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

Korábbi kezelés

• 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.

Más betegség

• 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.

Egyéb

• Planned participation in another interventional study during the course of this trial.
• Current pregnancy or breastfeeding.
• Weighing less than 40 kg (88 pounds)

• 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