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A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa | Clinical Trials | Clareo Health

RECRUITINGPHASE2

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

NCT07174726•Beacon Therapeutics

View on ClinicalTrials.gov

Summary

The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.

Detailed Description

XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision. The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.

Eligibility

Age

12 - 50 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•Provide written informed consent or assent (per local regulation) prior to the conduct of any study-related procedures. 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 providing informed consent and assent (as applicable)
•Be male (XY chromosome) and have at least 1 documented pathogenic or likely pathogenic variant in the RPGR gene, within exons 1-14 and/or ORF15, from an appropriately certified or accredited laboratory
•Have a clinical diagnosis of XLRP
•Be in good general health to withstand subretinal surgery and perioperative medications based on a complete physical examination and results from hematology, chemistry, and coagulation analyses performed at screening
•Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend all study visits
•If the participant has a parent or caregiver, the parent or caregiver must be able to follow study instructions, comply with the protocol, and attend study visits with the participant, as required
•Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200) in each eye based on an ETDRS chart at each screening visit. The ETDRS letter score is the main visual acuity inclusion criterion for participants. Participants unable to read the ETDRS letters may use a tumbling "E" chart for the BCVA assessments
•Have an LLVA ≤ 64 letters (approximately Snellen, 20/50) in both eyes based on an ETDRS chart at each screening visit. Participants unable to read the ETDRS letters may use a tumbling "E" chart for the LLVA assessments
•Have an LLD of \>10 letters
+3 more criteria

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 drug or the quality of the assessments
•For participants with herpes simplex virus (HSV):
•1. Have a history of oral or genital herpes and be unable and/or unwilling to use a prophylactic antiviral medication.
•2. Have a history of ocular herpes.
•3. Have active oral or genital herpes or are currently receiving treatment for active HSV infection
•Have complicating systemic diseases (e.g., medical conditions causing immunosuppression, autoimmunity, active systemic infection) that would preclude the gene transfer or ocular surgery if not adequately managed or treated
•Have a known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications
•Have used anticoagulant agents that may alter coagulation (e.g., warfarin, heparin, apixaban, or high-dose docosahexaenoic acid \[fish oil\]) within 7 days prior to study drug administration (ibuprofen, aspirin, or similar agents are acceptable
•Have received any vaccination/immunization within 60 days prior to Day 1 and/or during screening with the exception of the influenza vaccine, which is only exclusionary if received within 28 days prior to Day 1
•Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to study drug administration. Corticosteroids used on an as-needed basis administered by insufflation, inhalation, or local administration to the skin and mucosa such as Symbicort® (budesonide/formoterol), Flonase® (fluticasone propionate), and skin creams and ointments containing corticosteroids shall not be exclusionary
+14 more criteria

Locations (6)

University of Florida Jacksonville Ophthalmology

Jacksonville, Florida, United States

Bascom Palmer Eye Institute

Miami, Florida, United States

Duke Eye Center

Durham, North Carolina, United States

Cincinnati Eye Institute

Cincinnati, Ohio, United States

OHSU Casey Eye Institute

Portland, Oregon, United States

Retina Foundation of the Southwest

Dallas, Texas, United States

Key Dates

Start Date

September 10, 2025

Primary Completion Date

June 30, 2027

Completion Date

December 15, 2030

Last Updated

February 19, 2026

Enrollment

ESTIMATED participants

Conditions

X-Linked Retinitis Pigmentosa (XLRP)

Interventions

Adeno-associated virus vector expressing a human RPGR gene

BIOLOGICAL

Contacts

Serva Health Serva Health

CONTACT

855-467-2364 ProviderSupport@scenictrials.com

Amy Christenson

CONTACT

medical@beacontx.com

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCT06492850

X-Linked Retinitis Pigmentosa (XLRP)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 19, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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