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

X-Linked Retinitis Pigmentosa Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04868916
• Other study ID #: CR108926
• Secondary ID: 74765340RPG0001
• Status: Completed
• Start date: July 26, 2021
• Est. completion date: April 23, 2024

Study information

• Verified date: May 2024
• Source: Janssen Pharmaceutical K.K.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: April 23, 2024
• Est. primary completion date: April 23, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Locations

Country	Name	City	State
Japan	National Hospital Organization Tokyo Medical Center	Tokyo

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