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| ID | Type | Description | Link |
|---|---|---|---|
| MGT-RPGR-022 | Other Identifier | Janssen Research & Development, LLC | |
| 2020-002255-37 | EudraCT Number | ||
| 2024-511411-25-00 | Registry Identifier | EUCT number |
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A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferred Treatment From MGT-RPGR-021 of Intermediate Dose | Experimental | Deferred treatment |
|
| Already Treated in MGT-RPGR-021 | Experimental | Already treated |
|
| Deferred Treatment From MGT-RPGR-021 Low Dose | Experimental | Deferred treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Genetic: AAV5-hRKp.RPGR Intermediate Dose | Biological | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 - Month 60 |
| Number of Participants With Abnormalities in Laboratory Assessments | Number of participants with abnormalities in laboratory assessments will be assessed. | Day 1 - Month 60 |
| Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment | Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed. | From Baseline - Month 60 |
| Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment | Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed. | From Baseline- Month 60 |
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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 Los Angeles |
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97 participants randomized in MGT-RPGR-021. 64 participants randomized to Immediate treatment arm. 33 participants randomized to the deferred arm. These participants will be treated in MGT-RPGR-022. All participants to be followed for 60 months.
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| Genetic: AAV5-hRKp.RPGR Low Dose | Biological | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
|
|
| 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 Eye and Ear Infirmary | 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 |
| Ghent University Hospital | 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 | Rome | 00184 | 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 |