UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS)
Title of the study (Brief title)	Cardiac Progenitor Cell Transfer for Univentricular Heart
Region	Japan

Condition
Condition	Hypoplastic left heart syndrome Single right ventricle Single left ventricle
Classification by specialty	Cardiology Vascular surgery Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease. Patients with preoperative high-risk group or whose cardiac function did not recover postoperatively eventually have no choice other than heart transplantation.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Phase II

Assessment
Primary outcomes	The primary outcome measure is to evaluate the cardiac function improvement by echocardiography, ventriculography, and cardiac MRI, which are conducted before and 3 months after surgical treatment.
Key secondary outcomes	The secondary outcome measure is to determine the cardiac function improvements by three different imaging modalities at 12 months after treatment. Clinical symptoms and BNP levels will be assessed at 3- and 12-months after treatment and compared with control group.

Base
Study type	Interventional

Study design
Basic design	Cross-over
Randomization	Randomized
Randomization unit	Individual
Blinding	Open -no one is blinded
Control	No treatment
Stratification	YES
Dynamic allocation	YES
Institution consideration	Institution is considered as a block.
Blocking	YES
Concealment	Central registration

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Maneuver
Interventions/Control_1	(Stage 1) single dose, intracoronary infusion of 0.3 million cell/kg cardiac progenitor cells (cell infusion group).
Interventions/Control_2	(Stage 2) Rescuing transplantation is applicable in patients, along with their written consent, 4 months after palliations who were assigned as control group in stage 1.
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	20 years-old >=
Gender	Male and Female
Key inclusion criteria	1) Age: Age is 0 year or more and 20 years or less at the time of patient enrollment. 2) The patients have functional single ventricular physiology with the indication to have stage-2 or -3 palliative surgeries. 3) The ventricular ejection fraction <60%.
Key exclusion criteria	1) Cardiogenic shock 2) A patient with unstoppable extracorporeal circulation 3) A patient with lethal, uncontrollable arrhythmia 4) A patient with a complication of coronary artery disease 5) A patient with a complication of brain dysfunction due to circulatory failure 6) A patient with malignant neoplasm 7) A patient with a complication of serious neurologic disorder 8) A patient with high-grade pulmonary embolism or pulmonary hypertension 9) A patient with high-grade renal failure 10) A patient with multiple organ failure 11) Active infection (including endocarditis) 12) Sepsis 13) Active hemorrhagic disease (e. g. gastrointestinal bleeding, injury)
Target sample size	34

Research contact person
Name of lead principal investigator	Hidemasa Oh
Organization	Okayama University Hospital
Division name	Center for Innovative Clinical Medicine
Address	2-5-1 Shikata-cho, Kita-ku, Okayama
TEL	+81-086-235-6506
Email	hidemasa@md.okayama-u.ac.jp

Public contact
Name of contact person	Hidemasa Oh
Organization	Okayama University Hospital
Division name	Center for Innovative Clinical Medicine
Address	2-5-1 Shikata-cho, Kita-ku, Okayama
TEL	+81-086-235-6506
Homepage URL	http://shin-iryo.jp/section/index.html
Email	hidemasa@md.okayama-u.ac.jp

Sponsor
Institute	Okayama University Hospital
Institute
Department

Funding Source
Organization	Okayama University Hospital
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	Translational Research Informatics Center
Name of secondary funｄer(s)	Ministry of Health, labour and Welfare

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	岡山大学病院(岡山市）

Other administrative information
Date of disclosure of the study information	2013 Year 04 Month 10 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2012 Year 12 Month 03 Day
Anticipated trial start date	2013 Year 04 Month 15 Day
Last follow-up date	2016 Year 03 Month 31 Day
Date of closure to data entry	2016 Year 06 Month 30 Day
Date trial data considered complete	2016 Year 08 Month 31 Day
Date analysis concluded	2016 Year 09 Month 30 Day

Related information
URL releasing protocol
Publication of results	Published
URL releasing results
Results
Other related information

Management information
Registered date	2013 Year 04 Month 09 Day
Last modified on	2017 Year 01 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000012219