X-Linked Retinitis Pigmentosa
Conditions
Brief summary
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
Interventions
BIOLOGICALAAV5-hRKp.RPGR
Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.
Sponsors
Janssen Research & Development, LLC
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Received AAV5-hRKp.RPGR in the MGT009 Study
Exclusion criteria
* Unwilling or unable to meet with the requirements of the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants | 60 Months | Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA) | Up to 60 Months | Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported. |
| Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO) | Up to 60 Months | Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level. |
| Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score | Up to 60 Months | The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported. |
| Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions | Up to 60 Months | Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported. |
| Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry | Up to 60 months | Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported. |
| Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field | Up to 60 months | Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported. |
| Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field | Up to 60 months | Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported. |
| Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry | Up to 60 months | Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported. |
Countries
United Kingdom, United States
Contacts
STUDY_DIRECTORJanssen Research & Development, LLC Clinical Trial
Janssen Research & Development, LLC
Outcome results
None listed