Trials Sponsors Conditions Drugs Pricing

Conditions

X-Linked Retinitis Pigmentosa (XLRP)

Keywords

XLRPretinal degenerationRPGRadeno-associated virusAAVgene therapy

Outcome Measures

Primary Outcomes (1)

The number and percentage of participants experiencing Grade 3 or higher ocular or non-ocular treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (SAEs), at Month 12
Day 0 - Month 12

Secondary Outcomes (11)

The number and percentage of participants experiencing ocular or non-ocular TEAEs, including treatment-emergent SAEs during the Study period
Day 0-5 years
Change from baseline in low-luminance visual acuity (LLVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity at Month 12
Day 0 - Month 12
Change from baseline in mean sensitivity across the whole grid, as measured by macular integrity assessment (MAIA) microperimetry at Month 12
Day 0 - Month 12
Response, as measured by MAIA microperimetry, where response is defined as a ≥ 7 dB visual sensitivity improvement from baseline in at least 5 loci at Month 12
Day 0 - Month 12
Change from baseline in full-field stimulus threshold (FST) at Month 12
Day 0 - Month 12
+6 more secondary outcomes

Study Arms (1)

Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes

EXPERIMENTAL

On Day 1, participants will undergo a pars plana vitrectomy and receive a central subretinal administration of laruparetigene zovaparvovec in their first treated study eye. For the first six participants, participants will undergo a pars plana vitrectomy followed by a central subretinal administration of laruparetigene zovaparvovec in their second eye between 30 and 60 days after the first surgery. Once the first six study participants have been dosed in both eyes, the interval between dosing of the first and second eye may be reduced to a minimum of 7 days (and up to 14 days) for up to four of the remaining participants enrolled in the study, provided the following conditions are met: * No unexpected safety concerns were observed following dosing of the first eye. * An independent safety review by the Sponsor, Investigator, and DSMC Chair confirms that bilateral dosing may proceed.

Biological: Adeno-associated virus vector expressing a human RPGR gene

Interventions

Adeno-associated virus vector expressing a human RPGR geneBiological

Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501

Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age12 Years - 50 Years

Sexmale(Gender-based eligibility)

Gender Eligibility DetailsMale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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.

Sponsors & Collaborators

Beacon Therapeutics
Industrylead

Study Sites (6)

University of Florida Jacksonville Ophthalmology

Jacksonville, Florida, 32209, United States

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Duke Eye Center

Durham, North Carolina, 27710, United States

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

OHSU Casey Eye Institute

Portland, Oregon, 97239, United States

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

MeSH Terms

Conditions

Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal Diseases

Study Officials

None None
PRINCIPAL INVESTIGATOR

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

September 5, 2025

First Posted

September 16, 2025

Study Start

September 10, 2025

Actual (reported)

Primary Completion

You may not qualify if:

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):
Have a history of oral or genital herpes and be unable and/or unwilling to use a prophylactic antiviral medication.
Have a history of ocular herpes.
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
If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following study drug administration
Have any other condition or reason that, in the opinion of the Investigator, would prevent the participant from completing all study assessments
Have any other condition or reason that, in the opinion of the Investigator, makes the participant unsuitable for the study
Are currently participating or recently participated in any other research protocol involving investigational agents or therapies that, in the opinion of the Investigator, would make the participant unsuitable for the study. Recent participation is defined as participation within 90 days of initial screening for this study OR within 10 half-lives of the investigational drug, whichever is longer
Have previously received any adeno-associated virus (AAV) gene therapy product, stem cell therapy, cell-based therapy, or similar biologics
Have pre-existing eye conditions in either eye that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications (e.g., corneal opacities, diabetic retinopathy, retinal vasculitis, glaucoma, active cystoid macular edema)
Have significant media opacity impacting evaluation of the retina or vitreous in either eye. This includes cataracts considered to be a major contributor to reducing visual acuity and/or if the participant is likely to require cataract extraction within 3 months of study drug administration
+7 more criteria

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (11)

Study Arms (1)

Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (6)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (11)

Study Arms (1)

Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (6)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations