RecruitingPhase 1

Phase 1 Study of C.001 in Retinal Degeneration

United States15 participantsStarted 2026-04-07

Plain-language summary

This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium. Participants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner. The primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.

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: * Age ≥18 years * Diagnosis of retinal disease associated with retinal pigment epithelium degeneration (e.g., geographic atrophy or Stargardt disease) * Visual acuity within protocol-defined limits * Stable ocular condition prior to enrollment * Medically suitable for ophthalmic surgery and anesthesia * Willingness to comply with study procedures and follow-up Exclusion Criteria: * Active or prior choroidal neovascularization (CNV) * Recent myocardial infarction or significant uncontrolled cardiovascular disease * Uncontrolled glaucoma * Significant ocular inflammatory disease * Recent intraocular surgery within protocol-defined timeframe * Active systemic infection * Poorly controlled diabetes (e.g., HbA1c \>8.5%) * Participation in another investigational study within 3 months prior to screening

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 of Ocular and Systemic Adverse Events

Timeframe: 3 months

Trial details

NCT IDNCT07594236

SponsorCellio Therapeutics Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-12-31

View on ClinicalTrials.gov More Geographic Atrophy trials