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UMIN-CTR Clinical Trial |
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Name: | UMIN ID: |
Recruitment status | No longer recruiting |
Unique ID issued by UMIN | UMIN000017661 |
Receipt No. | 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 |
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 |
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Condition | |||
Condition | Pompe Disease | ||
Classification by specialty |
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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 | |
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Interventions/Control_4 | |
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Interventions/Control_6 | |
Interventions/Control_7 | |
Interventions/Control_8 | |
Interventions/Control_9 | |
Interventions/Control_10 |
Eligibility | ||||
Age-lower limit |
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Age-upper limit |
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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. |
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Key exclusion criteria | No exclusion criteria in this study. | |||
Target sample size | 150 |
Research contact person | |||||||
Name of lead principal investigator |
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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 |
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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 | |
Institute | Muscular Dystrophy Clinical Trial Network |
Institute | |
Department |
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 |
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Related information | |
URL releasing protocol | |
Publication of results | Unpublished |
Result | |
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Number of participants that the trial has enrolled | |
Results | |
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Baseline Characteristics | |
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Plan to share IPD | |
IPD sharing Plan description |
Progress | |||||||
Recruitment status | No longer recruiting | ||||||
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Anticipated trial start date |
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Date of closure to data entry |
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Date trial data considered complete |
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Date analysis concluded |
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Other | |
Other related information | Prospective observation has finished. |
Management information | |||||||
Registered date |
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Last modified on |
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Link to view the page | |
URL(English) | https://upload.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 |