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
Interventions
Product Name: AAV2/5-hRKp.RPGR
Pharmaceutical Form: Solution for injection
Sponsors
MeiraGTx UK II Ltd
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Inclusion in the study will be limited to individuals who meet the following criteria: • Are able to give informed consent or assent, with or without the guidance of their parent/guardian where appropriate • Received AAV2/5-hRKp.RPGR by sub retinal administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-003967-21) • Are willing to adhere to the protocol and long-term follow-up Are the trial subjects under 18? yes Number of subjects for this age range: 18 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): The primary outcome is longer term safety of subretinal administration of AAV2/5-hRKp.RPGR (administered in the XLRP RPGR clinical trial).;Timepoint(s) of evaluation of this end point: At 24, 36, 48 and 60 months after AAV2/5-hRKp.RPGR administration;Main Objective: The primary research objective is to assess the longer term safety of AAV2/5-hRKp.RPGR administered to participants in the RPGR trial, measured by the presence or absence of adverse events, the assessment of visual acuity, and loss of light perception.;Secondary Objective: The secondary research objective is to explore the longer-term efficacy of AAV2/5-hRKp.RPGR in improving visual and retinal function, and quality of life, and protecting against sight impairment from retinal degeneration. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: At 24, 36, 48 and 60 months after AAV2/5-hRKp.RPGR administration; Secondary end point(s): The secondary outcomes are measures of the longer-term efficacy of the original intervention, which will be performed on an individual participant basis and will be descriptive in nature. Efficacy will be assessed at several time points between 18 months and 5 years after the intervention: 1) Slowing or halting of progressive deterioration in retinal structure or visual function that is greater than the test-retest variation for that test and is sustained for at least two consecutive assessments. Slowing or halting of progression over time may be facilitated by comparing identical structural and functional assessments acquired prior to intervention/injection to better determine rate of change over time in this slowly progressive disease. 2) Any improvement in visual function from baseline that is greater than the test-retest variation for that test and is sustained for at least two consecutive assessments. 3) Any improvement in retinal function from pre-intervention that is greater than test-retest variation and measurable by electrophysiology (pattern ERG, multifocal ERG or full-field ERG). 4) Quality of life will be measured by the Impact of Visual Impairment (IVI) questionnaire and the EQ5D-5L and EQ-5D-Y | — |
Countries
United Kingdom, United States
Contacts
Public ContactJulie Bakobaki
MeiraGTx UK II Ltd
Outcome results
None listed