Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)

Last updated: June 10, 2025
Sponsor: Ray Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

1

Condition

Eye Disorders/infections

Retinitis Pigmentosa

Posterior Uveitis

Treatment

RTx-015

Clinical Study ID

NCT06460844
RTx-015-CP-101
  • Ages > 18
  • All Genders

Study Summary

A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Adults ≥18 years of age

  • Informed consent obtained from the patient

  • Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation ORClinical diagnosis of Choroideremia with known causative mutation

  • Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria

  • Presence of retinal ganglion cells and/or retinal nerve fiber layer on SpectralDomain Optical Coherence Tomography testing at Screening confirmed by central imagereading center

  • Adequate organ function and general good health

Exclusion

Exclusion Criteria:

  • Participation in a clinical study (ocular or non-ocular) with an investigationaldrug, 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 celltherapy

  • Pre-existing eye conditions in either eye that would preclude the planned treatmentor, in the opinion of the Investigator, are significant enough to interfere with theinterpretation of study endpoints or procedural complications

  • Known steroid responders if their intraocular pressure was not able to be managedeffectively with topical pressure-lowering medications after prior use of steroidmedications

  • Complicating systemic diseases; complicating systemic diseases include those inwhich the disease itself, or the treatment for the disease, can alter ocular and/orCentral Nervous System (CNS) function (e.g., radiation treatment of the orbit;leukemia with CNS/optic nerve involvement)

  • Any immunological response dysfunction including, immuno-compromising diseases oruse of immunosuppressive medications, among others

  • Cataract or other ocular (including refractive) surgery, intraocular and/orperi-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 tomedications planned for use in the peri-procedural period (e.g., povidone-iodine toprep 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-upexaminations during the study

Study Design

Total Participants: 18
Treatment Group(s): 1
Primary Treatment: RTx-015
Phase: 1
Study Start date:
August 27, 2024
Estimated Completion Date:
October 31, 2030

Study Description

This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa or choroideremia. Up to 4 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.

Connect with a study center

  • Retina Vitreous Associates Medical Group

    Beverly Hills, California 90211
    United States

    Active - Recruiting

  • UCI Alpha Clinic

    Orange, California 92868
    United States

    Active - Recruiting

  • UPMC Vision Institute

    Pittsburgh, Pennsylvania 15221
    United States

    Active - Recruiting

  • Retina Consultants of Texas Research Centers

    Bellaire, Texas 77401
    United States

    Active - Recruiting

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