Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa Patients

Retinitis Pigmentosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06460844
• Other study ID #: RTx-015-CP-101
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: August 2024
• Est. completion date: May 2026

Study information

• Verified date: June 2024
• Source: Ray Therapeutics, Inc.
• Contact: Executive Medical Director
• Phone: +1 858 617 8610
• Email: clinicaltrials[@]raytherapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 9 eligible patients with retinitis pigmentosa will be enrolled sequentially in up to 3 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 12 months.

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. Up to 3 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 9
• Est. completion date: May 2026
• Est. primary completion date: May 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Adults =18 years of age

• Informed consent obtained from the patient

• Clinical diagnosis of Retinitis Pigmentosa independent of 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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

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.

Locations

Country	Name	City	State
United States	Retina Vitreous Associates Medical Group	Beverly Hills	California
United States	UCI Alpha Clinic	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
Ray Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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