Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

NCT07439887 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: RTx-021-CP-101
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 3

Brief 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. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.

Conditions

Stargardt Disease

Keywords

AURORA; RTx-021

Outcome Measures

Primary Outcomes (1)

• 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

  6 Months

Secondary Outcomes (6)

• Best Corrected Visual Acuity (BCVA)

  6 Months

• Low Luminance Visual Acuity (LLVA)

  6 Months

• MNREAD Reading Assessment

  6 Months

• Contrast Sensitivity

  6 Months

• Visual Field

  6 Months

Study Arms (3)

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.

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.

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.

Genetic: RTx-021

Interventions

RTx-021 GENETIC

Optogenetic gene therapy

High Dose RTx-021; Low Dose RTx-021; Middle Dose RTx-021

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Ray Therapeutics, Inc.: lead

Study Sites (3)

RayTx Clinical Site

Bakersfield, California, 93309, United States

RECRUITING

UPMC Vision Institute

Pittsburgh, Pennsylvania, 15219, United States

RECRUITING

RayTx Clinical Site

Bellaire, Texas, 77401, United States

RECRUITING

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Executive Clinical Director

CONTACT

858-617-8610; clinicaltrials@raytherapeutics.com

Study Design

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

Study Record Dates

• First Submitted: February 18, 2026
• First Posted: February 27, 2026
• Study Start: January 28, 2026
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: April 15, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)