Clinical Trials
Myrtle
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.
Overview
This research project is testing a potential new treatment for the Optic Atrophy 1 (OPA1) mutation associated Autosomal Dominant Optic Atrophy (ADOA), an inherited eye condition that causes mild to moderate vision loss.
The treatment under investigation in this study is called PYC-001.
Normally, people have two working copies of the OPA1 gene, which is responsible for producing the OPA1 protein.
However, in people with OPA1 mutation associated ADOA, only one of these copies produce the OPA1 protein, resulting in a lower amount of OPA1 protein.
Without enough OPA1 protein, the optic nerve degenerates (this is called optic atrophy) and vision loss occurs.
The study drug (PYC-001) uses gene therapy technology to restore production of the OPA1 protein to cells in the eye.
It is hoped that this will help to prevent further vision loss in people with this OPA1 mutation associated ADOA.
The main purpose of the study is to see if the study drug PYC-001 is safe and well tolerated, when administered as an intravitreal injection (an injection into the back of your eye).
This study is an open-label research project, which means that everyone enrolled in the study will be treated with the study drug.
The study will enrol participants in 6 different groups known as cohorts.
Each cohort will receive a different dose of the study drug administered over different timepoints.
The study duration is 1.5 years, with the option of participating in an extension study.
For further information about this study please contact the Clinical Trials Research Centre on (03) 9929 8076 or email info@ceruleaclinicaltrials.org.au.
- Principal Investigator
Dr Doron Hickey
This research project is testing a potential new treatment for the Optic Atrophy 1 (OPA1) mutation associated Autosomal Dominant Optic Atrophy (ADOA), an inherited eye condition that causes mild to moderate vision loss.
The treatment under investigation in this study is called PYC-001.
Normally, people have two working copies of the OPA1 gene, which is responsible for producing the OPA1 protein.
However, in people with OPA1 mutation associated ADOA, only one of these copies produce the OPA1 protein, resulting in a lower amount of OPA1 protein.
Without enough OPA1 protein, the optic nerve degenerates (this is called optic atrophy) and vision loss occurs.
The study drug (PYC-001) uses gene therapy technology to restore production of the OPA1 protein to cells in the eye.
It is hoped that this will help to prevent further vision loss in people with this OPA1 mutation associated ADOA.
The main purpose of the study is to see if the study drug PYC-001 is safe and well tolerated, when administered as an intravitreal injection (an injection into the back of your eye).
This study is an open-label research project, which means that everyone enrolled in the study will be treated with the study drug.
The study will enrol participants in 6 different groups known as cohorts.
Each cohort will receive a different dose of the study drug administered over different timepoints.
The study duration is 1.5 years, with the option of participating in an extension study.
For further information about this study please contact the Clinical Trials Research Centre on (03) 9929 8076 or email info@ceruleaclinicaltrials.org.au.
- Principal Investigator
Learn more
View this study on ClinicalTrials.gov
See the ClinicalTrials.gov database listing for more detailed information about this study.