Single intravitreal RTx-021 injection for Stargardt disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a first-in-human, open-label, non-randomized dose-escalation program enrolling 9–18 patients across up to three dose cohorts. Each participant will receive a single unilateral intravitreal injection of the optogenetic gene therapy RTx-021 in the study eye, with cohorts initially of three patients and potential cohort expansion based on safety. Participants will be followed with frequent visits in the first 12 months and continued monitoring out to five years to characterize safety and preliminary signs of efficacy. The design supports selection of a dose for later-stage development.

Why it matters

Who can join: the full rules

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

Where it's running

• RayTx Clinical Site
  Bakersfield, California, United States Recruiting
• UPMC Vision Institute
  Pittsburgh, Pennsylvania, United States Recruiting
• RayTx Clinical Site
  Bellaire, Texas, United States Recruiting

Study contacts

• Study coordinator: Executive Clinical Director
• Email: clinicaltrials@raytherapeutics.com
• Phone: 858-617-8610

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.