UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Development of induced pluripotent stem (iPS) cells from patients with muscular disorders for the study of the pathogenesis of the diseases and development of therapies
Title of the study (Brief title)	iPS cell studies of muscular disorders
Region	Japan

Condition
Condition	Skeletal muscle disease Myotonic dystrophy Muscular dystrophies
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To generate induced pluripotent (iPS) stem cells derived from patients with muscular disorders and to use them to determine molecular mechanisms and to develop novel treatments.
Basic objectives2	Others
Basic objectives -Others	Basic pathomechanistic study
Trial characteristics_1
Trial characteristics_2
Developmental phase	Not applicable

Assessment
Primary outcomes	Phenotypes of cell lines derived from muscular disorders
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Either of the following subjects suffering from muscular disorders: 1) Subjects scheduled to undergo surgery or biopsy for medical reasons. 2) Subjects from which cultured cells was harvested with previous biopsy and gave consent for the research use of the biopsied samples.
Key exclusion criteria	None
Target sample size	20

Research contact person
Name of lead principal investigator	Masanori P. Takahashi
Organization	Osaka University Graduate School of Medicine
Division name	Department of Neurology
Address	2-2 Yamadaoka, Suita, Osaka JAPAN
TEL	06-6879-3571
Email	mtakahas@neurol.med.osaka-u.ac.jp

Public contact
Name of contact person	Masanori P. Takahashi
Organization	Osaka University Graduate School of Medicine
Division name	Department of Neurology
Address	2-2 Yamadaoka, Suita, Osaka JAPAN
TEL	06-6879-3571
Homepage URL
Email	mtakahas@neurol.med.osaka-u.ac.jp

Sponsor
Institute	Osaka University Graduate School of Medicine
Institute
Department

Funding Source
Organization	MHLW
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor	The National Center of Neurology and Psychiatry
Name of secondary funｄer(s)

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	大阪大学大学院医学系研究科（大阪府） 国立精神・神経医療研究センター（東京都）

Other administrative information
Date of disclosure of the study information	2012 Year 09 Month 23 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2011 Year 09 Month 09 Day
Anticipated trial start date	2011 Year 09 Month 12 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information	Non-Probability Sample

Management information
Registered date	2012 Year 09 Month 22 Day
Last modified on	2018 Year 03 Month 31 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000010201