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
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/
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
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
