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

NCT ID: NCT06460844

Last Updated: 2025-06-13

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-27
• Study Completion Date: 2030-10-31

Brief 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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Low Dose RTx-015

Single intravitreal injection of low dose RTx-015

Group Type: EXPERIMENTAL

RTx-015

Intervention Type: GENETIC

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.

Middle Dose RTx-015

Single intravitreal injection of middle dose RTx-015

Group Type: EXPERIMENTAL

RTx-015

Intervention Type: GENETIC

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.

High Dose RTx-015

Single intravitreal injection of high dose RTx-015

Group Type: EXPERIMENTAL

RTx-015

Intervention Type: GENETIC

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.

Higher Dose RTx-015

Single intravitreal injection of higher dose RTx-015

Group Type: EXPERIMENTAL

RTx-015

Intervention Type: GENETIC

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.

Interventions

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.

Intervention Type: GENETIC

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ray Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Retina Vitreous Associates Medical Group

Beverly Hills, California, United States

Site Status: RECRUITING

UCI Alpha Clinic

Orange, California, United States

Site Status: RECRUITING

UPMC Vision Institute

Pittsburgh, Pennsylvania, United States

Site Status: RECRUITING

Retina Consultants of Texas Research Centers

Bellaire, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Executive Medical Director

Role: CONTACT

clinicaltrials@raytherapeutics.com

+1 858 617 8610

Facility Contacts

David Boyer, MD

Role: primary

310-289-2478 ext. 3

UCI Alpha Clinic

Role: primary

alphaclinic@hs.uci.edu

(949) 824-3990

1622 Locust Street, 5th floor

Role: primary

412-642-7888

Jaclyn Henderson, MS, CCRP

Role: primary

jaclyn.henderson@retinaconsultantstexas.com

713.524.3434 ext. 1461

Other Identifiers

RTx-015-CP-101

Identifier Type: -

Identifier Source: org_study_id