C.001 for Macular Degeneration

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Cellio Therapeutics Inc

Disqualifiers: Choroidal neovascularization, Cardiovascular disease, Glaucoma, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called C.001, an experimental therapy for retinal diseases that cause degeneration of the eye's light-sensitive layer, such as geographic atrophy or Stargardt disease. The main goal is to determine the safety of C.001 and its effects on vision over time. Participants will receive one of three doses through a small eye injection and will be monitored for changes in vision and eye health. Ideal participants have been diagnosed with these retinal conditions and have a stable eye condition without recent surgeries or infections. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that C.001 is likely to be safe for humans?

Research has shown limited safety information for C.001 in humans, as this is its first test in people. In this study, researchers aim to assess how well participants tolerate the treatment and identify any side effects. As a Phase 1 study, the primary goal is to ensure the treatment's safety for humans. Researchers will begin with low doses and gradually increase them, closely monitoring participants for any adverse effects. This careful approach helps ensure participant safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for macular degeneration, such as anti-VEGF injections like Lucentis and Eylea, C.001 offers a unique approach with its varying dosage levels. Researchers are excited about C.001 because it targets the disease with potentially fewer side effects due to its customizable dosing: low, medium, and high. This flexibility in dosing could lead to more personalized treatment plans, potentially increasing effectiveness and patient comfort. Additionally, C.001 might work through a novel mechanism, setting it apart from current options that primarily focus on inhibiting blood vessel growth.

What evidence suggests that C.001 might be an effective treatment for macular degeneration?

Research is investigating C.001 as a potential treatment for eye diseases that cause vision loss. While its effectiveness in humans remains unknown, the primary focus is on its ability to protect or heal a layer of cells in the eye. This approach aligns with other treatments that aim to slow these eye diseases. Early results from similar studies suggest it might help slow the disease. However, the current main goal is to determine if C.001 is safe for human use. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults 18 or older with certain retinal diseases like Stargardt disease or age-related macular degeneration that involve damage to the retinal pigment epithelium. Participants must have stable eye conditions, meet specific vision requirements, and be healthy enough for eye surgery.

Inclusion Criteria

I am willing to follow all study procedures and attend follow-up visits.

I am 18 years old or older.

My vision meets the required standards for this study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single administration of C.001 delivered by subretinal injection. The study evaluates three dose levels in a sequential manner.

4 weeks

1 visit (in-person) for administration, followed by safety assessments

Dose Escalation

A sentinel dosing strategy is used with safety review by an independent DSMB prior to escalation to higher dose levels.

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments of visual function and retinal structure.

12 months

Regular follow-up visits for safety and exploratory evaluations

What Are the Treatments Tested in This Trial?

Interventions

• C.001

Trial Overview

The study is testing a new treatment called C.001 given as a single injection under the retina at one of three dose levels. The main goal is to see if it’s safe and well-tolerated in people with degenerative retinal diseases.

How Is the Trial Designed?

Group I: Arm 3 Experimental Treatment 1 Intervention

C.001 High Dose

Group II: Arm 2 Experimental Treatment 1 Intervention

C.001 Medium Dose

Group III: Arm 1 Experimental Treatment 1 Intervention

C.001Low Dose

Who Is Running the Clinical Trial?

Cellio Therapeutics Inc

Lead Sponsor

California Institute for Regenerative Medicine (CIRM)

Collaborator

Trials

70

Recruited

3,300+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07594236

Phase 1 Study of C.001 in Retinal Degeneration

Participants will be followed for up to 12 months after treatment for safety assessments and exploratory evaluation of visual and anatomical ...

2.

ophthalmologytimes.com

ophthalmologytimes.com/view/alkeus-pharmaceuticals-announces-results-from-the-saga-study-of-oral-gildeuretinol-in-patients-with-ga

Alkeus Pharmaceuticals announces results from the SAGA ...

Oral gildeuretinol demonstrated a clinically meaningful reduction in the geographic atrophy lesion growth rate at 24 months, supporting additional clinical ...

3.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40280279/

Pegcetacoplan Treatment for Geographic Atrophy in Age- ...

Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points.

4.

modernretina.com

modernretina.com/view/alkeus-pharmaceuticals-shares-results-of-saga-study-of-oral-gildeuretinol-acetate-for-ga

Alkeus Pharmaceuticals shares results of SAGA study ...

Oral gildeuretinol demonstrated a clinically meaningful reduction in the GA lesion growth rate at 24 months in the SAGA study.

5.

alkeuspharma.com

alkeuspharma.com/alkeus-pharmaceuticals-announces-results-from-the-saga-study-of-oral-gildeuretinol-in-patients-with-geographic-atrophy-secondary-to-age-related-macular-degeneration/

Alkeus Pharmaceuticals Announces Results from the ...

Oral gildeuretinol demonstrated a clinically meaningful reduction in the geographic atrophy (GA) lesion growth rate at 24 months, ...

6.

ophthalmologytimes.com

ophthalmologytimes.com/view/ctx001-enters-first-in-human-phase-i-ii-trial-for-geographic-atrophy

First patient dosed in phase I/II trial of complement- ...

No efficacy or safety data from human participants have yet been reported. As with other retinal gene therapies, key considerations will include ...

7.

ophthalmologyscience.org

ophthalmologyscience.org/article/S2666-9145(24)00012-5/fulltext

Safety Results for Geographic Atrophy Associated with Age ...

Safety results for geographic atrophy associated with age-related macular degeneration using subretinal cord blood platelet-rich plasma.

8.

withpower.com

withpower.com/trial/phase-1-stargardt-disease-3-2026-3a019

C.001 for Macular Degeneration

This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving ...