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Active, Not Recruiting

NCT Number: NCT05926583

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

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).

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Age range

5 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

National Hospital Organization Tokyo Medical Center

Meguro-ku, 1528902, Japan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AAV5-hRKp.RPGR

Genetic

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

Primary outcomes

  1. Number of Participants with Adverse Event (AEs)

    Time frame: 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.

  2. Number of Participants with Abnormalities in Clinical Laboratory Assessments

    Time frame: Up to 60 Months

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

Secondary outcomes

  1. Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment at Week 52

    Time frame: Baseline - Week 52

    Change from baseline in low luminance visual acuity by ETDRS chart score in monocular assessment at week 52 will be reported.

  2. Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Monocular Assessment at Week 52

    Time frame: Baseline - Week 52

    Change from baseline in BCVA by ETDRS chart letter score in monocular assessment at week 52 will be reported.

  3. Change From Baseline in Low Luminance Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Letter Score in Worse-seeing Eye at Week 52

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Janssen Pharmaceutical K.K.

Industry

Registry information

Official study 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)

Important dates

Study start
2023
Primary completion
2027
Study completion
2030
First posted
Jul 3, 2023
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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