TICAP-DCM: Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy

• Sponsor: Okayama University (Other)
• Overall Status: Completed
• CT.gov ID: NCT03129568
• Collaborator: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan (Other)
• Enrollment: 5
• Location: 1
• Arm: 1
• Actual Duration (Months): 43.9
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Dilated Cardiomyopathy	Biological: CDC infusion	Phase 1

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Transcoronary infusionTranscoronary infusion
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy
• Actual Study Start Date: Apr 14, 2017
• Actual Primary Completion Date: Sep 17, 2018
• Actual Study Completion Date: Dec 9, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: CDC infusion CDCs infusion by coronary intervention.	Biological: CDC infusion; Injection of CDCs (0.3 million per kg of body weight).

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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 Criteria

Criteria

• Ages Eligible for Study: N/A to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Okayama University Hospital	Okayama		Japan	700-8558

Sponsors and Collaborators

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

Investigators

• Study Chair: Shigeru Uemura, M.D., Ph.D., Shonan Tobu Clinic

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Dec 9, 2020

More Information

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.
• 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.
• 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.

• Responsible Party: Hidemasa Oh, MD, Professor, Okayama University
• ClinicalTrials.gov Identifier: NCT03129568
• Other Study ID Numbers: 01F1701003
• First Posted: Apr 26, 2017
• Last Update Posted: Nov 26, 2021
• Last Verified: Nov 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Cardiomyopathies; Cardiomyopathy, Dilated

Study Results

Participant Flow

Arm/Group Title	CDC Infusion
Arm/Group Description	CDCs infusion by coronary intervention. CDC infusion: Injection of CDCs (0.3 million per kg of body weight).
Period Title: Overall Study
STARTED	5
COMPLETED	5
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	CDC Infusion
Arm/Group Description	CDCs infusion by coronary intervention. CDC infusion: Injection of CDCs (0.3 million per kg of body weight).
Overall Participants	5
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	11.4 (6.7)
Sex: Female, Male (Count of Participants)
Female	3 60%
Male	2 40%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	5 100%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	0 0%
More than one race	0 0%
Unknown or Not Reported	0 0%
Cardiac Function (percentage of baseline ejection fraction) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [percentage of baseline ejection fraction]	28.5 (10.7)

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.
Description	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.
Time Frame	6 months after CDC treatment

Outcome Measure Data

Analysis Population Description

Adverse events

Arm/Group Title	CDC Infusion
Arm/Group Description	CDCs infusion by coronary intervention. CDC infusion: Injection of CDCs (0.3 million per kg of body weight).
Measure Participants	5
Count of Participants [Participants]	1 20%

2. Secondary Outcome

Title	Change in Ejection Fraction
Description	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.
Time Frame	6 months after protocol treatment

Outcome Measure Data

Analysis Population Description

Changes in cardiac function

Arm/Group Title	CDC Infusion
Arm/Group Description	CDCs infusion by coronary intervention. CDC infusion: Injection of CDCs (0.3 million per kg of body weight).
Measure Participants	5
Mean (Standard Deviation) [percentage of ejection fraction]	4.5 (10.2)

Adverse Events

Time Frame	1 year
Arm/Group Title	CDC Infusion
Arm/Group Description	CDCs infusion by coronary intervention. CDC infusion: Injection of CDCs (0.3 million per kg of body weight).
All Cause Mortality
	CDC Infusion
	Affected / at Risk (%)	# Events
Total	1/5 (20%)
Serious Adverse Events
	CDC Infusion
	Affected / at Risk (%)	# Events
Total	1/5 (20%)
Cardiac disorders
heart failure	1/5 (20%)	1
ischemic event	1/5 (20%)	1
Respiratory, thoracic and mediastinal disorders
infection	1/5 (20%)	1
Other (Not Including Serious) Adverse Events
	CDC Infusion
	Affected / at Risk (%)	# Events
Total	0/5 (0%)

Limitations/Caveats

Single arm study without placebo control comparator

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.