Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS)
- Sponsor: NightstaRx, Ltd
- Principal Investigator: Kimberly Stepien, MD
- Study Coordinator: Nickie Stangel
Study objective:
To gain a better understanding of disease progression over time in male subjects with X-linked retinitis pigmentosa (XLRP).
Study design:
The study will include 7 visits over a 24-month period. Study visits will comprise ophthalmic evaluations and a visual function questionnaire.
Inclusion criteria:
- Male patients ≥ 7 years of age
- Documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene
- Best-corrected visual acuity (BCVA) in at least one eye, as defined below:
- BCVA ≥ 74 letters using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts (equivalent to Snellen 20/32)
- BCVA 34-73 letters using ETDRS (equivalent to Snellen 20/40 – 20/200)
- Mean total retinal sensitivity in at least one eye as assessed by microperimetry ≥0.1 decibels (dB) and ≤20 dB
Exclusion criteria:
- History of amblyopia in the eligible eye
- Any other significant ocular or non-ocular disease/disorder which may put the subject at risk because of participation in the study, may influence the results of the study, may influence the subjects ability to perform study diagnostic tests, or impact the subjects ability to participate in the study. This includes clinically significant cataracts.
- Patients who have received gene/cell-based therapy at any time previously or participated in another research study involving an investigations medicinal product in the past 12 weeks.