Hypoplastic Left Heart SyndromePhase 2NCT01829750
Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS)
A Phase 2 study testing Cardiac progenitor cell infusion in people with Hypoplastic Left Heart Syndrome and Single Right Ventricle, run by Okayama University at 1 site in Japan.
Trial summary
The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease. Patients with preoperative high-risk group or whose cardiac function did not recover postoperatively eventually have no choice other than heart transplantation. Read more about this study
- Who is it for?
- Ages Up to 20 years
- Conditions the study is for
- Hypoplastic Left Heart Syndrome, Single Right Ventricle, Single Left Ventricle
- What is being tested?
- Cardiac progenitor cell infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 34 participants
- Where
- 1 siteJapan
- Study dates
- Apr 2013to Sep 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: Age is 0 year or more and 20 years or less at the time of enrollment.
- The patients have functional single ventricular physiology with the indication to have stage-2 or -3 palliative surgeries.
- The ventricular ejection fraction <60%.
Who cannot join
- 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
Show all 13Show fewer
- A patient with a complication of 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.
About this study
Official title: Phase 2 Study of Intracoronary Infusion of Cardiac Progenitor Cells in Patients With Univentricular Heart Disease
The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease. Patients with preoperative high-risk group or whose cardiac function did not recover postoperatively eventually have no choice other than heart transplantation.
Read the detailed description
Following the safety verification of the Phase I study (TICAP trial) that has been implemented and completed, the efficacy will be verified in this Phase II clinical study. The rationale of this study is based on the animal studies that transplanted cardiac progenitor cells may regenerate damaged myocardium by giving rise to cardiac muscle and vascular cell types. Preclinical and phase I studies indicate the possibilities of cardiac function improvements and reduced myocardial fibrosis by progenitor cell infusion.
The efficacy of cell transplantation is assessed as the primary endpoint of the Phase II clinical study. The improvements of cardiac ejection fraction assessed by echocardiography, ventriculography, and cardiac MRI, which are conducted before and after treatment, are assessed as the evaluation items. The absolute values of the improvement of cardiac ejection fraction obtained by the above three imaging modalities before and after treatment are compared between two groups of the transplantation group and non-transplantation group.
- Being tested
- Cardiac progenitor cell infusion
- Keywords
- Univentricular heart
- Medical subject headings
- Hypoplastic Left Heart Syndrome, Univentricular Heart
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cardiac progenitor cell infusionGenetic
(Stage 1) Cardiac progenitor cell infusion in patients who assigned as active comparator group
(Stage 2) Rescuing transplantation is applicable in patients, along with their written consent, 4 months after palliations who were allocated as control group in stage 1.
| Study group | Role | What they receive |
|---|---|---|
| Control | Sham comparator | (Stage 1) No active intervention after standard surgical treatment
(Stage 2) Rescuing transplantation by cardiac progenitor cell infusion is applicable in patients, along with their written consent, 4 months after palliations who were assigned as control group in stage 1. Receives: Genetic: Cardiac progenitor cell infusion |
| Cardiac progenitor cell infusion | Active comparator | (Stage 1) single dose, intracoronary infusion of 0.3 million cells/kg cardiac progenitor cells Receives: Genetic: Cardiac progenitor cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiac function
The primary outcome measure is to evaluate the cardiac function improvement by echocardiography, ventriculography, and cardiac MRI, which are conducted before and 3 months after surgical treatment.
Measured: 3 Months
Secondary outcomes (1)
Cardiac function
The secondary outcome measure is to determine the cardiac function improvements by three different imaging modalities at 12 months after treatment.
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2013 (estimated)
- Study startApr 2013
- Primary completionMar 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Hypoplastic Left Heart SyndromeStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Okayama University
- Sponsor type
- University, hospital or other
- Collaborators
- Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
- Responsible party
- Principal investigator, Hidemasa Oh, MD, Okayama University
- Investigators
- Hidemasa Oh, M.D., Ph.D., Principal investigator, Okayama University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01829750
- Sponsor's protocol ID
- 0329-18
- Other IDs
- MHLW25040101
- Status last verified
- Nov 2021
- First submitted
- 9 Apr 2013
- Last updated
- 7 Dec 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Oh H, Bradfute SB, Gallardo TD, Nakamura T, Gaussin V, Mishina Y, Pocius J, Michael LH, Behringer RR, Garry DJ, Entman ML, Schneider MD. Cardiac progenitor cells from adult myocardium: homing, differentiation, and fusion after infarction. Proc Natl Acad Sci U S A. 2003 Oct 14;100(21):12313-8. doi: 10.1073/pnas.2132126100. Epub 2003 Oct 6.
