CompletedNot Applicable

Natural History of the Progression of X-Linked Retinitis Pigmentosa

United States201 participantsStarted 2017-09-13

Plain-language summary

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

Who can participate

Age range7 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene.
✓. Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months
✓. 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).
✓. Mean retinal sensitivity in the eligible eye as assessed by microperimetry:
✓. If female, have symptomatic disease with impairment of visual function.

Exclusion criteria

✕. Have a history of amblyopia in the eligible eye.
✕. 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.
✕. 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).

What they're measuring

Change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) Best-corrected Visual Acuity (BCVA)

Timeframe: Up to Month 24

Change from Baseline in Retinal Sensitivity Assessed with Microperimetry

Timeframe: Up to Month 24

Trial details

NCT IDNCT04926129

SponsorNightstaRx Ltd, a Biogen Company

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2022-12-16

View on ClinicalTrials.gov More X-Linked Retinitis Pigmentosa trials