Clinical trial NCT05926583: A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
Official title: Phase 3 Study to Evaluate the Safety and Efficacy of AAV5-hRKp.RPGR for the Treatment of Japanese X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: X-Linked Retinitis Pigmentosa
Lead sponsor: Janssen Pharmaceutical K.K.
Sponsor class: INDUSTRY
First posted: 2023-07-03
Enrollment: 4
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants with Adverse Event (AEs)
timeFrame: Up to 60 Months
measure: Number of Participants with Abnormalities in Clinical Laboratory Assessments
timeFrame: Up to 60 Months
Secondary outcomes:
measure: Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10) Excluding Scotoma in Static Perimetry at Week 52
timeFrame: Baseline - Week 52
measure: Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma of Worse-seeing Eye in Static Perimetry at Week 52
timeFrame: Baseline - Week 52
measure: Pointwise Response in Full Visual Field at Week 52
timeFrame: At Week 52
measure: Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
timeFrame: At Week 52
measure: Pointwise Response in the Central 30 Degrees Visual Field at Week 52
timeFrame: At Week 52
measure: Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
timeFrame: At Week 52
measure: Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at Week 52
timeFrame: Baseline - Week 52
measure: Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) in Patient Reported Outcome - Extreme Lighting Domain Score at Week 52
timeFrame: Baseline - Week 52
measure: Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52
timeFrame: Baseline - Week 52
measure: Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52
timeFrame: Baseline - Week 52
measure: Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52
timeFrame: Baseline - Week 52
Other outcomes:
Locations:
city: Meguro-ku
country: Japan
facility: National Hospital Organization Tokyo Medical Center
Study officials:
affiliation: Janssen Pharmaceutical K.K.
name: Janssen Pharmaceutical K.K., Japan Clinical Trial
role: STUDY_DIRECTOR
References: