NCT06460844

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)

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Ray Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Retinitis Pigmentosa
• Choroideremia

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2024-08-27
• Primary completion: 2030-10-01
• Completion: 2030-10-01
• First posted: 2024-06-14
• Last updated: 2026-02-20

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study