A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
Cellio Therapeutics Inc
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.
Description
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 a…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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 d…
Interventions
- BiologicalInjection
C.001 administered once via subretinal injection
Location
- Clinical Trial Site 1Beverly Hills, California