Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
Recruiting now · Phase 1/Phase 2
Conditions studied: Stargardt Disease
In brief
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.
Key facts
- Study ID
- NCT07439887
- Run by
- Ray Therapeutics, Inc.
- People needed
- 18
- Starts
- 2026-01-28
- Expected to finish
- 2030-12-01
- Last updated by the study team
- 2026-04-15
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
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
Where it is running
- RayTx Clinical Site — Bakersfield, California, United States (enrolling)
- UPMC Vision Institute — Pittsburgh, Pennsylvania, United States (enrolling)
- RayTx Clinical Site — Bellaire, Texas, United States (enrolling)
Full record on ClinicalTrials.gov
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