Hypoplastic Left Heart Syndrome, Single Ventricle
Conditions
Keywords
cardiac stem/progenitor cells, autologous cell therapy, functional single ventricle, congenital heart disease
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
Interventions
GENETICAutologous cardiac stem cells (JRM-001)
3x 10\^5 cells/kg, single treatment
Sponsors
Metcela Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
0 Years to 6 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in ejection fraction (EF(%)) assessed by MRI from baseline | Baseline, 6 and 12 months |
Secondary
| Measure | Time frame |
|---|---|
| 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 EF(%) assessed by echocardiograms from baseline | Screening, Baseline, 3, 6 and 12 months |
| Change in Quality of Life (QOL) index from baseline | Baseline, 6 and 12 months |
| Number of adverse events | Up to 12 months |
| Change in heart failure index from baseline | Baseline, 3, 6 and 12 months |
Countries
Japan
Contacts
Primary ContactMetcela Inc. (former Japan Regenerative Medicine Co., Ltd.)
Outcome results
None listed