Stargardt DiseasePhase 1/2AURORANCT07439887
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
A Phase 1/2 study testing RTx-021 in people with Stargardt Disease, run by Ray Therapeutics, Inc. at 3 sites in United States.
Trial summary
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- RTx-021
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesUnited States
- Study dates
- 28 Jan 2026to 1 Dec 2030
- Phase
- Phase 1/2
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients >= 16 years of age
- Able to comply with the study visit schedule and all protocol assessments
- Diagnosis of Stargardt Disease (genetic testing required)
- Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
- Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center
- Adequate organ function and general good health
Who cannot join
- Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months
- Concurrent participation in another interventional clinical ocular study
- Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
- Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications
- Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications
- Complicating systemic diseases including those in which the disease itself, or the treatment of the disease, can alter ocular and/or central nervous system function (e.g. radiation treatment of the orbit; leukemia with optic nerve involvement)
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- Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others
- Cataract or other ocular (including refractive) surgery, intraocular and/or peri-ocular injection in either eye within the prior four months (i.e. 120 days) prior to screening
- Prior vitrectomy or aphakia in the study eye
- Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g. povidone-iodine to prep for intravitreal injection)
- Known contraindication to prophylactic steroid regimen
- Current pregnancy or breastfeeding
- Any other condition that would not allow the patient to complete follow-up examinations during the study
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
RayTx Clinical Site
Bakersfield, California, 93309
- Recruiting
RayTx Clinical Site
Bellaire, Texas, 77401
- Recruiting
UPMC Vision Institute
Pittsburgh, Pennsylvania, 15219
UPMC Vision Institute Clinical Trials Team, 412-642-7888, Clinicaltrialsoph@upmc.edu
About this study
Official title: Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.
Read the detailed description
This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.
- Conditions
- Stargardt Disease
- Being tested
- RTx-021
- Keywords
- AURORA, RTx-021
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RTx-021Genetic
Optogenetic gene therapy
| Study group | Role | What they receive |
|---|---|---|
| Low Dose RTx-021 | Experimental | Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. Receives: Genetic: RTx-021 |
| Middle Dose RTx-021 | Experimental | Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. Receives: Genetic: RTx-021 |
| High Dose RTx-021 | Experimental | Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. Receives: Genetic: RTx-021 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
Measured: 6 Months
Secondary outcomes (6)
Best Corrected Visual Acuity (BCVA)
Change from Baseline to Month 6 after injection with RTx-021 in BCVA
Measured: 6 Months
Low Luminance Visual Acuity (LLVA)
Change from Baseline to Month 6 after injection with RTx-021 in LLVA
Measured: 6 Months
MNREAD Reading Assessment
Change from Baseline to Month 6 after injection with RTx-021 in reading acuity and reading speed.
Measured: 6 Months
Contrast Sensitivity
Change from Baseline to Month 6 after injection with RTx-021 in contrast sensitivity
Measured: 6 Months
Visual Field
Change from Baseline to Month 6 after injection with RTx-021 in the total area in which objects can be seen
Measured: 6 Months
Low Vision Quality of Life (VA LV VFQ-48)
Change from Baseline to Month 6 after injection with RTx-021 in low vision quality of life questionnaire VA LV VFQ-48. A total score will be calculated with higher scores indicating an improvement.
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start28 Jan 2026
- Primary completion1 Dec 2030 (estimated)
- Study completion1 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ray Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07439887
- Sponsor's protocol ID
- RTx-021-CP-101
- Acronym
- AURORA
- Status last verified
- Feb 2026
- First submitted
- 18 Feb 2026
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: IPD is not currently being shared as this investigational treatment has not received regulatory approval.
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)
- ClinicalTrials.gov ID
- NCT07439887
- Acronym / sponsor ID
- AURORA / RTx-021-CP-101
- Conditions
- Stargardt Disease
- Keywords
- AURORA, RTx-021
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Ray Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Jan 2026 (actual)
- Primary completion
- 1 Dec 2030 (estimated)
- Study completion
- 1 Dec 2030 (estimated)
- First posted
- 27 Feb 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.
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.
Detailed description
This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose RTx-021 | Experimental | Genetic: RTx-021Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. |
| Middle Dose RTx-021 | Experimental | Genetic: RTx-021Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. |
| High Dose RTx-021 | Experimental | Genetic: RTx-021Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male and female patients >= 16 years of age
- Able to comply with the study visit schedule and all protocol assessments
- Diagnosis of Stargardt Disease (genetic testing required)
- Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
- Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center
Key exclusion criteria (13 listed)
- Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months
- Concurrent participation in another interventional clinical ocular study
- Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
- Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications
- Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications
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 |
|---|---|---|
| RTx-021Optogenetic gene therapy | Genetic | High Dose RTx-021, Low Dose RTx-021, Middle Dose RTx-021 |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 6 Months
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
6 secondary outcomes
Best Corrected Visual Acuity (BCVA)
Time frame: 6 Months
Low Luminance Visual Acuity (LLVA)
Time frame: 6 Months
MNREAD Reading Assessment
Time frame: 6 Months
Contrast Sensitivity
Time frame: 6 Months
Visual Field
Time frame: 6 Months
Low Vision Quality of Life (VA LV VFQ-48)
Time frame: 6 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 15 Apr 2026.
- Individual participant dataSharing planned: No. IPD is not currently being shared as this investigational treatment has not received regulatory approval.
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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 1/2 study testing RTx-021 in people with Stargardt Disease, run by Ray 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 13 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 15 Apr 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Executive Clinical Director (858-617-8610, clinicaltrials@raytherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: 6 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 15 Apr 2026; AzTrials last refreshed its copy on 27 Oct 2026.