Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)

Trial details

Who runs it

Ray Therapeutics, Inc.

Trial code

NCT07901751

Record updated

2026. október 28.

Original description

The purpose of this clinical trial is to evaluate the effectiveness and safety of a single injection into the eye of an investigational gene therapy in participants with retinitis pigmentosa (RP).Read full original descriptionShow less

Phase 2/3, Double-masked, Randomized, Sham-controlled, Single Dose Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)

The purpose of this clinical trial is to evaluate the effectiveness and safety of a single injection into the eye of an investigational gene therapy in participants with retinitis pigmentosa (RP).

Inclusion: Adults ≥18 years of age at the time of informed consent; Able and willing to provide written informed consent prior to any study-specific procedures, in accordance with ICH GCP and applicable regulations; Clinical diagnosis of RP, regardless of the underlying genetic mutation; Baseline best corrected distance visual acuity (BCVA) in the study eye of LogMAR 1.0 and 2.7 inclusive (approximately 20/200 - 20/9000), measured monocularly at the ETF by certified, masked personnel using grating visual acuity with conversion to logMAR equivalents; Presence of retinal ganglion cells and/or retinal nerve fiber layer on SD-OCT testing at Screening confirmed by central image reading center; Adequate organ function and general good health.

Exclusion: Participation in another interventional clinical study (ocular or systemic) involving administration of an investigational drug, biologic, or device within 6 months prior to Screening; Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy in either eye; Known or suspected steroid responders whose intraocular pressure could not be adequately controlled with topical pressure-lowering medications after prior use of steroid medications (ocular, periocular, or systemic); Complicating systemic diseases; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or CNS function or confound study outcomes (e.g., prior radiation treatment of the orbit; leukemia with CNS/optic nerve involvement, uncontrolled hypertension, or similar conditions); Cataract or other ocular surgery (including refractive), 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 contraindication to prophylactic steroid regimen specified in the protocol; Presence of any pre-existing ocular condition in either eye that would preclude the planned intravitreal injection, Increase the risk of procedure-related complication, or interfere with interpretation of efficacy or safety outcomes, including hyperreflective loci/foci (HRF) on OCT imaging; Known immunological response dysfunction including immuno-compromising diseases or use of immunosuppressive medications (other than physiologic corticosteroid replacement); Positive infectious disease screen (hepatitis B, C, and/or Human Immunodeficiency Virus [HIV]); Known hypersensitivity 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); Any condition, including cognitive, mobility, or geographical limitations, that would prevent completion of required study visits or ETF assessments; Any condition that, in the opinion of the investigator or medical monitor, makes the participant unsuitable for the study or would interfere with the interpretation of study endpoints; Planned participation in another interventional study during the course of this trial; Current pregnancy or breastfeeding; Weighing less than 40 kg (88 pounds).

ClinicalTrials.gov NCT07901751