Clinical trial NCT03129568: Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy
Official title: A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Dilated Cardiomyopathy
Lead sponsor: Okayama University
Sponsor class: OTHER
Collaborators: name: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
First posted: 2017-04-26
Enrollment: 5
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Transcoronary infusion
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.
timeFrame: 6 months after CDC treatment
Secondary outcomes:
measure: Change in Ejection Fraction
timeFrame: 6 months after protocol treatment
Other outcomes:
Locations:
city: Okayama
country: Japan
facility: Okayama University Hospital
Study officials:
affiliation: Shonan Tobu Clinic
name: Shigeru Uemura, M.D., Ph.D.
role: STUDY_CHAIR
References:
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.
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.
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.
citation: 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.