NCT06970106 Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)

Eligibility & Interventions

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What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.

This trial targets 18 participants in total. It began in 2025-09-30 with a primary completion date of 2027-12-01.

Brief 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

Eligibility Criteria

Inclusion Criteria: 1. Must give written informed consent before any study-related activity is carried out 2. Adult males and females, aged 18 years and above at screening; 3. Body mass index ≥18.0 and ≤35.0 kg/m2 4. 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. 5. 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). 6. 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) 7. Medically healthy (in the opinion of the PI), as determined by pre-study medical history 8. Female participants must be of non-childbearing potential or if female participants are of childbearing potential, they must: 1. Have a negative pregnancy test at the screening visit and on study Day -1; 2. Agree not to attempt to become pregnant or donate ova from signing of the consent form until at least 130 days after final IVT dose administration of PYC-001; 3. Agree to use adequate contraception 9. Male participants must: 1. Agree not to donate sperm 2. If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception 3. If engaging in sexual intercourse with a female partner who is not of childbearing potential or a same-sex partner, agree to use a condom 10. Willing and able to comply with all study assessments and protocol schedule/ restrictions Exclusion Criteria: 1. Participant has a known allergy to PYC-001 or any of its excipients; 2. Demonstrated clinically significant co-morbidities, which, in the opinion of the PI, would interfere with the participant's ability to participate in the study and/or confound study outcomes; 3. Females who are breastfeeding or planning to breastfeed; 4. Based on recent genetic testing, the participant has mutations in genes that cause ADOA, other than OPA1 (for example in case of dominant negative ADOA and ADOA Plus) or has other pathological variants that result in an ADOA-like optic atrophic phenotype or other pathologic genetic findings indicating presence of additional confounding ocular diseases based on comprehensive genetic screening. 5. Have received any prior cell or gene therapy for a retinal condition, excluding participation in study PYC-001-101; 6. Within three months prior to study Day -1, have undergone any vitreoretinal surgery or any other ocular surgery in the study eye. 7. Within three months prior to study Day -1, have placement of an Ozurdex® implant. T 8. Within three years prior to study Day -1, have placement of Retisert® of Iluvien® implants. 9. Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, ; 10. Macular edema (intraretinal, sub-retinal or other fluid) in the study eye requiring treatment. 11. History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation; 12. Have used within 30 days of the Screening visit or is using any investigational drug or over-the-counter drug such as Idebenone, Vitamin B6, Vitamin B12, A decision will be made on a case-by-case basis by the PI in consultation with the Sponsor. 13. Over-the-counter drugs like CoQ10 and other Nutraceutical usage will require a washout by five half-lives prior to baseline visit. 14. Have a recent history (\<6 months) of or current excessive recreational drug or alcohol use, in the opinion of the PI. 15. Positive alcohol breath test as assessed at screening, and on study Day -1 and study Day 1; 16. Positive urine drugs of abuse as assessed at screening and on study Day -1 and study Day 1; 17. Any retinal pathology other than ADOA or any other condition or prior therapy that in the opinion of the PI would make the volunteer unsuitable for this study 18. Presence of illness or pathology that, per investigator, include symptoms and/or the associated treatments that can alter visual function including current ocular infection. 19. Positive test for human immunodeficiency virus, hepatitis B or C virus; 20. Clinically significant findings in clinical chemistry, hematology, coagulation and urinalysis tests at screening

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