Recruiting
Phase 1

C.001

Sponsor:

Cellio Therapeutics Inc

Code:

NCT07594236

Conditions

Geographic Atrophy

Stargardt Disease

RPE-mediated Maculopathy

Age Related Macular Degeneration

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Injection

Study Details

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

Geographic Atrophy

Stargardt Disease

RPE-mediated Maculopathy

Age Related Macular Degeneration

Study ID

NCT07594236

Start date

Apr 7, 2026

Status verified date

Aug, 2026

Completion date

Dec 31, 2029

Anticipated

Primary completion date

Dec 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Enrollment

15 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm 1

C.001Low Dose

experimental: Arm 2

C.001 Medium Dose

experimental: Arm 3

C.001 High Dose

Interventions

Injection

C.001 administered once via subretinal injection

Primary outcome measure

  • Incidence of Ocular and Systemic Adverse Events [ Time Frame: 3 months ]

Central Contacts and Locations

Central contacts

Clinical Trial Administrator

999-999-9999trials@celliotherapeutics.com

Locations

Retina Vision Consultants

Recruiting

Beverly Hills, California, United States, 90210

Contacts

Pradeep Prasad, MD

(310) 269-8565trials@celliotherapeutics.com

More Information

Sponsor

Cellio Therapeutics Inc

Last update posted

Aug 17, 2026

Last verified

Aug, 2026

Keywords

  • macular degeneration
  • macular distrophy
  • C.001

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Cellio Therapeutics Inc on 2026-08-17.