UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000017661
Receipt number R000020406
Scientific Title Pompe disease high risk screening in Japan
Date of disclosure of the study information 2015/05/25
Last modified on 2020/05/26 10:43:01

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Basic information

Public title

Pompe disease high risk screening in Japan

Acronym

PHiRS-J

Scientific Title

Pompe disease high risk screening in Japan

Scientific Title:Acronym

PHiRS-J

Region

Japan


Condition

Condition

Pompe Disease

Classification by specialty

Neurology Pediatrics

Classification by malignancy

Others

Genomic information

YES


Objectives

Narrative objectives1

To unveil prevalence of late-onset Pompe disease (LOPD) in patients with muscular disorders of unclassified muscle weakness and/or high level of creatine kinase.

Basic objectives2

Others

Basic objectives -Others

Epidemiological surveillance of disease prevalence.

Trial characteristics_1

Exploratory

Trial characteristics_2

Others

Developmental phase

Not applicable


Assessment

Primary outcomes

Number of patients who showed decreased GAA activity at secondary test with lymphocyte as well as homozygous mutation of GAA gene ("definitive diagnosis group").

Key secondary outcomes

- Number of patients who did not show decreased GAA activity at secondary test with lymphocyte and/or homozygous mutation of GAA gene ("suspected diagnosis group").
- Difference of clinical information from "definitive diagnosis group", "suspected diagnosis group", and "exclusion group" that did not show decreased GAA activity at primary test.


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

1 years-old <=

Age-upper limit


Not applicable

Gender

Male and Female

Key inclusion criteria

Candidate patient who is eligible for inclusion is under receiving medical check in your hospital (no matter of outpatient and inpatient) and to fulfill following criteria: a)-c)
a) Older than 1 year old.
b) Fulfill following either or both conditions;
b-1) Muscle weakness which is considered clinically due to muscle disorders
b-2) High level of CK which is considered clinically due to muscle disorders
c) There is no clinical feature and medical results that indicate other disorders which shows b-1) and b-2) so far.

Study object must have fulfilled all of inclusion criteria a)-c) and able to obtain informed consent from patient or legally authorized representative for medical test and collecting clinical information in this study.

Key exclusion criteria

No exclusion criteria in this study.

Target sample size

150


Research contact person

Name of lead principal investigator

1st name Katsuhisa
Middle name
Last name Ogata

Organization

National Hospital Organization Higashisaitama National Hospital

Division name

Institute of Clinical Research

Zip code

349-0196

Address

4147 Kurohama, Hasuda, Saitama, Japan

TEL

048-768-1161

Email

ogata-neu@umin.ac.jp


Public contact

Name of contact person

1st name Mikiko
Middle name
Last name Shigemori

Organization

Muscular Dystrophy Clinical Trial Network

Division name

Secretariat Ofifce

Zip code

187-8551

Address

c/o National Center of Neurology and Psychiatry, 4-1-1 Ogawahigashi, Kodaira, Tokyo, Japan

TEL

042-341-2711(3052)

Homepage URL

http://www.mdctn.jp/

Email

shigemori@ncnp.go.jp


Sponsor or person

Institute

Muscular Dystrophy Clinical Trial Network

Institute

Department

Personal name



Funding Source

Organization

Sanofi K. K.

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization

Japan


Other related organizations

Co-sponsor

- National Hospital Organization Higashisaitama Hospital
- National Center of Neurology and Psychiatry
- National Center for Child Health and Development

Name of secondary funder(s)



IRB Contact (For public release)

Organization

Ethics Committee, National Hospital Organization Higashisaitama National Hospital

Address

4147 Kurohama, Hasuda, Saitama, Japan

Tel

048-768-1161

Email

210-cltrials@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

・国立病院機構旭川医療センター(北海道)
・国立病院機構青森病院(青森県)
・国立病院機構仙台西多賀病院(宮城県)
・国立病院機構あきた病院(秋田県)
・国立病院機構東埼玉病院(埼玉県)
・東京女子医科大学病院(東京都)
・国立精神・神経医療研究センター病院(東京都)
・名古屋市立大学病院(愛知県)
・国立病院機構鈴鹿病院(三重県)
・大阪大学医学部附属病院(大阪府)
・国立病院機構刀根山病院(大阪府)
・国立病院機構大牟田病院(福岡県)
・熊本大学医学部附属病院(熊本県)
・奈良県立医科大学附属病院(奈良県)
・国立病院機構下志津病院(千葉県)
・国立病院機構長良医療センター(岐阜県)


Other administrative information

Date of disclosure of the study information

2015 Year 05 Month 25 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

2014 Year 07 Month 16 Day

Date of IRB

2014 Year 07 Month 16 Day

Anticipated trial start date

2015 Year 06 Month 01 Day

Last follow-up date

2018 Year 08 Month 31 Day

Date of closure to data entry

2018 Year 08 Month 31 Day

Date trial data considered complete

2018 Year 08 Month 31 Day

Date analysis concluded

2020 Year 08 Month 31 Day


Other

Other related information

Prospective observation has finished.


Management information

Registered date

2015 Year 05 Month 24 Day

Last modified on

2020 Year 05 Month 26 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020406


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name