Myocardial IschemiaPhase 1NCT04696328
Clinical Trial of Human (Allogeneic) iPS Cell-derived Cardiomyocytes Sheet for Ischemic Cardiomyopathy
A Phase 1 study testing Human (allogeneic) iPS cell derived-cardiomyocyte sheet in people with Myocardial Ischemia, run by Osaka University at 1 site in Japan.
Trial summary
Targeting patients with severe ischemic cardiomyopathy, the purpose of this study is as follows: to confirm short-term efficacy by observing changes and transitions in cardiac function and clinical symptoms compared with each patient's baseline (before and after comparison) by human iPS cell-derived cardiomyocyte… Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Myocardial Ischemia
- What is being tested?
- Human (allogeneic) iPS cell derived-cardiomyocyte sheet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteJapan
- Study dates
- 2 Dec 2019to 30 May 2023
- Phase
- Phase 1
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with chronic ischemic heart disease
- Patients with Grade III-IV NYHA Functional Classification heart failure
- Patients who are in the state of heart failure despite maximal oral medications including digitalis, diuretics, ACE inhibitors, ARBs, beta-blockers, anti-aldosterone drugs, and oral cardiotonics
- Patients who are 20 years of age or older at the point of consent
- Patients at risk of worsening heart failure despite being under standard surgical treatment (coronary artery bypass surgery, mitral valve angioplasty, left ventricular angioplasty, cardiac resynchronization therapy, and percutaneous coronary intervention) for more than 3 months
- Patients with LVEF (Echocardiography) at rest of 35% or less
- Patients whose informed consent for clinical trial participation can be obtained from the subject himself/herself in writing
- Patients who can continue to visit to the clinical trial site for 52 weeks after obtaining consent, continue to live in Japan, and can be expected to have data collected by NRMD/PMS
Who cannot join
- Patients with autoimmune diseases
- Patients with allergies or hypersensitivity to the immunosuppressant used
- Patients with active infections
- Patients who remain in shock due to worsening heart failure
- Patients with irreversible organ failure other than heart
- Patients with malignant tumors
Show all 12Show fewer
- Patients who are or may be pregnant
- Patients with history of alcoholism or drug addiction within six months from the day of consent
- Patients with allergies or hypersensitivity to animals such as cattle from which the raw materials are derived
- Patients with severe pulmonary hypertension
- Patients within 6 months of completion of other clinical trials at the time of enrollment
- In addition, patients with other cardiovascular abnormalities who are determined to be unfit for this study as per the judgment of the patient enrollment study committee of physicians
Trial locations and contacts
1 site in 1 country.
Japan1 site, 1 recruiting
- Recruiting
Osaka University Hospital
Suita, Osaka, 5650871
Satoshi Kainuma, Ph.D, +81-6-6879-3154, saisentan@tissue.med.osaka-u.ac.jp
Masao Sasai, Ph.D, +81-6-6105-5240, sasai-masao@tissue.med.osaka-u.ac.jp
About this study
Official title: Clinical Trial of Human (Allogeneic) iPS Cell-derived Cardiomyocytes Sheet for Ischemic
Targeting patients with severe ischemic cardiomyopathy, the purpose of this study is as follows: to confirm short-term efficacy by observing changes and transitions in cardiac function and clinical symptoms compared with each patient's baseline (before and after comparison) by human iPS cell-derived cardiomyocyte sheet transplantation, and to evaluate the safety and tolerability including the combined use of immunosuppressants.
Read the detailed description
The objective of this study is to confirm the efficacy and safety of a human (allogeneic) iPS cell-derived cardiomyocyte sheet in combination with an immunosuppressant for ischemic cardiomyopathy patients. The primary evaluation items will be improvement of left ventricular systolic function (LVEF) for efficacy, and safety will be assessed by blood tests, general laboratory tests, and other safety-related evaluations. Secondary evaluation items are NYHA functional evaluation, left ventricular remodeling evaluation by echocardiography, hemodynamic evaluation, physical activity function evaluation such as 6MWD and SAS, QOL, and exercise tolerance evaluation by questionnaires.
