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| ID | Type | Description | Link |
|---|---|---|---|
| MGT010 | Other Identifier | Janssen Research & Development, LLC | |
| 2018-000425-31 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Follow up cohort | no intervention follow up study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV5-hRKp.RPGR | Biological | Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up. |
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| Measure | Description | Time Frame |
|---|---|---|
| Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants | Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported. | 60 Months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA) | Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported. | Up to 60 Months |
| Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO) |
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Inclusion Criteria:
Exclusion Criteria:
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Males receiving AAV5-hRKp.RPGR in the MGT009 study
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| Name | Affiliation | Role |
|---|---|---|
| Janssen Research & Development, LLC Clinical Trial | Janssen Research & Development, LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | United States | ||
| Univ of Michigan Medical Center |
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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. |
| Up to 60 Months |
| Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score | The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported. | Up to 60 Months |
| Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions | Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported. | Up to 60 Months |
| Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry | Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported. | Up to 60 months |
| Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field | Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported. | Up to 60 months |
| Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field | Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported. | Up to 60 months |
| Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry | Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported. | Up to 60 months |
| Ann Arbor |
| Michigan |
| 48105 |
| United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Leeds Teaching Hospitals NHS Trust | Leeds | LS9 7TF | United Kingdom |
| Moorfields Eye Hospital | London | EC1V 9EL | United Kingdom |