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

Conditions

• X-Linked Retinitis Pigmentosa (XLRP)

Eligibility Criteria

Sex

MALE

Age

12 Years - 50 Years

Healthy Volunteers

Not accepted

Interventions

• Adeno-associated virus vector expressing a human RPGR gene — BIOLOGICAL
  Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501

Study Details

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.

Key Dates

Start date

Sep 10, 2025

Status verified

Feb 2026

Primary completion

Jun 30, 2027

Completion

Dec 15, 2030

Study Design

Enrollment

10 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Participants undergo pars plana vitrectomy and receive a central subretinal administration both eyes
  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.

Primary Outcome Measure

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 [ Time Frame: Day 0 - Month 12 ]

Central Contacts

• Serva Health Serva Health
  855-467-2364
  [email protected]
• Amy Christenson
  [email protected]

Locations (6)

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