Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial) (APOLLON)
Primary Purpose
Hypoplastic Left Heart Syndrome, Single Ventricle
Status
Recruiting
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
Autologous cardiac stem cells (JRM-001)
Sponsored by
About this trial
This is an interventional treatment trial for Hypoplastic Left Heart Syndrome focused on measuring cardiac stem/progenitor cells, autologous cell therapy, functional single ventricle, congenital heart disease
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
Sites / Locations
- Kanagawa Children's Medical CenterRecruiting
- Okayama University HospitalRecruiting
- Shizuoka Children's HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
Autologous cardiac stem cells (JRM-001)
Usual care
Arm Description
Outcomes
Primary Outcome Measures
Change in ejection fraction (EF(%)) assessed by MRI from baseline
Secondary Outcome Measures
Change in EF(%) assessed by echocardiograms from baseline
Change in EF(%) assessed by cardiac catheterization from baseline
Change in Ea/Ees assessed by cardiac catheterization from baseline
Change in Ventricular Stiffness assessed by cardiac catheterization from baseline
Change in heart failure index from baseline
Change in Quality of Life (QOL) index from baseline
Number of adverse events
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02781922
Brief Title
Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Recruiting
Study Start Date
June 2016 (undefined)
Primary Completion Date
May 2023 (Anticipated)
Study Completion Date
May 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Metcela Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypoplastic Left Heart Syndrome, Single Ventricle
Keywords
cardiac stem/progenitor cells, autologous cell therapy, functional single ventricle, congenital heart disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Autologous cardiac stem cells (JRM-001)
Arm Type
Experimental
Arm Title
Usual care
Arm Type
No Intervention
Intervention Type
Genetic
Intervention Name(s)
Autologous cardiac stem cells (JRM-001)
Intervention Description
3x 10^5 cells/kg, single treatment
Primary Outcome Measure Information:
Title
Change in ejection fraction (EF(%)) assessed by MRI from baseline
Time Frame
Baseline, 6 and 12 months
Secondary Outcome Measure Information:
Title
Change in EF(%) assessed by echocardiograms from baseline
Time Frame
Screening, Baseline, 3, 6 and 12 months
Title
Change in EF(%) assessed by cardiac catheterization from baseline
Time Frame
Baseline, 6 and 12 months
Title
Change in Ea/Ees assessed by cardiac catheterization from baseline
Time Frame
Baseline, 6 and 12 months
Title
Change in Ventricular Stiffness assessed by cardiac catheterization from baseline
Time Frame
Baseline, 6 and 12 months
Title
Change in heart failure index from baseline
Time Frame
Baseline, 3, 6 and 12 months
Title
Change in Quality of Life (QOL) index from baseline
Time Frame
Baseline, 6 and 12 months
Title
Number of adverse events
Time Frame
Up to 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Metcela Inc. (former Japan Regenerative Medicine Co., Ltd.)
Email
info@j-rm.com
Facility Information:
Facility Name
Kanagawa Children's Medical Center
City
Kanagawa
Country
Japan
Individual Site Status
Recruiting
Facility Name
Okayama University Hospital
City
Okayama
Country
Japan
Individual Site Status
Recruiting
Facility Name
Shizuoka Children's Hospital
City
Shizuoka
Country
Japan
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
25403163
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.
Results Reference
background
PubMed Identifier
26232942
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.
Results Reference
background
PubMed Identifier
28052915
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.
Results Reference
background
Learn more about this trial
Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)
We'll reach out to this number within 24 hrs