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Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

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

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

NCT06970106•PYC Therapeutics

View on ClinicalTrials.gov

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 21 participants from across UK and Australia 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 8 weeks 7. Three doses of 60ug PYC-001 at an interval of 12 weeks

Detailed Description

This is a phase 1b open-label, randomized, single and repeat dose study to evaluate the safety and tolerability of IVT administered PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. The primary objective of this study is to gather safety data and determine the optimal dosing regimen for PYC-001. The exploratory objectives of this study include evaluating the ocular structural and functional changes following multiple doses of IVT administered PYC-001. The PK profile of PYC-001 following multiple doses will also be assessed. In this open-label study, PYC-001 will be injected in a single eye and ocular safety will be assessed in both eyes. Approximately 21 participants are expected to enroll and complete this study; the number of enrolled participants may vary and will be driven by decisions made by the safety review committee (SRC) throughout the course of the study.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Must give written informed consent before any study-related activity is carried out and must be able to understand the full nature and purpose of the study, including possible risks and adverse effects;
•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. In case of complex mutation profile, eligibility will be determined in consultation with the Sponsor. Rollover participants are exempt from this criterion as their genetic diagnosis was confirmed in PYC-001-101;
•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). If both eyes meet this eligibility criteria, the eye with better fixation as determined by the PI in consultation with the Sponsor will be selected as the study eye and the other eye will be designated as the fellow eye. In the event that both eyes are eligible and have adequate fixation to reliably perform all study assessments, the worse eye as determined by the physician will be taken as the study eye.
•PYC-001-101 participants are exempt from this criterion and will have the same study eye and fellow eye as determined in PYC-001-101;
•6. Treatment Naïve participants (participants from PYC-001-101 are exempt from this criterion) 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), specifically the outer ring (T, TS, NS, N, NI and TI), defined as:
•1. Mild disease = RNFL abnormalities (outside normal range) in no more than one of six sectors;
•2. Moderate disease = RNFL abnormalities (outside normal range) in no more than three of six sectors;
•3. Severe disease = RNFL abnormalities (outside normal range) in four of six sectors;
+17 more criteria

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 (within five years of screening \[for rollover PYC-001-101 participants, within five years of entry into PYC-001-101\]) 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. Eligibility will be determined by the PI in consultation with the Sponsor as needed;
•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 (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus or intraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive procedures or multiple filtration surgeries \[two or more\]) or any other ocular surgery in the study eye.
•7. Within three months prior to study Day -1, have placement of an Ozurdex® implant. This criterion does not apply for rollover participants from PYC-001-101;
•8. Within three years prior to study Day -1, have placement of Retisert® of Iluvien® implants. This criterion does not apply for rollover participants from PYC-001-101;
•9. Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the PI in the study eye ;
•10. Macular edema (intraretinal, sub-retinal or other fluid) in the study eye requiring treatment.
+16 more criteria

Locations (3)

Save Sight Institute - Sydney Eye Hospital

Sydney, New South Wales, Australia

Cerulea Clinical Trials

East Melbourne, Australia

Retina Specialists

Auckland, New Zealand

Key Dates

Start Date

September 30, 2025

Primary Completion Date

December 1, 2026

Completion Date

January 1, 2027

Last Updated

January 21, 2026

Enrollment

ESTIMATED participants

Conditions

OPA1 Gene MutationAutosomal Dominant Optic AtrophyHereditary Optic AtrophiesKjer Optic Atrophy

Interventions

PYC-001

DRUG

Contacts

Sreenivasu Mudumba

CONTACT

510-423-2680 adoa@pyctx.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 21, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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