Geographic AtrophyPhase 1NCT07594236
Phase 1 Study of C.001 in Retinal Degeneration
A Phase 1 study testing Injection in people with Geographic Atrophy and Stargardt Disease, run by Cellio Therapeutics Inc at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy
- What is being tested?
- Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesUnited States
- Study dates
- 7 Apr 2026to 31 Dec 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Retina Vision Consultants
Beverly Hills, California, 90210
Pradeep Prasad, MD, (310) 269-8565, trials@celliotherapeutics.com
- Not yet recruiting
Palo Alto Medical Foundation
Palo Alto, California, 94301
Ehsan Rahimy, MD, (650) 853-2974, trials@celliotherapeutics.com
- Recruiting
Retinal Consultants Medical Group
Sacramento, California, 95825
David Telander, MD, (916) 454-4861, trials@celliotherapeutics.com
About this study
Official title: A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
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.
Read the 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.
- Conditions
- Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
- Being tested
- Injection
- Keywords
- macular degeneration, macular distrophy, C.001
- Medical subject headings
- Geographic Atrophy, Stargardt Disease, Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InjectionBiological
C.001 administered once via subretinal injection
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | C.001Low Dose Receives: Biological: Injection |
| Arm 2 | Experimental | C.001 Medium Dose Receives: Biological: Injection |
| Arm 3 | Experimental | C.001 High Dose Receives: Biological: Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Ocular and Systemic Adverse Events
Measured: 3 months
Secondary outcomes (2)
Change in visual function measured in EDTRS letters
Measured: 3 months
Findings of anatomic change on optical coherence tomography (OCT)
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2026
- Study start7 Apr 2026
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellio Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07594236
- Sponsor's protocol ID
- CT-C.001-001
- Other IDs
- CLIN2-19416
- Status last verified
- Aug 2026
- First submitted
- 17 Apr 2026
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
- ClinicalTrials.gov ID
- NCT07594236
- Acronym / sponsor ID
- CT-C.001-001
- Conditions
- Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
- Keywords
- macular degeneration, macular distrophy, C.001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Cellio Therapeutics IncIndustry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 7 Apr 2026 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 18 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: InjectionC.001Low Dose |
| Arm 2 | Experimental | Biological: InjectionC.001 Medium Dose |
| Arm 3 | Experimental | Biological: InjectionC.001 High Dose |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InjectionC.001 administered once via subretinal injection | Biological | Arm 1, Arm 2, Arm 3 |
Primary outcome
Incidence of Ocular and Systemic Adverse Events
Time frame: 3 months
2 secondary outcomes
Change in visual function measured in EDTRS letters
Time frame: 3 months
Findings of anatomic change on optical coherence tomography (OCT)
Time frame: 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Injection in people with Geographic Atrophy and Stargardt Disease, run by Cellio Therapeutics Inc at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Administrator (999-999-9999, trials@celliotherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Ocular and Systemic Adverse Events (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.