PubMed 14530411Tateishi K, Ashihara E, Honsho S, Takehara N, Nomura T, Takahashi T, Ueyama T, Yamagishi M, Yaku H, Matsubara H, Oh H. Human cardiac stem cells exhibit mesenchymal features and are maintained through Akt/GSK-3beta signaling. Biochem Biophys Res Commun. 2007 Jan 19;352(3):635-41. doi: 10.1016/j.bbrc.2006.11.096. Epub 2006 Nov 27.
PubMed 17150190Tateishi K, Ashihara E, Takehara N, Nomura T, Honsho S, Nakagami T, Morikawa S, Takahashi T, Ueyama T, Matsubara H, Oh H. Clonally amplified cardiac stem cells are regulated by Sca-1 signaling for efficient cardiovascular regeneration. J Cell Sci. 2007 May 15;120(Pt 10):1791-800. doi: 10.1242/jcs.006122.
PubMed 17502484Tateishi K, Takehara N, Matsubara H, Oh H. Stemming heart failure with cardiac- or reprogrammed-stem cells. J Cell Mol Med. 2008 Dec;12(6A):2217-32. doi: 10.1111/j.1582-4934.2008.00487.x. Epub 2008 Aug 27.
PubMed 18754813Takehara N, Tsutsumi Y, Tateishi K, Ogata T, Tanaka H, Ueyama T, Takahashi T, Takamatsu T, Fukushima M, Komeda M, Yamagishi M, Yaku H, Tabata Y, Matsubara H, Oh H. Controlled delivery of basic fibroblast growth factor promotes human cardiosphere-derived cell engraftment to enhance cardiac repair for chronic myocardial infarction. J Am Coll Cardiol. 2008 Dec 2;52(23):1858-1865. doi: 10.1016/j.jacc.2008.06.052.
PubMed 19038683Ishigami 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 28052915Hirai K, Sawada R, Hayashi T, Araki T, Nakagawa N, Kondo M, Yasuda K, Hirata T, Sato T, Nakatsuka Y, Yoshida M, Kasahara S, Baba K, Oh H; TICAP/PERSEUS Study Group. Eight-Year Outcomes of Cardiosphere-Derived Cells in Single Ventricle Congenital Heart Disease. J Am Heart Assoc. 2024 Nov 19;13(22):e038137. doi: 10.1161/JAHA.124.038137. Epub 2024 Nov 11.
PubMed 39526355Sano T, Ousaka D, Goto T, Ishigami S, Hirai K, Kasahara S, Ohtsuki S, Sano S, Oh H. Impact of Cardiac Progenitor Cells on Heart Failure and Survival in Single Ventricle Congenital Heart Disease. Circ Res. 2018 Mar 30;122(7):994-1005. doi: 10.1161/CIRCRESAHA.117.312311. Epub 2018 Jan 24.
PubMed 29367212
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Intracoronary Infusion of Cardiac Progenitor Cells in Patients With Univentricular Heart Disease
- ClinicalTrials.gov ID
- NCT01829750
- Acronym / sponsor ID
- 0329-18
- Keywords
- Univentricular heart
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Okayama UniversityUniversity, hospital or other
- Collaborators
- Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
- Study start
- Apr 2013
- Primary completion
- Mar 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 11 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease. Patients with preoperative high-risk group or whose cardiac function did not recover postoperatively eventually have no choice other than heart transplantation.
Detailed description
Following the safety verification of the Phase I study (TICAP trial) that has been implemented and completed, the efficacy will be verified in this Phase II clinical study. The rationale of this study is based on the animal studies that transplanted cardiac progenitor cells may regenerate damaged myocardium by giving rise to cardiac muscle and vascular cell types. Preclinical and phase I studies indicate the possibilities of cardiac function improvements and reduced myocardial fibrosis by progenitor cell infusion.
The efficacy of cell transplantation is assessed as the primary endpoint of the Phase II clinical study. The improvements of cardiac ejection fraction assessed by echocardiography, ventriculography, and cardiac MRI, which are conducted before and after treatment, are assessed as the evaluation items. The absolute values of the improvement of cardiac ejection fraction obtained by the above three imaging modalities before and after treatment are compared between two groups of the transplantation group and non-transplantation group.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control | Sham comparator | Genetic: Cardiac progenitor cell infusion(Stage 1) No active intervention after standard surgical treatment (Stage 2) Rescuing transplantation by cardiac progenitor cell infusion is applicable in patients, along with their written consent, 4 months after palliations who were assigned as control group in stage 1. |
| Cardiac progenitor cell infusion | Active comparator | Genetic: Cardiac progenitor cell infusion(Stage 1) single dose, intracoronary infusion of 0.3 million cells/kg cardiac progenitor cells |
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age: Age is 0 year or more and 20 years or less at the time of enrollment.
