UMIN-CTR Clinical Trial

Public title

Evidence creation through nationwide surveillance of the progeria syndrome Werner syndrome and establishment of a case registration system

Acronym

Werner syndrome registry

Scientific Title

Evidence creation through nationwide surveillance of the progeria syndrome Werner syndrome and establishment of a case registration system

Scientific Title:Acronym

Werner syndrome registry

Region

Japan

Condition

Werner Syndrome

Classification by specialty

Medicine in general	Endocrinology and Metabolism	Geriatrics
Ophthalmology	Dermatology	Oto-rhino-laryngology
Orthopedics	Plastic surgery

Classification by malignancy

Others

Genomic information

YES

Narrative objectives1

In order to solve the clinical questions regarding with the adult progeria, Werner Syndrome, and establish high quality evidence that contributes to the revision of clinical practice guidelines of Werner Syndrome, we have the nationwide survey and establish the Werner Syndrome registration system. By the survey and registration system, we can reveal the current disease profile, the natural history, and prognosis of Werner Syndrome in Japan. We also have a clinical trial for Werner Syndrome. Therefore, we will be able to contribute to the revision of the current clinical practice guideline of Werner Syndrome, which was published based on the result of previous Practical Research Project for Rare Intractable Diseases. We are trying to improve the patient prognosis and supported the patient social reintegration, by the popularization and quality improvement of medical treatment for Werner Syndrome.

Basic objectives2

Others

Basic objectives -Others

(A) Establishment and management of Werner Syndrome registration system
(B) Data collection and analysis of Werner Syndrome

Trial characteristics_1

Trial characteristics_2

Developmental phase

Primary outcomes

(This is a patient registry.)
We will clarify current disease profile, natural history and prognosis of WS in Japan through the results of this research and improve patient data accuracy and use effectively, and improve medical system for WS. We will be able to rescue patients who do not have appropriate medical treatment and support patient's prognosis improvement and reintegration by improving the quality of medical treatment.

Key secondary outcomes

Study type

Others,meta-analysis etc

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

Age-lower limit

Not applicable

Age-upper limit

Not applicable

Gender

Male and Female

Key inclusion criteria

1. Patients whom diagnosed as Werner Syndrome or related diseases.
2. Patients who wrote informed consent.

Key exclusion criteria

N/A

Target sample size

200

Name of lead principal investigator

1st name	Koutaro
Middle name
Last name	Yokote

Organization

Chiba University

Division name

Graduate school of medicine

Zip code

260-8670

Address

1-8-1 Inohana, Chuo-ku, Chiba City, Chiba, Japan

TEL

0432262092

Email

kyokote@faculty.chiba-u.jp

Name of contact person

1st name	Masaya
Middle name
Last name	Koshizaka

Organization

Chiba University

Division name

Graduate school of medicine

Zip code

260-8670

Address

1-8-1 Inohana, Chuo-ku, Chiba City, Chiba, Japan

TEL

0432262092

Homepage URL

http://www.m.chiba-u.jp/class/clin-cellbiol/werner/result/registry.html

Email

overslope@chiba-u.jp

Institute

Ministry of Health, Labour and Welfare

Institute

Department

Personal name

Organization

Ministry of Health, Labour and Welfare

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Chiba University

Address

1-8-1 Inohana, Chuo-ku, Chiba City, Chiba, Japan

Tel

0432227171

Email

igaku-rinri@office.chiba-u.jp

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2017

Year

11

Month

03

Day

URL releasing protocol

Publication of results

Partially published

URL related to results and publications

https://www.aging-us.com/article/202441/text

Number of participants that the trial has enrolled

54

Results

Malignant neoplasms were observed from the fifth decade of life and were observed in approximately 30% of patients during the 3-year survey period. Regarding renal function, the mean estimated glomerular filtration rate calculated from serum creatinine (eGFRcre) and eGFRcys, which were calculated from cystatin C in the first year, were 98.3 and 83.2 mL/min/1.73 m2, respectively, and differed depending on the index used. In longitudinal analysis, the average eGFRcre showing a rapid decline.

Results date posted

2023

Year

05

Month

10

Day

Results Delayed

Results Delay Reason

Date of the first journal publication of results

2020

Year

12

Month

29

Day

Baseline Characteristics

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

No longer recruiting

Date of protocol fixation

2016

Year

01

Month

01

Day

Date of IRB

2016

Year

07

Month

27

Day

Anticipated trial start date

2016

Year

04

Month

01

Day

Last follow-up date

2024

Year

03

Month

31

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

(A) Establishment and management of WS registration system
We prepared a data sheet for WS registration system, based on the previous survey and referring to domestic and international intractable disease registration systems. We have established WS registration system, used DATATRACK ONE (NTT DATA), supported by the Chiba University Clinical Research Center. We also have employed a data manager and developed a registry infrastructure.

(B) Data collection and analysis of WS
We have conducted a nationwide primary survey to find patients who were not diagnosed, by collaboration with the National Health Labor Science Research Policy Research Project. We also have gathered and updated the primary information of WS patients, based on the results of the previous nationwide survey conducted in 2009. We have prepared for mail survey / guidance requesting participation in the WS registration system. We will ask each medical institution to participate in the WS registration system in the next year. We are also seeking cooperation from patients and family associations for the WS registration. We are requesting to acquire informed consent and have first survey of case information and secondary survey, in which we collect the data of clinical symptoms / natural history (course from onset to treatment start), mutation pattern of the causative gene, and treatment contents. We are also collecting and registering blood samples, gene specimens and skin slices for cases that agree to do. We will collect patients data annually, in order to have cross-sectional analysis. We will utilize the data obtained including the longitudinal analysis by follow-up data as evidence for revision of WS clinical practice guidelines (conducted by the laboratory science research intractable disease policy research project).

Registered date

2017

Year

11

Month

03

Day

Last modified on

2023

Year

11

Month

08

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034058