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

NCT06460844 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: RTx-015-CP-101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 4

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.

Conditions

Retinitis Pigmentosa; Choroideremia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Best Corrected Visual Acuity (BCVA) at Month 6 and Month 12

  6 and 12 Months

• Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12

  6 and 12 Months

• Multi luminance mobility at Month 6 and Month 12

  6 and 12 Months

• Contrast Sensitivity at Month 6 and Month 12

  6 and 12 Months

• Full-field static visual field testing at Month 6 and Month 12

  6 and 12 Months

Study Arms (4)

Low Dose RTx-015

EXPERIMENTAL

Single intravitreal injection of low dose RTx-015

Genetic: RTx-015

Middle Dose RTx-015

EXPERIMENTAL

Single intravitreal injection of middle dose RTx-015

Genetic: RTx-015

High Dose RTx-015

EXPERIMENTAL

Single intravitreal injection of high dose RTx-015

Genetic: RTx-015

Higher Dose RTx-015

EXPERIMENTAL

Single intravitreal injection of higher dose RTx-015

Genetic: RTx-015

Interventions

RTx-015 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; Higher Dose RTx-015; Low Dose RTx-015; Middle Dose RTx-015

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ray Therapeutics, Inc.: lead

Study Sites (4)

Retina Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

UCI Alpha Clinic

Orange, California, 92868, United States

Location

UPMC Vision Institute

Pittsburgh, Pennsylvania, 15221, United States

Location

Retina Consultants of Texas Research Centers

Bellaire, Texas, 77401, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Choroideremia

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Choroid Diseases; Uveal Diseases; Genetic Diseases, X-Linked

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2024
• First Posted: June 14, 2024
• Study Start: August 27, 2024
• Primary Completion (Estimated): October 1, 2030
• Study Completion (Estimated): October 1, 2030
• Last Updated: February 20, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)