Recruiting
Phase 1
Phase 2

RTx-021

Sponsor:

Ray Therapeutics, Inc.

Code:

NCT07439887

Conditions

Stargardt Disease

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

RTx-021

Study Details

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

Study ID

NCT07439887

Start date

Jan 28, 2026

Status verified date

Feb, 2026

Completion date

Dec 1, 2030

Anticipated

Primary completion date

Dec 1, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Design

Enrollment

18 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Low Dose RTx-021

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.

experimental: Middle Dose RTx-021

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.

experimental: High Dose RTx-021

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.

Interventions

RTx-021

Optogenetic gene therapy

Primary outcome measure

  • Incidence of Treatment-Emergent Adverse Events [ Time Frame: 6 Months ]

Central Contacts and Locations

Central contacts

Executive Clinical Director

858-617-8610clinicaltrials@raytherapeutics.com

Locations

RayTx Clinical Site

Recruiting

Bakersfield, California, United States, 93309

UPMC Vision Institute

Recruiting

Pittsburgh, Pennsylvania, United States, 15219

Contacts

UPMC Vision Institute Clinical Trials Team

412-642-7888Clinicaltrialsoph@upmc.edu

RayTx Clinical Site

Recruiting

Bellaire, Texas, United States, 77401

More Information

Sponsor

Ray Therapeutics, Inc.

Last update posted

Apr 15, 2026

Last verified

Feb, 2026

Keywords

  • AURORA
  • RTx-021

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ray Therapeutics, Inc. on 2026-04-15.