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

Hypoplastic Left Heart Syndrome Clinical Trial

Official title:

Phase 2 Study of Intracoronary Infusion of Cardiac Progenitor Cells in Patients With Univentricular Heart Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01829750
• Other study ID #: 0329-18
• Secondary ID: MHLW25040101
• Status: Completed
• Phase: Phase 2
• Start date: April 2013
• Est. completion date: September 2016

Study information

• Verified date: November 2021
• Source: Okayama University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: September 2016
• Est. primary completion date: March 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 20 Years

Eligibility

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)

Related Conditions & MeSH terms

• Heart Diseases
• Hypoplastic Left Heart Syndrome
• Single Left Ventricle
• Single Right Ventricle
• Univentricular Heart

Intervention

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.

Locations

Country	Name	City	State
Japan	Okayama University	Okayama

Sponsors (2)

Lead Sponsor	Collaborator
Okayama University	Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

Country where clinical trial is conducted

Japan, 

References & Publications (6)

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 P — View Citation

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. Epub 2003 Oct 6. — View Citation

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. — View Citation

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. Epub 2006 Nov 27. — View Citation

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. — View Citation

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. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Clinical symptoms	Clinical symptoms and BNP levels will be assessed at 3- and 12-months after treatment and compared with control group.	3 and 12 Months
Primary	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	Cardiac function	The secondary outcome measure is to determine the cardiac function improvements by three different imaging modalities at 12 months after treatment.	12 Months

External Links

•   Center for Innovative Clinical Medicine