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An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa

Sponsor
Janssen Pharmaceutical K.K. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04868916
Collaborator
(none)
15
Enrollment
1
Location
20.1
Anticipated Duration (Months)
0.7
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of the study is to identify a cohort of Japanese participants with X-linked retinitis pigmentosa (XLRP) associated with pathogenic variants in the retinitis pigmentosa GTPase regulator (RPGR) gene and to investigate their associated phenotype.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    15 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Prospective Observational Study to Estimate the Genotype and Phenotype of Japanese Patients With X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR)
    Actual Study Start Date :
    Jul 26, 2021
    Anticipated Primary Completion Date :
    Feb 28, 2023
    Anticipated Study Completion Date :
    Mar 31, 2023

    Arms and Interventions

    Arm Intervention/Treatment
    Participants with X-Linked Retinitis Pigmentosa (XLRP)

    Participants with confirmed diagnosis of XLRP associated with pathogenic variants in retinitis pigmentosa GTPase regulator (RPGR) in the Japanese population will be enrolled in the study and the data will be collected and observed. The primary data source for this study will be the medical records of each participant.

    Outcome Measures

    Primary Outcome Measures

    1. Visual Function [Up to Day 30]

      Visual function will be assessed using visual acuity.

    2. Retinal Structure [Up to Day 30]

      Retinal structure will be assessed using spectral domain optical coherence tomography (SDOCT).

    3. Retinal Function [Up to Day 30]

      Retinal function will be assessed using static visual field testing.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    5 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Have RPGR-associated retinal dystrophy

    • Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate

    • Are able to undertake age-appropriate clinical assessments as specified in the protocol

    Exclusion Criteria:

    • Are unable or unwilling to undertake consent or clinical testing

    • Participated in another research study and had intraocular surgery within 3 months of screening

    • Significant ophthalmologic diseases

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Hospital Organization Tokyo Medical Center Tokyo Japan 152-8902

    Sponsors and Collaborators

    • Janssen Pharmaceutical K.K.

    Investigators

    • Study Director: Janssen Pharmaceutical K.K., Japan Clinical Trial, Janssen Pharmaceutical K.K.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Janssen Pharmaceutical K.K.
    ClinicalTrials.gov Identifier:
    NCT04868916
    Other Study ID Numbers:
    • CR108926
    • 74765340RPG0001
    First Posted:
    May 3, 2021
    Last Update Posted:
    Aug 17, 2022
    Last Verified:
    Aug 1, 2022
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Retinitis
    Retinitis Pigmentosa

    Study Results

    No Results Posted as of Aug 17, 2022