X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene MedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
MeiraGTx UK II Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female. 2. 3 years of age or older. 3. Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by a sponsor-approved laboratory. Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 60 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration. 2. Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule. 3. Has undergone prior retinal surgery involving the macula, vitrectomy, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery). 4. History of an ocular implant, with the exception of an intraocular lens.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the effect of bilateral treatment with AAV5-hRKp.RPGR on retinal function as measured by static perimetry. ;Secondary Objective: To assess changes after treatment administration in retinal function, functional vision, visual function and to assess the safety and tolerability of bilateral subretinal delivery of AAV5-hRKp.RPGR;Primary end point(s): Change in retinal sensitivity by pointwise comparison of individual loci in a 185-point customized grid at Week 52. ;Timepoint(s) of evaluation of this end point: Baseline to week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Retinal Function assessed by Static perimetry: V30 from VFMA modeling Average threshold by microperimetry, under mesopic condition •Functional Vision assessed by Visual mobility assessment. Impact of Vision Impairment on Adults (IVI-A) - Reading and Accessing Information domain score – for adults (age 18 years and older) •Visual Function assessed by Low luminance BCVA by ETDRS chart letter score Contrast sensitivity by Pelli-Robson chart in LogMAR;Timepoint(s) of evaluation of this end point: Baseline to week 52 | — |
Countries
Belgium, Canada, Denmark, France, Germany, Ireland, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactDominic Fitzsimmons
MeiraGTx UK II Limited
Outcome results
None listed