night blindness tunnel vision
Conditions
Interventions
None listed
Sponsors
NightstaRx Ltd.
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: a. Subject / legal guardian (if applicable) is willing and able to provide informed consent and subject assent for participation in the study. b. Are male and >= 7 years of age. c. Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene d. Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months. e. Have a BCVA in at least 1 eye, as defined below: • ETDRS BCVA >=74 letters (Equivalent to: Snellen 6/9 or 20/32; decimal 0.63; LogMAR 0.2) • ETDRS BCVA 34-73 letters, inclusive (Equivalent to: Snellen 6/12 - 6/60 or 20/40 - 20/200; decimal 0.5 - 0.1; LogMAR 0.3-1.0) Eligibility by BCVA will be divided into these 2 subgroups, with a minimum of 120 eyes in the second subgroup (34-73 letters). f. Mean total retinal sensitivity in at least 1 eye as assessed by microperimetry >=0.1 decibels (dB) and
Exclusion criteria
Exclusion criteria: a. Have a history of amblyopia in the eligible eye. b. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may put the subject at risk because of participation in the study, may influence the results of the study, may influence the subject*s ability to perform study diagnostic tests, or impact the subject*s ability to participate in the study. This includes clinically significant cataracts. c. 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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints of the study are 1) change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) over time. 2) change from Baseline in retinal sensitivity assessed with microperimetry over time. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary endpoints are changes from Baseline in other functional and anatomical measures over time. | — |
Countries
Netherlands
Outcome results
None listed