- Conditions
- Myocardial Ischemia
- Being tested
- Human (allogeneic) iPS cell derived-cardiomyocyte sheet
- Keywords
- ischemic cardiomyopathy, D017202
- Medical subject headings
- Myocardial Ischemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human (allogeneic) iPS cell derived-cardiomyocyte sheetBiological
Transplantation
| Study group | Role | What they receive |
|---|---|---|
| Group of subjects undergoing cell transplantation | Experimental | Human (allogeneic) iPS cell derived-cardiomyocyte sheet transplantation (only once) Receives: Biological: Human (allogeneic) iPS cell derived-cardiomyocyte sheet |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of patients with improved LVEF
The number of patients with improved LVEF by echocardiography 26 weeks postoperatively compared with preoperatively.
Measured: 26 weeks
Incidence of adverse events and defects [Safety and Tolerability]
Regarding adverse events and side effects (of the adverse events, those whose causal relationship with the clinical trial product is determined to be other than "not related" will be treated as side effects.) the number of occurrences and the number of occurrence examples by event and severity will be obtained.
Measured: From postoperative to the end of the observation period (52 weeks)
Incidence of serious adverse events [Safety and Tolerability]
Regarding serious adverse events, the number of occurrences and the number of occurrence examples by event and severity will be obtained.
Measured: From postoperative to the end of the observation period (52 weeks)
Incidence of abnormal vital signs [Safety and Tolerability]
Regarding changes in vital signs(Body temperature, blood pressure (systolic, diastolic), and pulse rate), summary statistics and changes at each measurement time point will be obtained.
Measured: Before surgery, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Incidence of abnormal general blood tests [Safety and Tolerability]
Regarding changes in general blood tests(WBC, RBC, Hb, Ht, PLT), summary statistics and changes at each measurement time point will be obtained.
Measured: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Incidence of abnormal blood biochemical tests [Safety and Tolerability]
Regarding changes in blood biochemistry tests(AST(GOT), ALT(GPT), LDH, ALP, BUN, Cre, UA, TG, T-Cho, LDL-Cho, Alb, CK, CK-MB, electrolytes (Na, K, Cl, Ca, iP, Mg), CRP, blood sugar), summary statistics and changes at each measurement time point will be obtained.
Measured: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Incidence of abnormal tumor marker tests [Safety and Tolerability]
Regarding changes in tumor marker tests(AFP, CA19-9, CEA, hCG), summary statistics and changes at each measurement time point will be obtained.
Measured: Screening, 13 weeks, 26 weeks, 52 weeks
Incidence of cardiac function clinical events such as death and hospitalization [Safety and Tolerability]
With respect to the incidence of cardiac function clinical events such as death and hospitalization, the number of cases in which the causes of death are related to heart disease and those unrelated to heart disease will be determined for cases of death.
Measured: From postoperative to the end of the observation period (52 weeks)
Secondary outcomes (15)
Number of Responder patients 26 and 52 weeks after transplantation of this product
To comprehensively evaluate the efficacy of this product transplantation
Measured: 26 and 52 weeks
Contraction function of the entire left ventricle
To comprehensively evaluate the efficacy of this product transplantation
Measured: 26 weeks
Left ventricular remodeling (LVESVI)
Changes in left ventricular end systolic volume index (LVESVI) (echocardiography, CT (if available))
Measured: 26 weeks
Left ventricular remodeling (LVEDVI)
Changes in left ventricular end-diastolic volume index (LVEDVI) (echocardiography, CT (if available))
Measured: 26 weeks
New York Heart Association functional classification
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. Class I to Class IV, the more severe, the higher the number.
Measured: Before surgery, 26 weeks, 52 weeks
Specific Activity Scale (SAS)
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. This is a quantitative evaluation of the subjective symptoms of heart failure from the viewpoint of exercise tolerance. List various daily activities for which exercise intensity [oxygen uptake or metabolic equivalents (METs)] is almost known in advance, ask whether they are possible, and exercise with the lowest activity level that was not possible is evaluated value. The higher the number, the better the condition.
Measured: Before surgery, 26 weeks, 52 weeks
The Minnesota Living with Heart Failure Questionnaire
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. The lower the number, the better the condition.
Measured: Before surgery, 26 weeks, 52 weeks
36-Item Short Form Survey (SF-36)
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. The higher the number, the better the condition.
Measured: Before surgery, 26 weeks, 52 weeks
6-minute walking distance
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. The higher the number, the better the condition.
Measured: Before surgery, 26 weeks, 52 weeks
Brain natriuretic peptide (BNP)
Evaluation of the following changes and transitions.
