Active — Not RecruitingPhase 1

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

Australia18 participantsStarted 2024-10-31

Plain-language summary

A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.

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 have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial, including possible risks and adverse effects;
. Adult males and females, aged 18 years and above at screening;
. Body mass index (BMI) ≥18.0 and ≤32.0 kg/m2, with a body weight ≤ 100kg at screening;
. Have a molecular (genetic) diagnosis of OPA1 mutation at screening;
. Have a clinical diagnosis of OPA1 mutation-associated ADOA at screening;
. Participants with BCVA of between 20/40 (70 ETDRS letters) and 20/160 (39-43 ETDRS letters). If both eyes meet this eligibility criteria, the eye with worse BCVA will be selected as the study eye and the other eye will be designated as the "fellow eye";
. Medically healthy (in the opinion of the Investigator), as determined by pre-study medical history, and without clinically significant abnormalities including:
. Physical examination without any clinically relevant findings;

Exclusion criteria

. Participants has a known allergy to PYC-001 or any of its excipients;

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

Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye

Timeframe: 24 weeks

Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye

Timeframe: 48 weeks

Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye

Timeframe: 24 weeks

Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye

Timeframe: 48 weeks

Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs

Timeframe: 24 weeks

Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs

Timeframe: 48 weeks

Trial details

NCT IDNCT06461286

SponsorPYC Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-08

View on ClinicalTrials.gov More OPA1 Gene Mutation trials

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 have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial, including possible risks and adverse effects;
. Adult males and females, aged 18 years and above at screening;
. Body mass index (BMI) ≥18.0 and ≤32.0 kg/m2, with a body weight ≤ 100kg at screening;
. Have a molecular (genetic) diagnosis of OPA1 mutation at screening;
. Have a clinical diagnosis of OPA1 mutation-associated ADOA at screening;
. Participants with BCVA of between 20/40 (70 ETDRS letters) and 20/160 (39-43 ETDRS letters). If both eyes meet this eligibility criteria, the eye with worse BCVA will be selected as the study eye and the other eye will be designated as the "fellow eye";
. Medically healthy (in the opinion of the Investigator), as determined by pre-study medical history, and without clinically significant abnormalities including:
. Physical examination without any clinically relevant findings;

Exclusion criteria

. Participants has a known allergy to PYC-001 or any of its excipients;

What they're measuring

Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye

Timeframe: 24 weeks

Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye

Timeframe: 48 weeks

Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye

Timeframe: 24 weeks

Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye

Timeframe: 48 weeks

Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs

Timeframe: 24 weeks

Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs

Timeframe: 48 weeks