RecruitingPhase 1/2

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

Australia, New Zealand24 participantsStarted 2025-09-30

Plain-language 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 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

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Must give written informed consent before any study-related activity is carried out
. Adult males and females, aged 18 years and above at screening;
. Body mass index ≥18.0 and ≤35.0 kg/m2
. Have a recent (within five years) genetic diagnosis of OPA1 mutation-associated (haploinsufficiency) ADOA and/or confirmed diagnosis during pre-screening or screening, as determined by the PI.
. Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20/40 (≤70 Early Treatment of Diabetic Retinopathy Study \[ETDRS\] letters) and ≥20/200 (≥35 ETDRS letters).
. Treatment Naïve participants with mild to moderate visual field loss and retinal nerve fiber layer (RNFL) loss in the study eye only as determined by the Spectralis Glaucoma Module Premium Edition (GMPE) RNFL \& visual field structure function data (map)

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

[All Cohorts] Number of participants experiencing treatment emergent adverse events

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (heart rate)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (systolic and diastolic blood pressure)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (tympanic temperature)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (respiratory rate)

Timeframe: Up to 164 weeks

[All Cohorts] Change from baseline in white blood cells (WBC), platelets, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count

Timeframe: Up to 164 weeks

[All Cohorts] Change from baseline in red blood cells

Trial details

NCT IDNCT06970106

SponsorPYC Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-10

Contact for this trial

Jessica Stevenson

+61 8 6151 0992 adoa@pyctx.com

View on ClinicalTrials.gov More OPA1 Gene Mutation trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Must give written informed consent before any study-related activity is carried out
. Adult males and females, aged 18 years and above at screening;
. Body mass index ≥18.0 and ≤35.0 kg/m2
. Have a recent (within five years) genetic diagnosis of OPA1 mutation-associated (haploinsufficiency) ADOA and/or confirmed diagnosis during pre-screening or screening, as determined by the PI.
. Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20/40 (≤70 Early Treatment of Diabetic Retinopathy Study \[ETDRS\] letters) and ≥20/200 (≥35 ETDRS letters).
. Treatment Naïve participants with mild to moderate visual field loss and retinal nerve fiber layer (RNFL) loss in the study eye only as determined by the Spectralis Glaucoma Module Premium Edition (GMPE) RNFL \& visual field structure function data (map)

What they're measuring

[All Cohorts] Number of participants experiencing treatment emergent adverse events

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (heart rate)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (systolic and diastolic blood pressure)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (tympanic temperature)

Timeframe: Up to 164 weeks

[All Cohorts] Changes from baseline in vital signs (respiratory rate)

Timeframe: Up to 164 weeks

[All Cohorts] Change from baseline in white blood cells (WBC), platelets, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count

Timeframe: Up to 164 weeks

[All Cohorts] Change from baseline in red blood cells

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Contact for this trial

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Contact for this trial

Exclusion criteria