Natural History of the Progression of X-Linked Retinitis Pigmentosa

Natural History of the Progression of X-Linked Retinitis Pigmentosa

The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).

This study was previously posted by NightstaRx Ltd. In October, 2020, sponsorship of the trial was transferred to Biogen.

Key Inclusion Criteria:

1. Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene.

2. Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months

3. Have an ETDRS BCVA in at least 1 eye of ≥34 letters (Equivalent to Snellen ≥ 6/60 or 20/200; decimal 0.1; LogMAR 1.0).

4. Mean retinal sensitivity in the eligible eye as assessed by microperimetry:

   • Males with a mean retinal sensitivity of 68 loci ranging from ≥0.1 decibels (dB) and ≤20 dB.
   • Females with a mean retinal sensitivity of 68 loci ranging from ≥0.1 dB and ≤25 dB.

5. If female, have symptomatic disease with impairment of visual function.

Key Exclusion Criteria:

1. Have a history of amblyopia in the eligible eye.
2. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may put the participant at risk because of participation in the study, may influence the results of the study, may influence the participant's ability to perform study diagnostic tests, or impact the participant's ability to participate in the study. This includes clinically significant cataracts.
3. Have participated in another research study involving an investigational medicinal product in the past 12 weeks or received a gene/cell-based therapy at any time previously (including but not limited to Intelligent Implant System implantation, ciliary neurotrophic factor therapy, nerve growth factor therapy).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply