Dilated CardiomyopathyPhase 1TICAP-DCMNCT03129568

Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy

A Phase 1 study testing CDC infusion in people with Dilated Cardiomyopathy, run by Okayama University at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages Up to 17 years
Conditions the study is for: Dilated Cardiomyopathy
What is being tested?: CDC infusion
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 5 participants
Where: 1 siteJapan
Study dates: 14 Apr 2017to 9 Dec 2020
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Up to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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

Who cannot join

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.

Show all 14Show fewer

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).

Trial locations and contacts

1 site in 1 country.

Japan1 site

Okayama University Hospital
Okayama, 700-8558

Other trials in Japan

About this study

Official title: A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy

Read the 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.

Conditions: Dilated Cardiomyopathy
Being tested: CDC infusion
Medical subject headings: Cardiomyopathy, Dilated

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT03129568
Study group	Role	What they receive
CDC infusion	Experimental	CDCs infusion by coronary intervention. Receives: Biological: CDC infusion

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 6 months after CDC treatment

Secondary outcomes (1)

Change in Ejection Fraction
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.
Measured: 6 months after protocol treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Apr 20179 Dec 2020

First posted26 Apr 2017
Study start14 Apr 2017
Primary completion17 Sep 2018
Study completion9 Dec 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (4)Papers the sponsor has linked to this study.
Other trials for Dilated CardiomyopathyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Transcoronary infusion
Masking: None (open label)
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03129568
Sponsor's protocol ID: 01F1701003
Acronym: TICAP-DCM
Status last verified: Nov 2021
First submitted: 14 Apr 2017
Last updated: 26 Nov 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

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.
PubMed 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.
PubMed 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.
PubMed 25403163
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.
PubMed 33298561

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2/3
A Study of the Efficacy and Safety of Danicamtiv in Participants With Symptomatic Genetic and Familial Dilated Cardiomyopathy
Symptomatic Genetic Dilated Cardiomyopathy
RecruitingPhase 3
A Study of the Long-term Safety and Efficacy of Danicamtiv in Adults With Symptomatic Genetic and Familial Dilated Cardiomyopathy
Symptomatic Genetic Dilated Cardiomyopathy (DCM)
RecruitingObservational study
A 10-Minute Cardiovascular Magnetic Resonance Protocol for Cardiac Disease
Coronary Artery Disease, Myocarditis
RecruitingPhase 1/2
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders

All Dilated Cardiomyopathy trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy
ClinicalTrials.gov ID: NCT03129568
Acronym / sponsor ID: TICAP-DCM / 01F1701003
Conditions: Dilated Cardiomyopathy
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 5 participants (actual)
Lead sponsor: Okayama UniversityUniversity, hospital or other
Collaborators: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Study start: 14 Apr 2017 (actual)
Primary completion: 17 Sep 2018 (actual)
Study completion: 9 Dec 2020 (actual)
First posted: 26 Apr 2017 (actual)
Results: Posted 1 Nov 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Transcoronary infusion

Arm	Type	Receives
CDC infusion	Experimental	Biological: CDC infusionCDCs infusion by coronary intervention.

Who may qualify

Ages: Up to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

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

Key exclusion criteria (14 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
CDC infusionInjection of CDCs (0.3 million per kg of body weight).	Biological	CDC infusion

Primary outcome

Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.
Time frame: 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.

1 secondary outcome

Change in Ejection Fraction
Time frame: 6 months after protocol treatment

Results and safety

Results posted to the registry on 1 Nov 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
CDC Infusion	5	5

Primary outcome: Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.

Time frame: 6 months after CDC treatment. Count of participants. Unit: Participants

Measure	CDC Infusion n = 5
Value	1

Population analysed: Adverse events

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 1 year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
CDC Infusion	1 of 5 (20%)	0 of 5 (0%)	1 of 5

Limitations and caveats (from the sponsor): Single arm study without placebo control comparator

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 28052915Ishigami 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.
Background: PubMed 26232942Tarui 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.
Background: PubMed 25403163Ishigami 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 publication: PubMed 33298561Hirai 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.
Individual participant dataSharing planned: No

Related trials for Dilated Cardiomyopathy

RecruitingPhase 2/3
A Study of the Efficacy and Safety of Danicamtiv in Participants With Symptomatic Genetic and Familial Dilated Cardiomyopathy
Symptomatic Genetic Dilated Cardiomyopathy
RecruitingPhase 3
A Study of the Long-term Safety and Efficacy of Danicamtiv in Adults With Symptomatic Genetic and Familial Dilated Cardiomyopathy
Symptomatic Genetic Dilated Cardiomyopathy (DCM)
RecruitingObservational study
A 10-Minute Cardiovascular Magnetic Resonance Protocol for Cardiac Disease
Coronary Artery Disease, Myocarditis
RecruitingPhase 1/2
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Shigeru Uemura, M.D., Ph.D. study chair

Shonan Tobu Clinic

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CDC infusion in people with Dilated Cardiomyopathy, run by Okayama University at 1 site in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs. (time frame: 6 months after CDC treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03129568, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2021.