Laruparetigene Zovaparvovec for Retinitis Pigmentosa

(Landscape Trial)

Recruiting at 5 trial locations

Overseen By Jason Phelps

Age: < 65

Sex: Male

Trial Phase: Phase 2

Sponsor: Beacon Therapeutics

Must not be taking: Anticoagulants, Corticosteroids, Immunosuppressives, others

Disqualifiers: Herpes, Immunosuppression, Autoimmunity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 2 months

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, laruparetigene zovaparvovec, to determine if it can safely help individuals with X-linked Retinitis Pigmentosa (XLRP) preserve or improve vision. The treatment is administered through a procedure in both eyes. Suitable candidates for this trial are males diagnosed with XLRP who can undergo eye surgery. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in vision care.

Do I need to stop taking my current medications for the trial?

The trial requires that you stop taking anticoagulant agents (like warfarin or heparin) 7 days before the study drug is given. You also need to stop using systemic corticosteroids or other immunosuppressive medications 3 months before the study drug is given. If you have a history of herpes, you must be willing to use antiviral medication.

Is there any evidence suggesting that laruparetigene zovaparvovec is likely to be safe for humans?

Research has shown that laruparetigene zovaparvovec, also known as AGTC-501, has generally been well-tolerated. In one study, participants handled the treatment well for up to 36 months. Another study found positive results at 9 months, with no major safety issues. These findings suggest the treatment is relatively safe. However, individual experiences can differ, so discussing any concerns with the trial team is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for retinitis pigmentosa, which often focus on managing symptoms or slowing progression, laruparetigene zovaparvovec offers a novel gene therapy approach. This treatment is unique because it involves delivering a modified gene directly to the retinal cells through a subretinal injection, potentially addressing the root genetic cause of the disease. Researchers are excited about laruparetigene zovaparvovec because it has the potential to restore vision at a cellular level, offering hope for significant improvement rather than just symptom management.

What evidence suggests that laruparetigene zovaparvovec might be an effective treatment for retinitis pigmentosa?

Research has shown that laruparetigene zovaparvovec, also known as AGTC-501, may improve vision for people with X-linked retinitis pigmentosa (XLRP). Studies have found early improvements in seeing in low-light conditions and lasting benefits in overall eye sensitivity. Data from participants indicated positive changes in vision over several months, suggesting that the treatment might slow down or even improve vision loss. Most participants tolerated the therapy well, experiencing only minor side effects. These findings offer hope that laruparetigene zovaparvovec could effectively manage XLRP. ^{1 2 3 6 7}

Who Is on the Research Team?

None None

Principal Investigator

Are You a Good Fit for This Trial?

This trial is for male participants with X-Linked Retinitis Pigmentosa, a genetic eye condition that leads to vision loss. Details about who can join or reasons for exclusion are not provided, so interested individuals should contact the study organizers for full eligibility criteria.

Inclusion Criteria

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

Exclusion Criteria

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 a known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications

If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following study drug administration

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo pars plana vitrectomy and receive a central subretinal administration of laruparetigene zovaparvovec in both eyes

2 months

2 surgical visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Long-term follow-up

Participants are monitored for long-term safety and effectiveness

5 years

What Are the Treatments Tested in This Trial?

Interventions

• Laruparetigene Zovaparvovec

Trial Overview

The trial is testing laruparetigene zovaparvovec (AGTC-501), which is an adeno-associated virus vector designed to deliver a normal copy of the RPGR gene into the eyes, potentially preserving or improving vision in patients with XLRP.

How Is the Trial Designed?

Group I: Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Beacon Therapeutics

Lead Sponsor

Trials

13

Recruited

390+

Citations

1.

beacontx.com

beacontx.com/news-and-events/beacon-therapeutics-announces-positive-interim-9-month-results-fromdawn-trial-and-36-month-phase-2-skyline-trial-data-for-laru-zova-in-patients/

Beacon Therapeutics Announces Positive Interim 9+ Month ...

The data demonstrated that laru-zova was generally well-tolerated by SKYLINE participants through month 36 and DAWN participants at 9 months or ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07174726

NCT07174726 | A Phase 2 Open-label Study to Evaluate ...

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, ...

3.

modernretina.com

modernretina.com/view/beacon-therapeutics-announces-positive-topline-data-from-dawn-and-skyline-trials

Beacon Therapeutics announces positive topline data from ...

Data continued to show early improvements in LLVA and early and sustained improvements in mean sensitivity in study eyes, as observed by ...

4.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/beacon-laru-zova-xlrp/

Enrolment concludes in Beacon's trial of laru-zova for XLRP

It is designed to assess the safety, tolerability, and efficacy of the therapy in males with XLRP occurred due to mutations in the retinitis ...

5.

prnewswire.com

prnewswire.com/news-releases/beacon-therapeutics-announces-arvo-2025-presentation-on-preliminary-6-month-data-from-its-phase-2-dawn-trial-of-laru-zova-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-302437302.html

Beacon Therapeutics Announces ARVO 2025 ...

The study will evaluate the proportion of participants with a 15 or more letter increase from baseline in LLVA and additional measures of ...

6.

retinalphysician.com

retinalphysician.com/issues/2025/may/arvo8/

Beacon Reports Interim Data on XLRP Therapy

The study aims to assess 2 dose levels of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously ...

7.

fiercebiotech.com

fiercebiotech.com/biotech/beacons-rare-eye-disease-gene-therapy-signals-vision-function-improvements-early-phase-2

Beacon's rare eye disease gene therapy signals vision ...

The company has so far built a “compelling body” of safety and efficacy data on the drug over five years through three clinical studies, CEO ...