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

April 23, 2024 updated by: Janssen Pharmaceutical K.K.

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)

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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

15

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Japan
      • Tokyo, Japan, 152-8902
        • Recruiting
        • National Hospital Organization Tokyo Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Study population consists of Japanese participants with X-linked retinitis pigmentosa (XLRP) associated with pathogenic variants in retinitis pigmentosa GTPase regulator (RPGR).

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Visual Function
Time Frame: Up to Day 30
Visual function will be assessed using visual acuity.
Up to Day 30
Retinal Structure
Time Frame: Up to Day 30
Retinal structure will be assessed using spectral domain optical coherence tomography (SDOCT).
Up to Day 30
Retinal Function
Time Frame: Up to Day 30
Retinal function will be assessed using static visual field testing.
Up to Day 30

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Janssen Pharmaceutical K.K.

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 26, 2021

Primary Completion (Estimated)

April 22, 2024

Study Completion (Estimated)

April 22, 2024

Study Registration Dates

First Submitted

April 28, 2021

First Submitted That Met QC Criteria

April 28, 2021

First Posted (Actual)

May 3, 2021

Study Record Updates

Last Update Posted (Actual)

April 24, 2024

Last Update Submitted That Met QC Criteria

April 23, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CR108926
  • 74765340RPG0001 (Other Identifier: Janssen Pharmaceutical K.K., Japan)

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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