A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy
| Registry | NCT03129568, acronym TICAP-DCM |
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| Status | Completed |
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| Phase | Phase 1 |
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| Study type | Interventional; na, single group, masking: none, treatment |
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| Enrollment | 5 (Actual) |
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| Sponsor | Okayama University (Other); collaborators: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan |
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| Conditions | Dilated Cardiomyopathy |
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| Interventions | CDC infusion (Biological) |
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| Dates | start 2017-04-14, primary completion 2018-09-17, completion 2020-12-09, first posted 2017-04-26, last updated 2021-11-26 |
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| Locations | 1 site in Japan |
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-Summary
A phase 1 prospective study to determine the procedural feasibility and safety and preliminary efficacy of intracoronary infusion of cardiosphere-derived cells (CDCs) in patients with dilated cardiomyopathy.
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+Detailed description
Five consecutive patients will be enrolled to a phase 1 study to verify the procedural feasibility and safety with the CDC infusion group.
+Arms and interventions
| Arm | Type | Interventions | Description |
|---|
| CDC infusion | Experimental | Biological: CDC infusion | CDCs infusion by coronary intervention. |
- CDC infusion (Biological): Injection of CDCs (0.3 million per kg of body weight).
+Outcome measures
Primary outcomes
- Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs. [6 months after CDC treatment]: Assessment of major adverse cardiac events include death, sustained/symptomatic ventricular tachycardia, aggravation of heart failure, acute coronary syndrome, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.
Secondary outcomes
- Change in Ejection Fraction [6 months after protocol treatment]: To determine the changes in cardiac function by ejection fraction with cardiac MRI evaluation in CDC-treated participants from baseline to 6 months of follow up.
+Eligibility
Sex: All, ages: any to 17 Years
Inclusion Criteria:
* Patients diagnosed as dilated cardiomyopathy.
* Patients aged under 18 years old.
* Cardiac ejection fraction < 40%.
Exclusion Criteria:
* Contradiction to cardiac magnetic resonance imaging.
* Cardiogenic shock.
* A patient with unstoppable extracorporeal circulation.
* A patient with lethal, uncontrollable arrhythmia.
* A patient with a complication of coronary artery disease.
* A patient with a complication of brain dysfunction due to circulatory failure.
* A patient with malignant neoplasm.
* A patient with a complication of a serious neurologic disorder.
* A patient with high-grade pulmonary embolism or pulmonary hypertension.
* A patient with high-grade renal failure.
* A patient with multiple organ failure.
* Active infection (including endocarditis).
* Sepsis.
* Active hemorrhagic disease (e.g. gastrointestinal bleeding, injury).
+Posted results
Participant flow
| Milestone | CDC Infusion |
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| Started | 5 |
| Completed | 5 |
| Not completed | 0 |
Outcome results
- Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs. (Primary), Count of participants, Participants: CDC Infusion 1
- Change in Ejection Fraction (Secondary), Mean, percentage of ejection fraction: CDC Infusion 4.5 +/- 10.2
Adverse events
Time frame: 1 year
| Arm | Deaths | Serious adverse events | Other adverse events |
|---|
| CDC Infusion | 1/5 (20.0%) | 1/5 (20.0%) | 0/5 (0.0%) |
Serious adverse events by term
| Term | Organ system | CDC Infusion |
|---|
| heart failure | Cardiac disorders | 1/5 |
| infection | Respiratory, thoracic and mediastinal disorders | 1/5 |
| ischemic event | Cardiac disorders | 1/5 |
Limitations and caveats: Single arm study without placebo control comparator
+Locations (1)
| Facility | City | Country | Status |
|---|
| Okayama University Hospital | Okayama | Japan | |
+Publications
- 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
- 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
- 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
- Results publication: Hirai K, Ousaka D, Fukushima Y, Kondo M, Eitoku T, Shigemitsu Y, Hara M, Baba K, Iwasaki T, Kasahara S, Ohtsuki S, Oh H. Cardiosphere-derived exosomal microRNAs for myocardial repair in pediatric dilated cardiomyopathy. Sci Transl Med. 2020 Dec 9;12(573):eabb3336. doi: 10.1126/scitranslmed.abb3336. PMID 33298561
+Study documents
+Investigators