UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000044475
Receipt number R000050807
Scientific 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)
Date of disclosure of the study information 2021/06/20
Last modified on 2022/12/12 15:29:36

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


Basic information

Public title

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

Acronym

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

Scientific 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)

Scientific Title:Acronym

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)

Region

Japan


Condition

Condition

X-Linked Retinitis Pigmentosa

Classification by specialty

Ophthalmology

Classification by malignancy

Others

Genomic information

YES


Objectives

Narrative objectives1

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.

Basic objectives2

Others

Basic objectives -Others

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.

Trial characteristics_1


Trial characteristics_2


Developmental phase



Assessment

Primary outcomes

Visual Function
Up to Day 30
Visual function will be assessed using visual acuity.

Retinal Structure
Up to Day 30
Retinal structure will be assessed using spectral domain optical coherence tomography (SDOCT).

Retinal Function
Up to Day 30
Retinal function will be assessed using static visual field testing.

Key secondary outcomes



Base

Study type

Observational


Study design

Basic design


Randomization


Randomization unit


Blinding


Control


Stratification


Dynamic allocation


Institution consideration


Blocking


Concealment



Intervention

No. of arms


Purpose of intervention


Type of intervention


Interventions/Control_1


Interventions/Control_2


Interventions/Control_3


Interventions/Control_4


Interventions/Control_5


Interventions/Control_6


Interventions/Control_7


Interventions/Control_8


Interventions/Control_9


Interventions/Control_10



Eligibility

Age-lower limit

5 years-old <=

Age-upper limit


Not applicable

Gender

Male and Female

Key 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

Key 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

Target sample size

15


Research contact person

Name of lead principal investigator

1st name Hirotaka
Middle name
Last name Numaguchi

Organization

Janssen Pharmaceutical K.K.

Division name

R&D Div. Clinical Science Div. Clinical Development Dept. Medical Group

Zip code

101-0065

Address

3-5-2 Nishikanda, Chiyoda-ku, Tokyo, Japan

TEL

0120-183-275

Email

DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com


Public contact

Name of contact person

1st name Personnel
Middle name
Last name Medical Information Center

Organization

Janssen Pharmaceutical K.K.

Division name

Medical Information Center

Zip code

101-0065

Address

3-5-2 Nishikanda, Chiyoda-ku, Tokyo, Japan

TEL

0120-183-275

Homepage URL


Email

DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com


Sponsor or person

Institute

Janssen Pharmaceutical K.K.

Institute

Department

Personal name



Funding Source

Organization

Janssen Pharmaceutical K.K.

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization



Other related organizations

Co-sponsor


Name of secondary funder(s)



IRB Contact (For public release)

Organization

Tokyo Medical Center Ethical Committee

Address

2-5-1 Higashigaoka, Meguro-ku, Tokyo, Japan

Tel

03-3411-0111

Email

215-rinri@mail.hosp.go.jp


Secondary IDs

Secondary IDs

NO

Study ID_1


Org. issuing International ID_1


Study ID_2


Org. issuing International ID_2


IND to MHLW



Institutions

Institutions



Other administrative information

Date of disclosure of the study information

2021 Year 06 Month 20 Day


Related information

URL releasing protocol


Publication of results

Unpublished


Result

URL related to results and publications


Number of participants that the trial has enrolled


Results


Results date posted


Results Delayed


Results Delay Reason


Date of the first journal publication of results


Baseline Characteristics


Participant flow


Adverse events


Outcome measures


Plan to share IPD


IPD sharing Plan description



Progress

Recruitment status

No longer recruiting

Date of protocol fixation

2020 Year 09 Month 10 Day

Date of IRB

2021 Year 03 Month 26 Day

Anticipated trial start date

2021 Year 07 Month 30 Day

Last follow-up date

2023 Year 08 Month 03 Day

Date of closure to data entry


Date trial data considered complete


Date analysis concluded



Other

Other related information

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


Management information

Registered date

2021 Year 06 Month 08 Day

Last modified on

2022 Year 12 Month 12 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000050807


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name