Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)

Hypoplastic Left Heart Syndrome Clinical Trial

— APOLLON  

Official title:

Efficacy and Safety Study of Autologous Cardiac Stem Cells (JRM-001) Treated After Reconstructive Surgery in Pediatric Patients With Congenital Heart Disease: A Multicenter Randomized Single-blind Parallel-group Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02781922
• Other study ID #: 001-CLJ-001
• Status: Recruiting
• Phase: Phase 3
• Start date: June 2016
• Est. completion date: May 2023

Study information

• Verified date: September 2022
• Source: Metcela Inc.
• Contact: Metcela Inc. (former Japan Regenerative Medicine Co., Ltd.)
• Email: info[@]j-rm.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy and safety of intracoronary injection of JRM-001 after reconstructive surgery in pediatric patients with functional single ventricle

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: May 2023
• Est. primary completion date: May 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 6 Years

Eligibility

Inclusion Criteria:

• Functional single ventricle patient with heart failure who is scheduled for stage 2 (Glenn) or stage 3 (Fontan) surgery
• EF(%) by echocardiography = 55%
• Able to obtain written informed consent of participation in the study by a parent of the patient

Exclusion Criteria:

• Known medical history of cardiogenic shock
• Lethal, uncontrollable arrhythmia
• Complication of coronary artery disease
• Eisenmenger syndrome
• Complication of brain dysfunction due to circulatory failure
• Malignant neoplasm
• Complication of severe neurologic disorder
• Severe pulmonary embolism or pulmonary hypertension
• Severe renal failure
• Multiple organ failure
• Active infection (including endocarditis)
• Sepsis
• Active hemorrhagic disease (e.g. gastrointestinal bleeding, injury)
• Known history of hypersensitivity to anti-infective drugs
• Inability to complete the protocol treatment and baseline to follow-up examinations

Related Conditions & MeSH terms

• Hypoplastic Left Heart Syndrome
• Single Ventricle
• Univentricular Heart

Intervention

Genetic:
• Autologous cardiac stem cells (JRM-001)
3x 10^5 cells/kg, single treatment

Locations

Country	Name	City	State
Japan	Kanagawa Children's Medical Center	Kanagawa
Japan	Okayama University Hospital	Okayama
Japan	Shizuoka Children's Hospital	Shizuoka

Sponsors (1)

Lead Sponsor	Collaborator
Metcela Inc.

Country where clinical trial is conducted

Japan, 

References & Publications (3)

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. — View Citation

Ishigami S, Ohtsuki S, Tarui S, Ousaka D, Eitoku T, Kondo M, Okuyama M, Kobayashi J, Baba K, Arai S, Kawabata T, Yoshizumi K, Tateishi A, Kuroko Y, Iwasaki T, Sato S, Kasahara S, Sano S, Oh H. Intracoronary autologous cardiac progenitor cell transfer in patients with hypoplastic left heart syndrome: the TICAP prospective phase 1 controlled trial. Circ Res. 2015 Feb 13;116(4):653-64. doi: 10.1161/CIRCRESAHA.116.304671. Epub 2014 Nov 17. — View Citation

Tarui S, Ishigami S, Ousaka D, Kasahara S, Ohtsuki S, Sano S, Oh H. Transcoronary infusion of cardiac progenitor cells in hypoplastic left heart syndrome: Three-year follow-up of the Transcoronary Infusion of Cardiac Progenitor Cells in Patients With Single-Ventricle Physiology (TICAP) trial. J Thorac Cardiovasc Surg. 2015 Nov;150(5):1198-1207, 1208.e1-2. doi: 10.1016/j.jtcvs.2015.06.076. Epub 2015 Jul 8. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in ejection fraction (EF(%)) assessed by MRI from baseline		Baseline, 6 and 12 months
Secondary	Change in EF(%) assessed by echocardiograms from baseline		Screening, Baseline, 3, 6 and 12 months
Secondary	Change in EF(%) assessed by cardiac catheterization from baseline		Baseline, 6 and 12 months
Secondary	Change in Ea/Ees assessed by cardiac catheterization from baseline		Baseline, 6 and 12 months
Secondary	Change in Ventricular Stiffness assessed by cardiac catheterization from baseline		Baseline, 6 and 12 months
Secondary	Change in heart failure index from baseline		Baseline, 3, 6 and 12 months
Secondary	Change in Quality of Life (QOL) index from baseline		Baseline, 6 and 12 months
Secondary	Number of adverse events		Up to 12 months