🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Clinical Trials in the USA / NCT06333249
Active, not recruiting Phase 2

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

NCT06333249 · tracked via the Priya Life Science USA tracker
Sponsor
Beacon Therapeutics
Phase
Phase 2
Started
2021-04-13
Last updated
2024-08-12

Condition(s) studied

X-Linked Retinitis Pigmentosa

Investigational drug(s) / intervention(s)

rAAV2tYF-GRK1-RPGR

rAAV2tYF-GRK1-RPGR: Adeno-associated virus vector expressing a human RPGR gene

Study summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Eligibility

Sex
MALE
Min age
8 Years
Max age
50 Years
Healthy volunteers
No
Inclusion Criteria: * Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Subjects who provide assent must have a parent, guardian, or legal representative provide written informed consent. * Be between 8 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable). * Be male and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene * Have a clinical diagnosis of XLRP. * Have a BCVA no better than 75 letters and no worse than 35 letters based on an ETDRS chart at each screening visit. * Be able to perform all tests of visual and retinal function and structure in both eyes based on the subject's reliability, and fixation, per the investigator's discretion. * Have detectable baseline mean macular sensitivity measured by (MAIA) microperimetry, as determined by the investigator and confirmed by the Central Reading Center (CRC). * Have detectable EZ line in both eyes as assessed by SD-OCT and confirmed by the CRC. Exclusion Criteria: * Have other known disease-causing mutations documented in the subject'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 subjects with herpes simplex virus (HSV): 1. Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication. 2. Have a history of ocular herpes. 3. Have active oral or genital herpes or are currently receiving treatment for HSV infection. * Have complicating systemic diseases (e.g., medical conditions causing immunosuppression, active systemic infection) that would preclude the gene transfer or ocular surgery. * Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications. * Have used anti-coagulant agents that may alter coagulation * Have received any vaccination/immunization within 28 days prior to screening and/or during screening with the exception of the influenza vaccine, which is only exclusionary if they have received the influenza vaccine within 28 days prior to randomization. * Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. * Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics. Ocular Exclusion Criteria (Either Eye): * Have pre-existing eye conditions that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications * Have significant media opacity impacting evaluation of the retina or vitreous. * Had intraocular surgery within 90 days of study treatment administration. * Have any active ocular/intraocular infection or inflammation * Have a history of steroid-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 OCT images. * Have any history of rhegmatogenous retinal detachment. * Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of \>30 mm if PI deems it appropriate to measure) or presence of pathologic myopia in the study eye. * Have passed the Low Contrast Ora-VNC™ mobility course in either eye or binocularly at any screening visit.

Primary outcome measure(s)

Trial sites (6)

FacilityCityRegionStatus
University of Florida Jacksonville Florida
Boston Children's Hosptial Boston Massachusetts
Cincinnati Eye Institute Cincinnati Ohio
Cleveland Clinic Cleveland Ohio
Casey Eye Institute Portland Oregon
Retina Foundation of the Southwest Dallas Texas

More Beacon Therapeutics trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06333249 on ClinicalTrials.gov ↗ ← All trials in the USA