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AGTC-501 in X-linked Retinitis Pigmentosa
NCT04850118Phase 3
Key facts
- Status
- Active, not recruiting
- Start
- Mar 2024
- Primary completion
- Jun 2026
- Countries
- 4
- Sponsor
- Beacon Therapeutics
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Conditions
- X-linked retinitis pigmentosa (XLRP)
01
Overview
Phase 3, randomised, single-blind trial of laruparetigene zovaparvovec in X-linked retinitis pigmentosa (XLRP) (3 arms), sponsored by Beacon Therapeutics. Primary endpoint: Proportion of participants with a >=15 letter increase from baseline in Low Luminance Visual Acuity (LLVA) at 12 months. Started Mar 2024; status: active, not recruiting.
02
Design
3 arms- Study design
- RandomizedSingle-blind
Group 1: Dose 1
Male participants treated by subretinal injection with Dose 1 of AGTC-501
Group 2: Dose 2
Male participants treated by subretinal injection with Dose 2 of AGTC-501
Group 3: Control
Untreated control group followed for a minimum of 24 months, with option to receive treatment after Month 12
03
Who it enrols
Male patients aged 12-50 with X-linked retinitis pigmentosa caused by RPGR mutations, with specific visual acuity and retinal sensitivity requirements, excluding those with prior gene/cell therapy or active ocular infections.
04
Endpoints
Primary
Proportion of participants with a >=15 letter increase from baseline in Low Luminance Visual Acuity (LLVA)
Secondary
Change from baseline in Low Luminance Visual Acuity (LLVA)
Change from baseline in mean sensitivity across the whole grid, as measured by MAIA microperimetry
Change from baseline in full-field stimulus threshold (FST)
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