A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
- Conditions
- X-Linked Retinitis Pigmentosa
- Interventions
- Genetic: AAV5-hRKp.RPGR
- Registration Number
- NCT05926583
- Lead Sponsor
- Janssen Pharmaceutical K.K.
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 4
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group 2: AAV5-hRKp.RPGR High Dose AAV5-hRKp.RPGR 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. Group 1: AAV5-hRKp.RPGR Low Dose AAV5-hRKp.RPGR 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.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52 At Week 52 Pointwise response in worse-seeing eye in the central 30 degrees visual field 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.
Pointwise Response in the Central 30 Degrees Visual Field at Week 52 At Week 52 Pointwise response in the central 30 degrees visual field at week 52 will be reported.
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) in Patient Reported Outcome - Extreme Lighting Domain Score at Week 52 Baseline - Week 52 Change from baseline in mLLQ in patient reported outcome - extreme lighting domain score at week 52 will be reported.
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10) Excluding Scotoma in Static Perimetry at Week 52 Baseline - Week 52 Change from baseline in MRS10 excluding scotoma in static perimetry at week 52 will be reported.
Pointwise Response in Full Visual Field at Week 52 At Week 52 Pointwise response in full visual field at week 52 will be reported.
Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52 At Week 52 Pointwise response in worse-seeing eye in full visual field at week 52 will be reported.
Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at Week 52 Baseline - Week 52 Change from baseline in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry at week 52 will be reported.
Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma of Worse-seeing Eye in Static Perimetry at Week 52 Baseline - Week 52 Change from baseline in MRS10 excluding scotoma of worse-seeing eye in static perimetry at week 52 will be reported.
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 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.
Trial Locations
- Locations (1)
National Hospital Organization Tokyo Medical Center
🇯🇵Meguro-ku, Japan