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)
Verified date | May 2024 |
Source | Janssen Pharmaceutical K.K. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
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 |
Country | Name | City | State |
---|---|---|---|
Japan | National Hospital Organization Tokyo Medical Center | Tokyo |
Lead Sponsor | Collaborator |
---|---|
Janssen Pharmaceutical K.K. |
Japan,
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 |
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