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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)

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

Fukushima Takashi

Janssen Pharmaceutical K.K.

5-2, Nishi-kanda 3-chome, Chiyoda-ku, Tokyo

DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com

Medical Information Center

Janssen Pharmaceutical K.K.

5-2, Nishi-kanda 3-chome, Chiyoda-ku, Tokyo

+81-120-183-275

DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com

Not Recruiting

Sept. 25, 2023

Interventional

non-randomized controlled trial

double blind

dose comparison control

parallel assignment

treatment purpose

- 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

Both

Retinitis Pigmentosa

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

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

1. 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.
[Time Frame: Up to 60 Months]

2. 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.
[Time Frame: Up to 60 Months]

3. 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.
[Time Frame: Baseline - Week 52]

4. 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.
[Time Frame: Baseline - Week 52]

5. 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.
[Time Frame: Baseline - Week 52]

+81-3-3411-0111

Aug. 01, 2022

Yes

The data sharing policy of Johnson & Johnson Innovative Medicine is available at www.innovativemedicine.jnj.com/our-innovation/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

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