Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy

NCT03129568 | Completed Phase 1 Results DMC

• 1 other identifier: 01F1701003
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.

  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.

  6 months after CDC treatment

Secondary Outcomes (1)

• Change in Ejection Fraction

  6 months after protocol treatment

Study Arms (1)

CDC infusion

EXPERIMENTAL

CDCs infusion by coronary intervention.

Biological: CDC infusion

Interventions

CDC infusion BIOLOGICAL

Injection of CDCs (0.3 million per kg of body weight).

CDC infusion

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients diagnosed as dilated cardiomyopathy.
• Patients aged under 18 years old.
• Cardiac ejection fraction \< 40%.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Okayama University: lead
• Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan: collaborator

Study Sites (1)

Okayama University Hospital

Okayama, 700-8558, Japan

Location

Related Publications (4)

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 RESULT

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

Cardiomegaly; Heart Diseases; Cardiovascular Diseases; Cardiomyopathies; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

Single arm study without placebo control comparator

Results Point of Contact

• Title: Dr. Hidemasa Oh
• Organization: Okayama University Hospital

hidemasa@okayama-u.ac.jp

086-235-6506

Study Officials

• Shigeru Uemura, M.D., Ph.D.

  Shonan Tobu Clinic

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Transcoronary infusion

Study Record Dates

• First Submitted: April 14, 2017
• First Posted: April 26, 2017
• Study Start: April 14, 2017
• Primary Completion: September 17, 2018
• Study Completion: December 9, 2020
• Last Updated: November 26, 2021
• Results First Posted: November 1, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)