UMIN-CTR Clinical Trial

Basic information
Public title	exploratory study for children severe cardiomyopathy using YS0001
Acronym	exploratory study for children severe cardiomyopathy using YS0001
Scientific Title	exploratory study for children severe cardiomyopathy using YS0001
Scientific Title:Acronym	exploratory study for children severe cardiomyopathy using YS0001
Region	Japan

Condition
Condition	Severe Cardiomyopathy in Pediatric patietns
Classification by specialty	Cardiology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Safety and efficacy study of autologous myoblast sheet for children Severe cardiomyopasthy
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Primary endopoint is to assess the safty
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Culture of autologous skeletal myoblasts and engineering myoblast sheets. Transplantation of myoblast sheets to heart
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	18 years-old >=
Gender	Male and Female
Key inclusion criteria	Severe myopathy NYHA Class iii Ejection fraction =<35%]
Key exclusion criteria	Cardiomyopathy without other surgical options persisitent NYHA Class =>iii HF symptoms refractory to maximal medical therapy Ejection fraction =<35%
Target sample size	3

Research contact person
Last name of lead principal investigator	1st name Middle name Last name Yoshiki Sawa
Organization	Osaka University hospital
Division name	Cardiovascular Surgery
Zip code
Address	2-15 Yamadaoka Suita Osaka Japan
TEL	06-6879-3154
Email	sawa@surg1.med.osaka-u.ac.jp

Public contact
1st name of contact person	1st name Middle name Last name Masaki Taira
Organization	Osaka University hospital
Division name	Cardiovascular Surgery
Zip code
Address	2-15 Yamadaoka Suita Osaka Japan
TEL	06-6879-3154
Homepage URL
Email	taira@surg1.med.osaka-u.ac.jp

Sponsor
Institute	Department of Cardiovascular Surgery, Graduate School of medicine, Osaka University
Institute
Department

Funding Source
Organization	Japan Agency for medical research and development
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

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

Other administrative information
Date of disclosure of the study information	2015 Year 05 Month 01 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	Completed
Date of protocol fixation	2014 Year 05 Month 01 Day
Date of IRB	2016 Year 02 Month 05 Day
Anticipated trial start date	2016 Year 04 Month 01 Day
Last follow-up date	2018 Year 10 Month 12 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2014 Year 12 Month 10 Day
Last modified on	2019 Year 05 Month 07 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000016520