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| ID | Type | Description | Link |
|---|---|---|---|
| 74765340RPG3001 | Other Identifier | Janssen Research & Development, LLC |
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The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group 1: AAV5-hRKp.RPGR Low Dose | Experimental | Participants will receive bilateral subretinal administration of AAV5-hRKp.RPGR low dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
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| Group 2: AAV5-hRKp.RPGR High Dose | Experimental | Participants will receive bilateral subretinal administration of AAV5 hRKp.RPGR high dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV5-hRKp.RPGR | Genetic | AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Event (AEs) | An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment. | Up to 60 Months |
| Number of Participants with Abnormalities in Clinical Laboratory Assessments | Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported. | Up to 60 Months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52 | Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported. | Baseline - Week 52 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Janssen Pharmaceutical K.K., Japan Clinical Trial | Janssen Pharmaceutical K.K. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Hospital Organization Tokyo Medical Center | Meguro-ku | 1528902 | Japan |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40547876 | Derived | Wang CY, Chen L, Lin TY, Huang SP. Systematic Identification of Candidate Genes for Inherited Retinal Disease Gene Therapy Integrating Worldwide IRD Cohort and Single-Cell Analysis. J Ophthalmol. 2025 Jun 12;2025:7014745. doi: 10.1155/joph/7014745. eCollection 2025. |
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The data sharing policy of Johnson & Johnson Innovative Medicine is available at www.innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu.
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
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| AAV5-hRKp.RPGR | Genetic | AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure. |
|
| Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52 |
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported. |
| Baseline - Week 52 |
| Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52 | Change from baseline in low luminance visual acuity by ETDRS chart letter score in worse-seeing eye at week 52 will be reported. | Baseline - Week 52 |
| D012164 |
| Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |