A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

A regiszterben nem jelölt feltételek

• General Inclusion Criteria:
• Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
• Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).
• Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene.
• Have a clinical diagnosis of XLRP.
• Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend study visits for the duration of the study.
  • Ocular Inclusion Criteria (Study Eye):
• Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)
• Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye
• Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability, and fixation, in the study eye per the Investigator's discretion.
• Have an LLD of > 10 letters in the study eye
• Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, between 1-12 decibels (dB) in the study eye, as determined by the Investigator and confirmed by the CRC with fixation loss ≤20% at each screening visit.
• Have a detectable sub-foveal EZ line in the study eye as assessed by spectral domain-optical coherence tomography (SD-OCT) and confirmed by the CRC.
  • General Exclusion Criteria:
• Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel, that in the opinion of the Investigator would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.
• For participants with herpes simplex virus (HSV):
  • Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.
  • Have a history of ocular herpes.
  • Have active oral or genital herpes or are currently receiving treatment for HSV infection.
• Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
• Have used anti-coagulant agents that may alter coagulation
• Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. Corticosteroids used on an as-needed basis administered by insufflation, inhalation or local administration to the skin
• If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following treatment administration.
• Are currently participating or recently participated in any other research
• Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
• Have significant media opacity impacting evaluation of the retina or vitreous. administration.
• Had intraocular surgery within 90 days of study treatment administration.
• Have any active ocular/intraocular infection or inflammation
• Have a history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
• Have any artificial retinal implant or prosthesis.
• Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images.
• Have any history of rhegmatogenous retinal detachment.
• Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of >30 mm if the Principal Investigator [PI] deems it appropriate to measure) or presence of pathologic myopia in the study eye.
• Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level in either eye or binocularly at any screening visit.