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

NCT02781922 | Recruiting Phase 3 DMC

• 1 other identifier: 001-CLJ-001
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 4

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

Conditions

Hypoplastic Left Heart Syndrome; Single Ventricle

Keywords

cardiac stem/progenitor cells; autologous cell therapy; functional single ventricle; congenital heart disease

Outcome Measures

Primary Outcomes (1)

• Change in ejection fraction (EF(%)) assessed by MRI from baseline

  Baseline, 6 and 12 months

Secondary Outcomes (7)

• Change in EF(%) assessed by echocardiograms from baseline

  Screening, Baseline, 3, 6 and 12 months

• Change in EF(%) assessed by cardiac catheterization from baseline

  Baseline, 6 and 12 months

• Change in Ea/Ees assessed by cardiac catheterization from baseline

  Baseline, 6 and 12 months

• Change in Ventricular Stiffness assessed by cardiac catheterization from baseline

  Baseline, 6 and 12 months

• Change in heart failure index from baseline

  Baseline, 3, 6 and 12 months

Study Arms (2)

Autologous cardiac stem cells (JRM-001)

EXPERIMENTAL

Genetic: Autologous cardiac stem cells (JRM-001)

Usual care

NO INTERVENTION

Interventions

Autologous cardiac stem cells (JRM-001) GENETIC

3x 10\^5 cells/kg, single treatment

Autologous cardiac stem cells (JRM-001)

Eligibility Criteria

• Age: 0 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Metcela Inc.: lead

Study Sites (4)

Kanagawa Children's Medical Center

Kanagawa, Japan

RECRUITING

Okayama University Hospital

Okayama, Japan

RECRUITING

Saitama Prefectural Children's Medical Center

Saitama, Japan

RECRUITING

Shizuoka Children's Hospital

Shizuoka, Japan

RECRUITING

Related Publications (3)

• 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.

  PMID: 25403163 BACKGROUND

• 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.

  PMID: 26232942 BACKGROUND

• 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: 28052915 BACKGROUND

MeSH Terms

Conditions

Hypoplastic Left Heart Syndrome; Univentricular Heart; Heart Defects, Congenital

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Metcela Inc. (former Japan Regenerative Medicine Co., Ltd.)

CONTACT

info@j-rm.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2016
• First Posted: May 25, 2016
• Study Start: June 1, 2016
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: September 30, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

JP (4)