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

Primary Purpose

X-Linked Retinitis Pigmentosa

Status
Recruiting
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
AAV5-hRKp.RPGR
AAV5-hRKp.RPGR
Sponsored by
Janssen Pharmaceutical K.K.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for X-Linked Retinitis Pigmentosa

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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

Sites / Locations

  • National Hospital Organization Tokyo Medical CenterRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Group 1: AAV5-hRKp.RPGR Low Dose

Group 2: AAV5-hRKp.RPGR High Dose

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Number of Participants with Adverse Event (AEs)
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
Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.

Secondary Outcome Measures

Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry
Mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.
Change In Retinal Sensitivity by Pointwise Comparison in Full Visual Field at Week 52
Change in retinal sensitivity by pointwise comparison in full visual field at Week 52 will be reported.
Change in Retinal Sensitivity by Pointwise Comparison in the Central 30 Degrees Visual field at Week 52
Change in retinal sensitivity by pointwise comparison in the central 30 degrees visual field at Week 52 will be reported.
Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at Week 52
Mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry at Week 52 will be reported.
Low Luminance Questionnaire (LLQ) in Patient Reported Outcome - Extreme Lighting Domain score at Week 52
LLQ in patient reported outcome - extreme lighting domain score at Week 52 will be reported.
Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Binocular Assessment at Week 52
Low luminance visual acuity by ETDRS chart score in binocular assessment at Week 52 will be reported.
Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Binocular Assessment at Week 52
BCVA by ETDRS chart letter score in binocular assessment at Week 52 will be reported.

Full Information

First Posted
June 23, 2023
Last Updated
October 10, 2023
Sponsor
Janssen Pharmaceutical K.K.
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1. Study Identification

Unique Protocol Identification Number
NCT05926583
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 12, 2023 (Actual)
Primary Completion Date
September 14, 2024 (Anticipated)
Study Completion Date
May 10, 2029 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Janssen Pharmaceutical K.K.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-Linked Retinitis Pigmentosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Non-Randomized
Enrollment
4 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group 1: AAV5-hRKp.RPGR Low Dose
Arm Type
Experimental
Arm Description
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.
Arm Title
Group 2: AAV5-hRKp.RPGR High Dose
Arm Type
Experimental
Arm Description
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.
Intervention Type
Genetic
Intervention Name(s)
AAV5-hRKp.RPGR
Intervention Description
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
Intervention Type
Genetic
Intervention Name(s)
AAV5-hRKp.RPGR
Intervention Description
AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure.
Primary Outcome Measure Information:
Title
Number of Participants with Adverse Event (AEs)
Description
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.
Time Frame
Up to 60 Months
Title
Number of Participants with Abnormalities in Clinical Laboratory Assessments
Description
Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.
Time Frame
Up to 60 Months
Secondary Outcome Measure Information:
Title
Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (MRS10) in Static Perimetry
Description
Mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.
Time Frame
At Week 52
Title
Change In Retinal Sensitivity by Pointwise Comparison in Full Visual Field at Week 52
Description
Change in retinal sensitivity by pointwise comparison in full visual field at Week 52 will be reported.
Time Frame
At Week 52
Title
Change in Retinal Sensitivity by Pointwise Comparison in the Central 30 Degrees Visual field at Week 52
Description
Change in retinal sensitivity by pointwise comparison in the central 30 degrees visual field at Week 52 will be reported.
Time Frame
At Week 52
Title
Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at Week 52
Description
Mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry at Week 52 will be reported.
Time Frame
At Week 52
Title
Low Luminance Questionnaire (LLQ) in Patient Reported Outcome - Extreme Lighting Domain score at Week 52
Description
LLQ in patient reported outcome - extreme lighting domain score at Week 52 will be reported.
Time Frame
At Week 52
Title
Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Binocular Assessment at Week 52
Description
Low luminance visual acuity by ETDRS chart score in binocular assessment at Week 52 will be reported.
Time Frame
At Week 52
Title
Best Corrected Visual Acuity (BCVA) by ETDRS Chart Letter Score in Binocular Assessment at Week 52
Description
BCVA by ETDRS chart letter score in binocular assessment at Week 52 will be reported.
Time Frame
At Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Contact
Phone
844-434-4210
Email
Participate-In-This-Study@its.jnj.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Janssen Pharmaceutical K.K., Japan Clinical Trial
Organizational Affiliation
Janssen Pharmaceutical K.K.
Official's Role
Study Director
Facility Information:
Facility Name
National Hospital Organization Tokyo Medical Center
City
Meguro-ku
ZIP/Postal Code
1528902
Country
Japan
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Learn more about this trial

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

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