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

X-Linked Retinitis Pigmentosa Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05926583
• Other study ID #: CR109143
• Secondary ID: 74765340RPG3001
• Status: Recruiting
• Phase: Phase 3
• Start date: September 12, 2023
• Est. completion date: October 9, 2029

Study information

• Verified date: June 2024
• Source: Janssen Pharmaceutical K.K.
• Contact: Study Contact
• Phone: 844-434-4210
• Email: Participate-In-This-Study[@]its.jnj.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 4
• Est. completion date: October 9, 2029
• Est. primary completion date: September 16, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

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

Locations

Country	Name	City	State
Japan	National Hospital Organization Tokyo Medical Center	Meguro-ku

Sponsors (1)

Lead Sponsor	Collaborator
Janssen Pharmaceutical K.K.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Up to 60 Months
Primary	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.	Up to 60 Months
Secondary	Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees (MRS10) Excluding Scotoma in Static Perimetry at Week 52	Change from baseline in MRS10 excluding scotoma in static perimetry at week 52 will be reported.	Baseline - Week 52
Secondary	Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma of Worse-seeing Eye in Static Perimetry at Week 52	Change from baseline in MRS10 excluding scotoma of worse-seeing eye in static perimetry at week 52 will be reported.	Baseline - Week 52
Secondary	Pointwise Response in Full Visual Field at Week 52	Pointwise response in full visual field at week 52 will be reported.	At Week 52
Secondary	Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52	Pointwise response in worse-seeing eye in full visual field at week 52 will be reported.	At Week 52
Secondary	Pointwise Response in the Central 30 Degrees Visual Field at Week 52	Pointwise response in the central 30 degrees visual field at week 52 will be reported.	At Week 52
Secondary	Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52	Pointwise response in worse-seeing eye in the central 30 degrees visual field at week 52 will be reported.	At Week 52
Secondary	Change From Baseline in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry at 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.	Baseline - Week 52
Secondary	Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) in Patient Reported Outcome - Extreme Lighting Domain Score at Week 52	Change from baseline in mLLQ in patient reported outcome - extreme lighting domain score at week 52 will be reported.	Baseline - Week 52
Secondary	Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52	Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported.	Baseline - Week 52
Secondary	Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52	Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported.	Baseline - Week 52
Secondary	Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at 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.	Baseline - Week 52