NCT01829750

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2013

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2013

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

April 9, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 11, 2013

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2016

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2016

Completed
Last Updated

December 7, 2021

Status Verified

November 1, 2021

Enrollment Period

2.9 years

First QC Date

April 9, 2013

Last Update Submit

November 23, 2021

Conditions

Hypoplastic Left Heart SyndromeSingle Right VentricleSingle Left Ventricle

Keywords

Univentricular heart

Outcome Measures

Primary Outcomes (1)

  • Cardiac function

    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.

    3 Months

Secondary Outcomes (1)

  • Cardiac function

    12 Months

Other Outcomes (1)

  • Clinical symptoms

    3 and 12 Months

Study Arms (2)

Control

SHAM COMPARATOR

(Stage 1) No active intervention after standard surgical treatment (Stage 2) Rescuing transplantation by cardiac progenitor cell infusion is applicable in patients, along with their written consent, 4 months after palliations who were assigned as control group in stage 1.

Genetic: Cardiac progenitor cell infusion

Cardiac progenitor cell infusion

ACTIVE COMPARATOR

(Stage 1) single dose, intracoronary infusion of 0.3 million cells/kg cardiac progenitor cells

Genetic: Cardiac progenitor cell infusion

Interventions

Cardiac progenitor cell infusionGENETIC

(Stage 1) Cardiac progenitor cell infusion in patients who assigned as active comparator group (Stage 2) Rescuing transplantation is applicable in patients, along with their written consent, 4 months after palliations who were allocated as control group in stage 1.

Cardiac progenitor cell infusionControl

Eligibility Criteria

AgeUp to 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age: Age is 0 year or more and 20 years or less at the time of enrollment.
  • The patients have functional single ventricular physiology with the indication to have stage-2 or -3 palliative surgeries.
  • The ventricular ejection fraction \<60%.

You may not qualify if:

  • Cardiogenic shock
  • A patient with unstoppable extracorporeal circulation
  • A patient with lethal, uncontrollable arrhythmia
  • A patient with a complication of coronary artery disease
  • A patient with a complication of brain dysfunction due to circulatory failure
  • A patient with malignant neoplasm
  • A patient with a complication of serious neurologic disorder
  • A patient with high-grade pulmonary embolism or pulmonary hypertension
  • A patient with high-grade renal failure
  • A patient with multiple organ failure
  • Active infection (including endocarditis)
  • Sepsis
  • Active hemorrhagic disease (e. g. gastrointestinal bleeding, injury)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Okayama University

Okayama, 700-8558, Japan

Location

Related Publications (8)

  • Oh H, Bradfute SB, Gallardo TD, Nakamura T, Gaussin V, Mishina Y, Pocius J, Michael LH, Behringer RR, Garry DJ, Entman ML, Schneider MD. Cardiac progenitor cells from adult myocardium: homing, differentiation, and fusion after infarction. Proc Natl Acad Sci U S A. 2003 Oct 14;100(21):12313-8. doi: 10.1073/pnas.2132126100. Epub 2003 Oct 6.

    PMID: 14530411BACKGROUND

  • Tateishi K, Ashihara E, Honsho S, Takehara N, Nomura T, Takahashi T, Ueyama T, Yamagishi M, Yaku H, Matsubara H, Oh H. Human cardiac stem cells exhibit mesenchymal features and are maintained through Akt/GSK-3beta signaling. Biochem Biophys Res Commun. 2007 Jan 19;352(3):635-41. doi: 10.1016/j.bbrc.2006.11.096. Epub 2006 Nov 27.

    PMID: 17150190BACKGROUND

  • Tateishi K, Ashihara E, Takehara N, Nomura T, Honsho S, Nakagami T, Morikawa S, Takahashi T, Ueyama T, Matsubara H, Oh H. Clonally amplified cardiac stem cells are regulated by Sca-1 signaling for efficient cardiovascular regeneration. J Cell Sci. 2007 May 15;120(Pt 10):1791-800. doi: 10.1242/jcs.006122.

    PMID: 17502484BACKGROUND

  • Tateishi K, Takehara N, Matsubara H, Oh H. Stemming heart failure with cardiac- or reprogrammed-stem cells. J Cell Mol Med. 2008 Dec;12(6A):2217-32. doi: 10.1111/j.1582-4934.2008.00487.x. Epub 2008 Aug 27.

    PMID: 18754813BACKGROUND

  • Takehara N, Tsutsumi Y, Tateishi K, Ogata T, Tanaka H, Ueyama T, Takahashi T, Takamatsu T, Fukushima M, Komeda M, Yamagishi M, Yaku H, Tabata Y, Matsubara H, Oh H. Controlled delivery of basic fibroblast growth factor promotes human cardiosphere-derived cell engraftment to enhance cardiac repair for chronic myocardial infarction. J Am Coll Cardiol. 2008 Dec 2;52(23):1858-1865. doi: 10.1016/j.jacc.2008.06.052.

    PMID: 19038683BACKGROUND

  • Ishigami S, Ohtsuki S, Eitoku T, Ousaka D, Kondo M, Kurita Y, Hirai K, Fukushima Y, Baba K, Goto T, Horio N, Kobayashi J, Kuroko Y, Kotani Y, Arai S, Iwasaki T, Sato S, Kasahara S, Sano S, Oh H. Intracoronary Cardiac Progenitor Cells in Single Ventricle Physiology: The PERSEUS (Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease) Randomized Phase 2 Trial. Circ Res. 2017 Mar 31;120(7):1162-1173. doi: 10.1161/CIRCRESAHA.116.310253. Epub 2017 Jan 4.

    PMID: 28052915RESULT

  • Hirai K, Sawada R, Hayashi T, Araki T, Nakagawa N, Kondo M, Yasuda K, Hirata T, Sato T, Nakatsuka Y, Yoshida M, Kasahara S, Baba K, Oh H; TICAP/PERSEUS Study Group. Eight-Year Outcomes of Cardiosphere-Derived Cells in Single Ventricle Congenital Heart Disease. J Am Heart Assoc. 2024 Nov 19;13(22):e038137. doi: 10.1161/JAHA.124.038137. Epub 2024 Nov 11.

    PMID: 39526355DERIVED

  • Sano T, Ousaka D, Goto T, Ishigami S, Hirai K, Kasahara S, Ohtsuki S, Sano S, Oh H. Impact of Cardiac Progenitor Cells on Heart Failure and Survival in Single Ventricle Congenital Heart Disease. Circ Res. 2018 Mar 30;122(7):994-1005. doi: 10.1161/CIRCRESAHA.117.312311. Epub 2018 Jan 24.

    PMID: 29367212DERIVED

MeSH Terms

Conditions

Hypoplastic Left Heart SyndromeUniventricular Heart

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Hidemasa Oh, M.D., Ph.D.

    Okayama University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

April 9, 2013

First Posted

April 11, 2013

Study Start

April 1, 2013

Primary Completion

March 1, 2016

Study Completion

September 1, 2016

Last Updated

December 7, 2021

Record last verified: 2021-11

Data Sharing

IPD Sharing
Will not share

Locations

JP(1)