RecruitingStargardt Disease
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
Eligible age
16+ yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
See if you qualify for this study
Answer a few quick questions about your location and health. Takes about a minute.
About this study
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.
Sponsor: Ray Therapeutics, Inc.
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
Where it's recruiting
Source: ClinicalTrials.gov · NCT07439887 · last updated 2026-04-15