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| ID | Type | Description | Link |
|---|---|---|---|
| MGT-RPGR-021 | Other Identifier | Janssen Research & Development, LLC | |
| 2020-002873-88 | EudraCT Number |
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A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental - Immediate Treatment | Experimental | Intermediate dose. |
|
| Deferred Treatment | Other | Deferred Treatment |
|
| Experimental Immediate Treatment | Experimental | Low dose. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Genetic: AAV5-hRKp.RPGR | Biological | Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze | Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze. | From Baseline to 52 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52 | Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed. | From Baseline to Week 52 |
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Inclusion Criteria:
Exclusion Criteria:
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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 |
|---|---|---|---|---|---|---|
| Shiley Eye Institute Jacobs Retina Center | La Jolla | California | 92093 0946 | United States | ||
| Childrens Hospital |
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Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
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No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
|
| Genetic: AAV5-hRKp.RPGR | Biological | No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
| Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52 | Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed. | From Baseline to Week 52 |
| Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52 | Pointwise response in full visual field at Week 52 will be assessed. | From Baseline to Week 52 |
| Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52 | Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed. | From Baseline to Week 52 |
| Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52 | Pointwise response in the central 30 degrees visual field at Week 52 will be assessed. | From Baseline to Week 52 |
| Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52 | Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed. | From Baseline to Week 52 |
| Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52 | Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed. | From Baseline to Week 52 |
| Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52 | Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52. | From Baseline to Week 52 |
| Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52 | Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52. | From Baseline to Week 52 |
| Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52 | Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52. | From Baseline to Week 52 |
| Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52 | Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52. | From Baseline to Week 52 |
| Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52 | Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52. | From Baseline to Week 52 |
| Number of Participants with Ocular and Non-ocular Adverse Events | Number of participants with ocular and non-ocular adverse events will be assessed. | Day 1 - Week 52 |
| Number of Participants With Abnormalities in Laboratory Assessments | Number of participants with abnormalities in laboratory assessments will be assessed. | Day 1 - 52 Weeks |
| Los Angeles |
| California |
| 90027 |
| United States |
| Stanford Health Care | Palo Alto | California | 94303 | United States |
| VitreoRetinal Associates, PA | Gainesville | Florida | 32607 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Massachusetts General Hospital - Center for Celiac Research and Treatment | Boston | Massachusetts | 02114 | United States |
| Univ of Michigan Medical Center | Ann Arbor | Michigan | 48105 | United States |
| Duke Eye Center | Durham | North Carolina | 27705 | United States |
| University of Pittsburgh Medical Center (UPMC) | Pittsburgh | Pennsylvania | 15213 | United States |
| Retina Consultants of Houston | Bellaire | Texas | 77401 | United States |
| UZ Gent | Ghent | 9000 | Belgium |
| Hospital For Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Rigshospitalet Glostrup | Glostrup Municipality | 2600 | Denmark |
| Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | Paris | 75012 | France |
| Hadassah Medical Center | Jerusalem | 91120 | Israel |
| Azienda Ospedaliero Universitaria Careggi | Florence | 50134 | Italy |
| Ospedale San Paolo | Milan | 20142 | Italy |
| Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli | Naples | 80131 | Italy |
| IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS | Roma | 00198 | Italy |
| VUMC Amsterdam | Amsterdam | 1105AZ | Netherlands |
| Radboudumc | Nijmegen | 6525EX | Netherlands |
| Hosp Univ Fund Jimenez Diaz | Madrid | 28040 | Spain |
| University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical | Basel | 4031 | Switzerland |
| Universite de Lausanne, Hopital ophtalmique Jules-Gonin | Lausanne | 1004 | Switzerland |
| NHS Lothian | Edinburgh | EH3 9HA | United Kingdom |
| Gartnavel General Hospital | Glasgow | G12 0YN | United Kingdom |
| St James University Hospital | Leeds | LS9 7TF | United Kingdom |
| Moorfields Eye Hospital | London | EC1V 2PD | United Kingdom |