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

Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients With Retinitis Pigmentosa or Choroideremia (ENVISION)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Retinitis Pigmentosa; Choroideremia
• Intervention / Treatment: Genetic: RTx-015

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Beverly Hills, California, United States, 90211
      • Retina Vitreous Associates Medical Group
    • Orange, California, United States, 92868
      • UCI Alpha Clinic
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15221
      • UPMC Vision Institute
  • Texas
    • Bellaire, Texas, United States, 77401
      • Retina Consultants of Texas Research Centers

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adults ≥18 years of age
• Informed consent obtained from the patient
• Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical 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 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, in the opinion of the Investigator, 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; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or Central 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 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low Dose RTx-015; Single intravitreal injection of low dose RTx-015	Genetic: RTx-015; 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-015; Single intravitreal injection of middle dose RTx-015	Genetic: RTx-015; 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-015; Single intravitreal injection of high dose RTx-015	Genetic: RTx-015; 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: Higher Dose RTx-015; Single intravitreal injection of higher dose RTx-015	Genetic: RTx-015; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events	The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Best Corrected Visual Acuity (BCVA) at Month 6 and Month 12	Change from baseline to Month 6 and Month 12 after injection with RTx-015 in BCVA	6 and 12 Months
Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12	Change from baseline to Month 6 and Month 12 after injection with RTx-015 in LLVA	6 and 12 Months
Multi luminance mobility at Month 6 and Month 12	Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Visual Change from baseline to Month 6 and 12 in mobility performance at multiple luminance levels	6 and 12 Months
Contrast Sensitivity at Month 6 and Month 12	Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Contrast Sensitivity	6 and 12 Months
Full-field static visual field testing at Month 6 and Month 12	Change from baseline to Month 6 and Month 12 after injection with RTx-015 in the total area in which objects can be seen	6 and 12 Months
Low Vision Quality of Life at Month 6 and Month 12	Change from baseline in low vision quality of life at Month 6 and Month 12. An algorithm will be applied to assess a total score. Higher scores are a worsening of the condition.	6 and 12 Months

Collaborators and Investigators

• Sponsor: Ray Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 27, 2024
• Primary Completion (Estimated): October 1, 2030
• Study Completion (Estimated): October 1, 2030

Study Registration Dates

• First Submitted: June 10, 2024
• First Submitted That Met QC Criteria: June 13, 2024
• First Posted (Actual): June 14, 2024

Study Record Updates

• Last Update Posted (Actual): February 20, 2026
• Last Update Submitted That Met QC Criteria: February 18, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, X-Linked; Uveal Diseases; Retinal Diseases; Retinal Dystrophies; Retinal Degeneration; Choroid Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinitis Pigmentosa; Choroideremia
• Other Study ID Numbers: RTx-015-CP-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No