RecruitingPhase 1/2

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

Australia, New Zealand18 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 range18 Years

SexALL

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)
✓. Medically healthy (in the opinion of the PI), as determined by pre-study medical history
✓. Female participants must be of non-childbearing potential or if female participants are of childbearing potential, they must:

Exclusion criteria

What they're measuring

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 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 96 weeks

[All Cohorts] Change from baseline in red blood cells

Timeframe: Up to 96 weeks

Trial details

NCT IDNCT06970106

SponsorPYC Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-12-01

Contact for this trial

Sreenivasu Mudumba

510-423-2680 adoa@pyctx.com

View on ClinicalTrials.gov More OPA1 Gene Mutation trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

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)
✓. Medically healthy (in the opinion of the PI), as determined by pre-study medical history
✓. Female participants must be of non-childbearing potential or if female participants are of childbearing potential, they must:

Exclusion criteria

What they're measuring

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 weeks

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

Timeframe: Up to 96 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 96 weeks

[All Cohorts] Change from baseline in red blood cells

Timeframe: Up to 96 weeks