye structure over time.
While it is possible that participation in this trial will stabilize vision loss for ADOA patients that participate, there are no guaranteed benefits for participating in this trial.
This trial will recruit participants in the UK and Australia.
This trial is funded by PYC Therapeutics, LLC.REC name
London - Central Research Ethics Committee
REC reference
25/LO/0664
Date of REC Opinion
6 Nov 2025
REC opinion
Further Information Unfavourable Opinion