Measured: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
N-terminal pro-brain natriuretic peptide (NT-proBNP)
Evaluation of the following changes and transitions.
Measured: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Exercise tolerance (VO2max)
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. Measure the maximum oxygen uptake (VO2max) using the bicycle ergometer.
Measured: Before surgery, 26 weeks, 52 weeks
Exercise tolerance (AT)
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. Measure the anaerobic metabolism threshold (AT) using the bicycle ergometer.
Measured: Before surgery, 26 weeks, 52 weeks
Exercise tolerance (VE/VCO2)
Evaluation of the following changes and transitions before and 26 and 52 weeks after surgery. Measure the expiratory minute volume (VE)/the CO2 uptake (VCO2) using the bicycle ergometer.
Measured: Before surgery, 26 weeks, 52 weeks
Cumulative number of rejections that occurred during the observation period
Cumulative number of rejections from transplant up to 26 weeks after surgery
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jan 2021
- Study start2 Dec 2019
- Primary completion30 May 2022 (estimated)
- Study completion30 May 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myocardial IschemiaStudies 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
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Osaka University
- Sponsor type
- University, hospital or other
- Collaborators
- Cuorips Inc.
- Responsible party
- Principal investigator, Koichi Toda, Osaka University
- Investigators
- Yoshiki Sawa, Ph.D, Study director, Osaka University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04696328
- Sponsor's protocol ID
- CVSC0005
- Status last verified
- Apr 2021
- First submitted
- 11 Dec 2020
- Last updated
- 20 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Not planned at this time.
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial of Human (Allogeneic) iPS Cell-derived Cardiomyocytes Sheet for Ischemic
- ClinicalTrials.gov ID
- NCT04696328
- Acronym / sponsor ID
- CVSC0005
- Conditions
- Myocardial Ischemia
- Keywords
- ischemic cardiomyopathy, D017202
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Osaka UniversityUniversity, hospital or other
- Collaborators
- Cuorips Inc.
- Study start
- 2 Dec 2019 (actual)
- Primary completion
- 30 May 2022 (estimated)
- Study completion
- 30 May 2023 (estimated)
- First posted
- 6 Jan 2021 (actual)
- 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.
Targeting patients with severe ischemic cardiomyopathy, the purpose of this study is as follows: to confirm short-term efficacy by observing changes and transitions in cardiac function and clinical symptoms compared with each patient's baseline (before and after comparison) by human iPS cell-derived cardiomyocyte sheet transplantation, and to evaluate the safety and tolerability including the combined use of immunosuppressants.
Detailed description
The objective of this study is to confirm the efficacy and safety of a human (allogeneic) iPS cell-derived cardiomyocyte sheet in combination with an immunosuppressant for ischemic cardiomyopathy patients. The primary evaluation items will be improvement of left ventricular systolic function (LVEF) for efficacy, and safety will be assessed by blood tests, general laboratory tests, and other safety-related evaluations. Secondary evaluation items are NYHA functional evaluation, left ventricular remodeling evaluation by echocardiography, hemodynamic evaluation, physical activity function evaluation such as 6MWD and SAS, QOL, and exercise tolerance evaluation by questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Group of subjects undergoing cell transplantation | Experimental | Biological: Human (allogeneic) iPS cell derived-cardiomyocyte sheetHuman (allogeneic) iPS cell derived-cardiomyocyte sheet transplantation (only once) |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patients with chronic ischemic heart disease
- Patients with Grade III-IV NYHA Functional Classification heart failure
- Patients who are in the state of heart failure despite maximal oral medications including digitalis, diuretics, ACE inhibitors, ARBs, beta-blockers, anti-aldosterone drugs, and oral cardiotonics
- Patients who are 20 years of age or older at the point of consent
- Patients at risk of worsening heart failure despite being under standard surgical treatment (coronary artery bypass surgery, mitral valve angioplasty, left ventricular angioplasty, cardiac resynchronization therapy, and percutaneous coronary intervention) for more than 3 months
Key exclusion criteria (12 listed)
- Patients with autoimmune diseases
- Patients with allergies or hypersensitivity to the immunosuppressant used
- Patients with active infections
- Patients who remain in shock due to worsening heart failure
- Patients with irreversible organ failure other than heart
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 |
|---|---|---|
| Human (allogeneic) iPS cell derived-cardiomyocyte sheetTransplantation | Biological | Group of subjects undergoing cell transplantation |
Primary outcome
The number of patients with improved LVEF
Time frame: 26 weeks
The number of patients with improved LVEF by echocardiography 26 weeks postoperatively compared with preoperatively.
