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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-09-12

Study Completion Date

2030-11-12

Brief Summary

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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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Detailed Description

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Conditions

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Retinitis Pigmentosa

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Group 1: AAV5-hRKp.RPGR Low Dose

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.

Group Type EXPERIMENTAL

AAV5-hRKp.RPGR

Intervention Type GENETIC

AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Group 2: AAV5-hRKp.RPGR High Dose

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 Type EXPERIMENTAL

AAV5-hRKp.RPGR

Intervention Type GENETIC

AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Interventions

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AAV5-hRKp.RPGR

AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Intervention Type GENETIC

AAV5-hRKp.RPGR

AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.

Intervention Type GENETIC

Eligibility Criteria

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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

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No