Phase 1 Study of C.001 in Retinal Degeneration
Purpose
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.
Conditions
- Geographic Atrophy
- Stargardt Disease
- RPE-mediated Maculopathy
- Age Related Macular Degeneration
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts 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 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
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
- Masking Description
- Open-Label
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Arm 1 |
C.001Low Dose |
|
|
Experimental Arm 2 |
C.001 Medium Dose |
|
|
Experimental Arm 3 |
C.001 High Dose |
|
Recruiting Locations
Beverly Hills, California 90210
More Details
- Status
- Recruiting
- Sponsor
- Cellio Therapeutics Inc
Study Contact
Detailed 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 and anatomical outcomes.