A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants with X-linked Retinitis Pigmentosa

JRCT ID: jRCT2031230141

Registered date:16/06/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: X-Linked Retinitis Pigmentosa
• Date of first enrollment: 25/12/2023
• Target sample size: 4
• Study type: Interventional

Intervention(s)

Group 1:AAV5-hRKp.RPGR Low Dose
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Group 2:AAV5-hRKp.RPGR High Dose
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Outcome(s)

Primary Outcome

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.

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.

Secondary Outcome

Chang 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 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.

Pointwise Response in Full Visual Field at Week 52
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
Week 52
Pointwise response in worse-seeing eye in full visual field at week 52 will be reported.

Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Week 52
Pointwise response in the central 30 degrees visual field at week 52 will be reported.

Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
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 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 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 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.

Key inclusion & exclusion criteria

• Age minimum: >= 5age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Paticipants 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 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
- Has a mean retinal sensitivity of less than or equal to (<=)20 dB by Octopus static perimetry
- Otherwise, healthy participant on the basis of physical examination, medical history, and vital signs, performed at screening. Any abnormalities, must be consistent with the underlying illness in the study population and this determination must be recorded in the participant's source documents and initialed by the investigator
- 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

Exclude 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
- 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
- Has undergone prior retinal surgery involving the macula, vitrectomy, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery)
- History of an ocular implant, with the exception of an intraocular lens
- Uncontrolled hypertension defined as a systolic value greater than or equal to >=140 millimeter of mercury (mm Hg) or diastolic value >=90 mm Hg at screening

Related Information

• Primary Sponsor: Numaguchi Hirotaka
• Secondary ID(s): NCT05926583