A Phase 1 interventional study of Injection in Geographic Atrophy, Stargardt Disease and RPE-mediated Maculopathy, sponsored by Cellio Therapeutics Inc. Recruiting at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-17.
Sponsored by Cellio Therapeutics Inc · Phase 1, Interventional, and Treatment
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.
This is a Phase 1, single-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of C.001 administered via subretinal injection.
Participants will be enrolled into three sequential dose cohorts. A sentinel dosing strategy will be used, with safety review by an independent Data Safety Monitoring Board (DSMB) prior to escalation to higher dose levels. A minimum observation period of 4 weeks will be required between dose escalations.
Participants will be followed for up to 12 months after treatment for safety assessments and exploratory evaluation of visual and anatomical outcomes.
173 studies on the registry are indexed under Geographic Atrophy; 44 are open to participants now.
This study's planned enrollment of 15 is below the median of 60 across 131 interventional studies indexed under Geographic Atrophy.
Browse Geographic Atrophy studies →This is the only study on the registry with Cellio Therapeutics Inc as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
C.001Low Dose
Biological: Injection
C.001 Medium Dose
Biological: Injection
C.001 High Dose
Biological: Injection
C.001 administered once via subretinal injection
Incidence of Ocular and Systemic Adverse Events
Time frame: 3 months
Change in visual function measured in EDTRS letters
Time frame: 3 months
Findings of anatomic change on optical coherence tomography (OCT)
Time frame: 3 months
Plan to share: No
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