- The patients have functional single ventricular physiology with the indication to have stage-2 or -3 palliative surgeries.
- The ventricular ejection fraction <60%.
Key exclusion criteria (13 listed)
- 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
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 |
|---|---|---|
| Cardiac progenitor cell infusion(Stage 1) Cardiac progenitor cell infusion in patients who assigned as active comparator group (Stage 2) Rescuing transplantation is applicable in patients, along with their written consent, 4 months after palliations who were allocated as control group in stage 1. | Genetic | Cardiac progenitor cell infusion, Control |
Primary outcome
Cardiac function
Time frame: 3 Months
The primary outcome measure is to evaluate the cardiac function improvement by echocardiography, ventriculography, and cardiac MRI, which are conducted before and 3 months after surgical treatment.
1 secondary outcome
Cardiac function
Time frame: 12 Months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2021.
- Background: PubMed 14530411Oh H, Bradfute SB, Gallardo TD, Nakamura T, Gaussin V, Mishina Y, Pocius J, Michael LH, Behringer RR, Garry DJ, Entman ML, Schneider MD. Cardiac progenitor cells from adult myocardium: homing, differentiation, and fusion after infarction. Proc Natl Acad Sci U S A. 2003 Oct 14;100(21):12313-8. doi: 10.1073/pnas.2132126100. Epub 2003 Oct 6.
- Background: PubMed 17150190Tateishi K, Ashihara E, Honsho S, Takehara N, Nomura T, Takahashi T, Ueyama T, Yamagishi M, Yaku H, Matsubara H, Oh H. Human cardiac stem cells exhibit mesenchymal features and are maintained through Akt/GSK-3beta signaling. Biochem Biophys Res Commun. 2007 Jan 19;352(3):635-41. doi: 10.1016/j.bbrc.2006.11.096. Epub 2006 Nov 27.
- Background: PubMed 17502484Tateishi K, Ashihara E, Takehara N, Nomura T, Honsho S, Nakagami T, Morikawa S, Takahashi T, Ueyama T, Matsubara H, Oh H. Clonally amplified cardiac stem cells are regulated by Sca-1 signaling for efficient cardiovascular regeneration. J Cell Sci. 2007 May 15;120(Pt 10):1791-800. doi: 10.1242/jcs.006122.
- Background: PubMed 18754813Tateishi K, Takehara N, Matsubara H, Oh H. Stemming heart failure with cardiac- or reprogrammed-stem cells. J Cell Mol Med. 2008 Dec;12(6A):2217-32. doi: 10.1111/j.1582-4934.2008.00487.x. Epub 2008 Aug 27.
- Background: PubMed 19038683Takehara N, Tsutsumi Y, Tateishi K, Ogata T, Tanaka H, Ueyama T, Takahashi T, Takamatsu T, Fukushima M, Komeda M, Yamagishi M, Yaku H, Tabata Y, Matsubara H, Oh H. Controlled delivery of basic fibroblast growth factor promotes human cardiosphere-derived cell engraftment to enhance cardiac repair for chronic myocardial infarction. J Am Coll Cardiol. 2008 Dec 2;52(23):1858-1865. doi: 10.1016/j.jacc.2008.06.052.
- Results publication: 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.
- Derived from this study: PubMed 39526355Hirai K, Sawada R, Hayashi T, Araki T, Nakagawa N, Kondo M, Yasuda K, Hirata T, Sato T, Nakatsuka Y, Yoshida M, Kasahara S, Baba K, Oh H; TICAP/PERSEUS Study Group. Eight-Year Outcomes of Cardiosphere-Derived Cells in Single Ventricle Congenital Heart Disease. J Am Heart Assoc. 2024 Nov 19;13(22):e038137. doi: 10.1161/JAHA.124.038137. Epub 2024 Nov 11.
- Derived from this study: PubMed 29367212Sano T, Ousaka D, Goto T, Ishigami S, Hirai K, Kasahara S, Ohtsuki S, Sano S, Oh H. Impact of Cardiac Progenitor Cells on Heart Failure and Survival in Single Ventricle Congenital Heart Disease. Circ Res. 2018 Mar 30;122(7):994-1005. doi: 10.1161/CIRCRESAHA.117.312311. Epub 2018 Jan 24.
- Individual participant dataSharing planned: No
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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.
Hidemasa Oh, M.D., Ph.D. principal investigator
Okayama University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cardiac progenitor cell infusion in people with Hypoplastic Left Heart Syndrome and Single Right Ventricle, 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 13 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 7 Dec 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Cardiac function (time frame: 3 Months). 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 NCT01829750, which also lists 8 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 7 Dec 2021.