Infusing a child's own heart stem cells during single-ventricle surgery (APOLLON)

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

This trial will try injecting a child's own cardiac stem/progenitor cells into the coronary arteries after Glenn or Fontan surgery to help children with a functional single ventricle and reduced ejection fraction.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 0 Years to 6 Years
• Sex: All
• Sponsor: Metcela Inc. (industry)
• Locations: 4 sites (Kanagawa and 3 other locations)
• Trial ID: NCT02781922 on ClinicalTrials.gov

What this trial studies

This is a phase 3 interventional study testing intracoronary delivery of autologous cardiac stem/progenitor cells (JRM-001) given after reconstructive surgery in pediatric patients with a functional single ventricle. Eligible patients are those scheduled for stage 2 (Glenn) or stage 3 (Fontan) surgery with echocardiographic ejection fraction ≤55% and parental consent. The intervention uses each patient’s own harvested cardiac cells, expanded and infused into the coronary circulation, with follow-up to monitor heart function and safety outcomes. The trial is being conducted at multiple pediatric cardiac centers in Japan.

Who should consider this trial

Good fit: Children with a functional single ventricle who are scheduled for Glenn or Fontan surgery, have an ejection fraction ≤55%, and whose parent can provide written informed consent are the intended candidates.

Not a fit: Patients with severe comorbid conditions listed as exclusions (for example active infection, multi-organ failure, severe pulmonary hypertension, lethal arrhythmia, malignant neoplasm, or severe renal failure) or those not meeting the timing or EF criteria are unlikely to benefit.

Why it matters

Potential benefit: If successful, the treatment could improve ventricular function and reduce heart-failure-related complications, potentially delaying the need for transplant and improving quality of life.

How similar studies have performed: Smaller pilot and adult studies of autologous cardiac cell therapies have shown mixed but sometimes promising signals, while large randomized phase 3 evidence in single-ventricle congenital heart disease remains limited.

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

Where this trial is running

Kanagawa and 3 other locations

• 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)

Study contacts

• Study coordinator: Metcela Inc. (former Japan Regenerative Medicine Co., Ltd.)

Conditions: Hypoplastic Left Heart Syndrome, Single Ventricle, cardiac stem/progenitor cells, autologous cell therapy, functional single ventricle, congenital heart disease