Retinitis Pigmentosa
Conditions
Brief summary
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).
Interventions
GENETICAAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
Sponsors
Janssen Pharmaceutical K.K.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants who are Japanese male or female aged 5 years or older * Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with pathogenic or likely pathogenic variants in the retinitis pigmentosa guanosine triphosphatase regulator(RPGR) gene * Has evidence of preserved retinal function as defined by a mean retinal sensitivity of greater than or equal to (\>=) 2 decibel (dB) by Octopus static perimetry and evidence of preserved outer retinal structure (namely the presence of discernible ellipsoid zone) as determined by spectral domain-optical coherence tomography (SD-OCT) in both eyes * Otherwise, healthy participant on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel or hematology outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator
Exclusion criteria
* Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the AAV5-hRKp.RPGR administration * Is unable to perform the imaging assessments as required (for example: reliable static perimetry \[reliability factor less than or equal to {\<=}19\], optical coherence tomography \[OCT\], or fundus autofluorescence \[FAF\]). * Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Abnormalities in Clinical Laboratory Assessments | Up to 60 Months | Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported. |
| Number of Participants with Adverse Event (AEs) | Up to 60 Months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52 | Baseline - Week 52 | Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported. |
| Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52 | Baseline - Week 52 | Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported. |
| Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52 | Baseline - 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. |
Countries
Japan
Contacts
STUDY_DIRECTORJanssen Pharmaceutical K.K., Japan Clinical Trial
Janssen Pharmaceutical K.K.
Outcome results
None listed