Clinical trial NCT02781922: Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)
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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Hypoplastic Left Heart Syndrome
Single Ventricle
Keywords: cardiac stem/progenitor cells
autologous cell therapy
functional single ventricle
congenital heart disease
Lead sponsor: Metcela Inc.
Sponsor class: INDUSTRY
First posted: 2016-05-25
Enrollment: 40
Sex: ALL
Minimum age (years): 0
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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


Eligibility criteria: 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

Primary outcomes:
measure: Change in ejection fraction (EF(%)) assessed by MRI from baseline
timeFrame: Baseline, 6 and 12 months
Secondary outcomes:
measure: Change in EF(%) assessed by echocardiograms from baseline
timeFrame: Screening, Baseline, 3, 6 and 12 months
measure: Change in EF(%) assessed by cardiac catheterization from baseline
timeFrame: Baseline, 6 and 12 months
measure: Change in Ea/Ees assessed by cardiac catheterization from baseline
timeFrame: Baseline, 6 and 12 months
measure: Change in Ventricular Stiffness assessed by cardiac catheterization from baseline
timeFrame: Baseline, 6 and 12 months
measure: Change in heart failure index from baseline
timeFrame: Baseline, 3, 6 and 12 months
measure: Change in Quality of Life (QOL) index from baseline
timeFrame: Baseline, 6 and 12 months
measure: Number of adverse events
timeFrame: Up to 12 months
Other outcomes:
Locations:
city: Kanagawa
country: Japan
facility: Kanagawa Children's Medical Center
city: Okayama
country: Japan
facility: Okayama University Hospital
city: Shizuoka
country: Japan
facility: Shizuoka Children's Hospital
Study officials:
References:
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.
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.
citation: 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.