Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2013 by Okayama University
Sponsor:
Collaborator:
Translational Research Informatics Center, Kobe, Hyogo, Japan
Information provided by (Responsible Party):
Hidemasa Oh, Okayama University
ClinicalTrials.gov Identifier:
NCT01829750
First received: April 9, 2013
Last updated: April 11, 2013
Last verified: April 2013
  Purpose

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.

Following the safety verification of the Phase I study (TICAP trial) that has been implemented and completed, the efficacy will be verified in this Phase II clinical study. The rationale of this study is based on the animal studies that transplanted cardiac progenitor cells may regenerate damaged myocardium by giving rise to cardiac muscle and vascular cell types. Preclinical and phase I studies indicate the possibilities of cardiac function improvements and reduced myocardial fibrosis by progenitor cell infusion.

The efficacy of cell transplantation is assessed as the primary endpoint of the Phase II clinical study. The improvements of cardiac ejection fraction assessed by echocardiography, ventriculography, and cardiac MRI, which are conducted before and after treatment, are assessed as the evaluation items. The absolute values of the improvement of cardiac ejection fraction obtained by the above three imaging modalities before and after treatment are compared between two groups of the transplantation group and non-transplantation group.


Condition Intervention Phase
Hypoplastic Left Heart Syndrome
Single Right Ventricle
Single Left Ventricle
Genetic: Cardiac progenitor cell infusion
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 2 Study of Intracoronary Infusion of Cardiac Progenitor Cells in Patients With Univentricular Heart Disease

Resource links provided by NLM:


Further study details as provided by Okayama University:

Primary Outcome Measures:
  • Cardiac function [ Time Frame: 3 Months ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Cardiac function [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]
    The secondary outcome measure is to determine the cardiac function improvements by three different imaging modalities at 12 months after treatment.


Other Outcome Measures:
  • Clinical symptoms [ Time Frame: 3 and 12 Months ] [ Designated as safety issue: Yes ]
    Clinical symptoms and BNP levels will be assessed at 3- and 12-months after treatment and compared with control group.


Estimated Enrollment: 34
Study Start Date: April 2013
Estimated Study Completion Date: March 2018
Estimated Primary Completion Date: March 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Sham Comparator: Control

(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

(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.

Active Comparator: Cardiac progenitor cell infusion
(Stage 1) single dose, intracoronary infusion of 0.3 million cells/kg cardiac progenitor cells
Genetic: Cardiac progenitor cell infusion

(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.


  Eligibility

Ages Eligible for Study:   up to 20 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age: Age is 0 year or more and 20 years or less at the time of 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%.

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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01829750

Contacts
Contact: Hidemasa Oh, M.D., Ph.D. +81-086-235-6506 hidemasa@md.okayama-u.ac.jp
Contact: Shunji Sano, M.D., Ph.D +81-086-235-7359 s_sano@cc.okayama-u.ac.jp

Locations
Japan
Okayama University Recruiting
Okayama, Japan, 700-8558
Contact: Hidemasa Oh, M.D., Ph.D.    +81-086-235-6506    hidemasa@md.okayama-u.ac.jp   
Contact: Shunji Sano, M.D., Ph.D.    +81-086-235-7359    s_sano@cc.okayama-u.ac.jp   
Principal Investigator: Hidemasa Oh, M.D., Ph.D.         
Sponsors and Collaborators
Okayama University
Translational Research Informatics Center, Kobe, Hyogo, Japan
Investigators
Principal Investigator: Hidemasa Oh, M.D., Ph.D. Okayama University
  More Information

Additional Information:
Publications:
Responsible Party: Hidemasa Oh, Professor, Okayama University
ClinicalTrials.gov Identifier: NCT01829750     History of Changes
Other Study ID Numbers: 0329-18, MHLW25040101
Study First Received: April 9, 2013
Last Updated: April 11, 2013
Health Authority: Japan: Ministry of Health, Labor and Welfare

Keywords provided by Okayama University:
Univentricular heart

Additional relevant MeSH terms:
Heart Diseases
Hypoplastic Left Heart Syndrome
Cardiovascular Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Congenital Abnormalities

ClinicalTrials.gov processed this record on September 18, 2014