C.001 for retinal degeneration: a first-in-human dose-escalation safety test
Official title: A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
Run by Cellio Therapeutics Inc · NCT07594236 · Data updated 2026-11-12
A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | Cellio Therapeutics Inc Industry-sponsored |
| Locations | 3 sites (Beverly Hills, California and 2 other locations) |
| Status | Recruiting |
| Started | April 7, 2026 |
| Main results expected | December 31, 2028 |
| Study ends | December 31, 2029 |
| Registry record updated | November 3, 2026 |
| Trial ID | NCT07594236 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults 18 and older with retinal diseases involving RPE degeneration (e.g., geographic atrophy or Stargardt disease) and stable ocular condition who are medically suitable for ophthalmic surgery and anesthesia.
✕Probably not a fit
Patients with active CNV, uncontrolled cardiovascular disease, uncontrolled glaucoma, significant ocular inflammation, recent intraocular surgery, systemic infection, or poorly controlled diabetes may have little to no benefit and higher risk.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a Phase 1, single-center, open-label, dose-escalation trial evaluating C.001 delivered by subretinal injection. Three sequential dose cohorts will be enrolled with sentinel dosing and independent DSMB review before escalating doses, and at least 4 weeks between dose steps. Participants will be followed up to 12 months for safety and exploratory assessment of visual function and retinal structure. Primary goal is safety over 3 months; secondary observations include changes in vision and retinal anatomy. The study will enroll adults with retinal diseases linked to RPE degeneration and will assess feasibility and tolerability of the intervention.
Why it matters
Potential benefit: If successful, participants may experience stabilization or improvement in visual function or retinal structure, and future patients could benefit from the approach.
How similar studies have gone: This approach is early in development; prior similar gene or cellular therapies for retinal diseases have shown safety signals in Phase 1 settings, but C.001 remains investigational with unknown efficacy.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
-
Retina Vision Consultants
Beverly Hills, California, United States Recruiting -
Retinal Consultants Medical Group
Sacramento, California, United States Recruiting -
Palo Alto Medical Foundation
Palo Alto, California, United States Not yet recruiting
Study contacts
- Study coordinator: Clinical Trial Administrator
- Email: trials@celliotherapeutics.com
- Phone: 999-999-9999
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Understanding Age-Related Macular Degeneration and Its Progression
This study looks at how age-related macular degeneration develops and progresses by comparing people with the condition to healthy individuals, using non-invasive tests and personal feedback.
-
Central microperimetry and radial OCT in geographic atrophy
We will test whether central microperimetry and radial OCT scans can more accurately measure how geographic atrophy progresses toward the fovea in people with or at risk for GA from age-related macular degeneration.
-
Eye injections of ABBV-6628 for geographic atrophy from age-related macular degeneration
This trial will test whether intravitreal ABBV-6628 is safe and how the drug moves through the body in adults aged 50 and older with geographic atrophy from AMD.
-
Assessing a vision test for patients with macular degeneration and Stargardt disease
This study is testing a new vision test to see how well it works for people with macular degeneration and Stargardt disease compared to standard vision tests.
-
Evaluating a stem cell treatment for geographic atrophy from dry age-related macular degeneration.
This study is testing a new stem cell treatment for people with geographic atrophy from dry age-related macular degeneration to see if it can help improve their vision.
-
Monitoring progression of non-foveal age-related macular degeneration using artificial intelligence
This study is testing how well artificial intelligence can help track the progression of a specific type of age-related macular degeneration in patients over two years.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.