Incidence of adverse events and defects [Safety and Tolerability]
Time frame: From postoperative to the end of the observation period (52 weeks)
Regarding adverse events and side effects (of the adverse events, those whose causal relationship with the clinical trial product is determined to be other than "not related" will be treated as side effects.) the number of occurrences and the number of occurrence examples by event and severity will be obtained.
Incidence of serious adverse events [Safety and Tolerability]
Time frame: From postoperative to the end of the observation period (52 weeks)
Regarding serious adverse events, the number of occurrences and the number of occurrence examples by event and severity will be obtained.
Incidence of abnormal vital signs [Safety and Tolerability]
Time frame: Before surgery, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Regarding changes in vital signs(Body temperature, blood pressure (systolic, diastolic), and pulse rate), summary statistics and changes at each measurement time point will be obtained.
Incidence of abnormal general blood tests [Safety and Tolerability]
Time frame: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Regarding changes in general blood tests(WBC, RBC, Hb, Ht, PLT), summary statistics and changes at each measurement time point will be obtained.
Incidence of abnormal blood biochemical tests [Safety and Tolerability]
Time frame: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Regarding changes in blood biochemistry tests(AST(GOT), ALT(GPT), LDH, ALP, BUN, Cre, UA, TG, T-Cho, LDL-Cho, Alb, CK, CK-MB, electrolytes (Na, K, Cl, Ca, iP, Mg), CRP, blood sugar), summary statistics and changes at each measurement time point will be obtained.
Incidence of abnormal tumor marker tests [Safety and Tolerability]
Time frame: Screening, 13 weeks, 26 weeks, 52 weeks
Regarding changes in tumor marker tests(AFP, CA19-9, CEA, hCG), summary statistics and changes at each measurement time point will be obtained.
Incidence of cardiac function clinical events such as death and hospitalization [Safety and Tolerability]
Time frame: From postoperative to the end of the observation period (52 weeks)
With respect to the incidence of cardiac function clinical events such as death and hospitalization, the number of cases in which the causes of death are related to heart disease and those unrelated to heart disease will be determined for cases of death.
15 secondary outcomes
Number of Responder patients 26 and 52 weeks after transplantation of this product
Time frame: 26 and 52 weeks
Contraction function of the entire left ventricle
Time frame: 26 weeks
Left ventricular remodeling (LVESVI)
Time frame: 26 weeks
Left ventricular remodeling (LVEDVI)
Time frame: 26 weeks
New York Heart Association functional classification
Time frame: Before surgery, 26 weeks, 52 weeks
Specific Activity Scale (SAS)
Time frame: Before surgery, 26 weeks, 52 weeks
The Minnesota Living with Heart Failure Questionnaire
Time frame: Before surgery, 26 weeks, 52 weeks
36-Item Short Form Survey (SF-36)
Time frame: Before surgery, 26 weeks, 52 weeks
6-minute walking distance
Time frame: Before surgery, 26 weeks, 52 weeks
Brain natriuretic peptide (BNP)
Time frame: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
N-terminal pro-brain natriuretic peptide (NT-proBNP)
Time frame: Before surgery, 1 days, 7 days, 14 days, 4 weeks, 13 weeks, 26 weeks, 52 weeks
Exercise tolerance (VO2max)
Time frame: Before surgery, 26 weeks, 52 weeks
Exercise tolerance (AT)
Time frame: Before surgery, 26 weeks, 52 weeks
Exercise tolerance (VE/VCO2)
Time frame: Before surgery, 26 weeks, 52 weeks
Cumulative number of rejections that occurred during the observation period
Time frame: 26 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2021.
- Individual participant dataSharing planned: No. Not planned at this time.
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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.
Yoshiki Sawa, Ph.D study director
Osaka University
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 Human (allogeneic) iPS cell derived-cardiomyocyte sheet in people with Myocardial Ischemia, run by Osaka 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 8 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 20 Apr 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Takuji Kawamura, Ph.D (+81-6-6879-3154, saisentan@tissue.med.osaka-u.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number of patients with improved LVEF (time frame: 26 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Apr 2021.