Phase 1 Study of C.001 in Retinal Degeneration

NCT07594236 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: CT-C.001-001CLIN2-19416
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Stargardt Disease; RPE-mediated Maculopathy; Geographic Atrophy; Age Related Macular Degeneration

Keywords

macular degeneration; macular distrophy; C.001

Outcome Measures

Primary Outcomes (1)

• Incidence of Ocular and Systemic Adverse Events

  3 months

Secondary Outcomes (2)

• Change in visual function measured in EDTRS letters

  3 months

• Findings of anatomic change on optical coherence tomography (OCT)

  3 months

Study Arms (3)

Arm 1

EXPERIMENTAL

C.001Low Dose

Biological: Injection

Arm 2

EXPERIMENTAL

C.001 Medium Dose

Biological: Injection

Arm 3

EXPERIMENTAL

C.001 High Dose

Biological: Injection

Interventions

Injection BIOLOGICAL

C.001 administered once via subretinal injection

Arm 1; Arm 2; Arm 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cellio Therapeutics Inc: lead
• California Institute for Regenerative Medicine (CIRM): collaborator

Study Sites (1)

Clinical Trial Site 1

Beverly Hills, California, 90210, United States

RECRUITING

MeSH Terms

Conditions

Geographic Atrophy; Stargardt Disease; Macular Degeneration

Interventions

Injections

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Open-Label
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2026
• First Posted: May 18, 2026
• Study Start: April 7, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: May 18, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)