Eye gene therapy for retinitis pigmentosa with a single RTx-015 injection

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2/3, double-masked study enrolling about 60 adults with retinitis pigmentosa. Participants will be randomized to receive a lower dose RTx-015, a higher dose RTx-015, or a sham injection in one study eye and followed for 12 months. The study uses a randomized, 1:1:1 design with masked participants and investigators to assess safety and visual outcomes. Inclusion requires confirmed RP, specific baseline vision ranges, and certain eye structure criteria; key exclusions include recent interventional ocular studies. The investigational therapy is RTx-015 delivered by intravitreal injection.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* Participation in another interventional clinical study (ocular or systemic) involving administration of an investigational drug, biologic, or device within 6 months prior to Screening
* Planned participation in another interventional study during the course of this trial.
* Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy in either eye
* 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 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).
* 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 \[HIV\]).
* 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 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)
* Known contraindication to prophylactic steroid regimen specified in the protocol.
* Current pregnancy or breastfeeding.
* Weighing less than 40 kg (88 pounds)
* 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.

Where it's running

• RayTx Clinical Site
  Bakersfield, California, United States Recruiting
• RayTx Clinical Site
  Beverly Hills, California, United States Recruiting
• RayTx Clinical Site
  Oak Park, Illinois, United States Recruiting
• RayTx Clinical Site
  Greensboro, North Carolina, United States Recruiting
• RayTx Clinical Site
  Portland, Oregon, United States Recruiting
• RayTx Clinical Site
  Mt. Pleasant, South Carolina, United States Recruiting

Study contacts

• Study coordinator: Executive Medical Director
• Email: clinicaltrials@raytherapeutics.com
• Phone: 858-617-8610

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.