XOLARIS: A 24-Month, Prospective, Natural History Study of 201 Participants with <i>Retinitis Pigmentosa GTPase Regulator</i>-Associated X-Linked Retinitis Pigmentosa.

ABSTRACT:

Objective

To improve the understanding of the natural disease progression of retinitis pigmentosa GTPase regulator (RPGR)-associated X-linked retinitis pigmentosa (XLRP).

Design

A multicenter, prospective, observational natural history study over 24 months.

Participants

Male participants aged ≥7 years with a pathogenic variant in the RPGR gene, a best-corrected visual acuity (BCVA) score of ≥34 ETDRS letters, and a mean 68-loci retinal sensitivity (assessed by microperimetry) of 0.1 to 20 decibels (dB).

Methods

Participants were divided into subgroups based on their BCVA score at baseline: 34 to 73 (lower BCVA) or ≥74 (higher BCVA) ETDRS letters. There were 7 visits over 24 months.

Main outcome measures

Change from base

SUBMITTER: MacLaren RE 

PROVIDER: S-EPMC11531613 | biostudies-literature | 2025 Jan-Feb

REPOSITORIES: biostudies-literature