Cardiovascular DiseasesPhase 1NCT04982081
Treating Congestive HF With hiPSC-CMs Through Endocardial Injection
A Phase 1 study testing hiPSC-CM therapy in people with Cardiovascular Diseases and Congestive Heart Failure, run by HELP Therapeutics Co., Ltd. at 1 site in China.
Trial summary
Heart failure is the primary cause of morbidity and mortality worldwide. Currently drug treatments for heart failure manage the symptoms, but not restore the loss cardiomyocytes due to the very limited regenerative capability in the adult heart. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Cardiovascular Diseases, Congestive Heart Failure, Dilated Cardiomyopathy
- What is being tested?
- hiPSC-CM therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 21 Sep 2021to 31 Jul 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged 18-75 years (including 18 and 75).
- Signed the informed consent.
- Patients with congestive heart failure who have received regular treatment for heart failure.
- New York Heart Association (NYHA) Class III or IV despite optimal standard of care
- Left Ventricular Ejection Fraction (LVEF)<40% as assessed by echocardiography ( measure in the 3 months of recruit is included, excluding the measured values within 1 month of myocardial infarction ）
- The thickness of left ventricular ≥8mm
- Female patient who is not pregnant or nursing during the clinical trial
Who cannot join
- PRA ≥ 20% or DSA positive.
- Patients received treatments such as pacemakers, ICD or CRT device.
- Patient with severe valvular disease or presence of a mechanical valve replacement, such as PCI implantation, or patients requiring simultaneous radiofrequency ablation of atrial fibrillation.
- Patient with any therapeutic traumatic heart surgery within 30 days.
- Hemodynamic instability or cardiogenic shock.
- Right heart failure.
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- Restrictive cardiomyopathy such as amyloidosis, sarcoidosis or hematochromia, constrictive pericarditis.
- Myocardial infarction occurred within 30 days or stroke occurred within 60 days before enrollment.
- Thickness at left ventricular free wall infarction < 6 mm.
- Severe ventricular arrhythmias (persistent ventricular tachycardia or other conditions that the investigator considers necessary to exclude).
- Baseline glomerular filtration rate < 30 ml/min / 1.73 m2.
- Abnormal liver function: ALT or AST 3 times higher than the normal value.
- Have a hematologic abnormality as evidenced by hematocrit < 25%, white blood cells count <2,500/ul or platelet count <100000 / ul.
- Known allergies to penicillin, streptomycin or radiocontrast agent.
- Abnormal coagulation function, INR > 1.3, which cannot be corrected.
- Contra-indication to performance of a magnetic resonance imaging scan and PET/ECT examinations.
- Organ transplant recipient
- Patients with other malignant disease within 5 years prior to enrollment.
- Non-cardiac condition that limits lifespan to < 1 year
- On chronic therapy with immunosuppressant medication such as glucocorticoid or TNFα antagonist
- Contra-indication to take immunosuppressant medication.
- Serum positive for infectious diseases (HIV, HBV, HCV, TP).
- Participated in other clinical trials within the previous 3 months .
- Female patient who is pregnant or nursing.
- Other condition that the investigator considers inappropriate for participation in the study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Help Therapeutics
Nanjing, Jiangsu, 210000
Jiaxian Wang, MD,PhD, +8618565616060, wangjx@helpsci.com.cn
Ling Tao, MD
About this study
Official title: Treating Congestive Heart Failure Patients With Human iPSC-derived Cardiomyocytes Through Catheter-based Endocardial Injection
Heart failure is the primary cause of morbidity and mortality worldwide. Currently drug treatments for heart failure manage the symptoms, but not restore the loss cardiomyocytes due to the very limited regenerative capability in the adult heart. Novel reparative therapies that replace the cardiomyocytes loss are highly demanded to restore the cardiac function. The main purposes of this explanatory study is to investigate the safety and efficacy of the catheter-based endocardial delivery of human iPSC-derived cardiomyocytes in patients with congestive heart failure.
Read the detailed description
Patients with severe congestive heart failure will be treated with allogeneic human iPSC-derived cardiomyocytes (HiCM-188) through the catheter-based injections. HiCM188, produced by Help therapeutics with cGMP condition, will be transplanted into the myocardium through a transcatheter endocardial injection system with two dosage (100 million cells or 400 million cells). The safety and efficacy assessments will be conducted at1, 3, 6 and 12 months after the cell transplantation.
- Being tested
- hiPSC-CM therapy
- Keywords
- induced pluripotent stem cell, Congestive heart failure, Regenerative medicine, dilated cardiomyopathy
- Medical subject headings
- Cardiovascular Diseases, Heart Failure, Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hiPSC-CM therapyBiological
20 patients with congestive heart failure who met the inclusion and exclusion criteria will be recruited. After being fully informed and signed the informed consent, the patients will be randomly divided into two dosage groups: 100 million cells (10 patients) and 400 million cells (10 patients). Human iPSC-derived cardiomyocytes will be injected into the myocardium through a transcatheter endocardial injection system.
| Study group | Role | What they receive |
|---|---|---|
| hiPSC-CM therapy low dosage | Experimental | Not described Receives: Biological: hiPSC-CM therapy |
| hiPSC-CM therapy high dosage | Experimental | Not described Receives: Biological: hiPSC-CM therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of major serious adverse events (SAEs)
Incidence of SAEs is defined as the composite of: death, fatal myocardial infarction, stroke, tamponade, cardiac perforation, ventricular arrhythmias affecting hemodynamics (> 15s), and tumorigenicity related to the hiPSC-CM .
Measured: First month post-catheterization
Secondary outcomes (9)
Incidence of severe arrhythmia
Clinically significant arrhythmias will be recorded by 24-hour ambulatory electrocardiogram
Measured: 1-6 months post-catheterization
Incidence of newly formed tumors
by comparing chest, abdominal and pelvic CT scan and PET-CT scan
Measured: Baseline, 1,3,6 and 12 months post-catheterization
Changes in penal reactive antibodies (PRA)
Changes in penal reactive antibodies (PRA) as assessed via blooddraw
Measured: Baseline, 1, 3 and 6 months post-catheterization
Changes in donor specific antibodies (DSA)
Changes in donor specific antibodies (DSA) as assessed via blooddraw
Measured: Baseline, 1, 3 and 6 months post-catheterization
Overall Left Ventricular systolic performance as assessed by MRI
Left ventricular ejection fraction (LVEF), left ventricular end-diastolic dimension (LVEDV), left ventricular end-systolic dimension (LVESV), evaluated and compared to baseline values.
Measured: Baseline, 1, 3, 6 and 12 months post-catheterization
Overall Left Ventricular systolic performance as assessed by PET/ECT Scan
Myocardial contraction and relaxation and myocardial perfusion, evaluated and compared to baseline values.
Measured: Baseline, 6 and 12 months post-catheterization
Functional status by 6 minute walk test
valuate Functional Capacity via the Six Minute Walk Test
Measured: Baseline, 1,3,6 and 12 months post-catheterization
Functional status by New York Heart Association (NYHA) Classification
Evaluate Functional Capacity via New York Heart Association (NYHA) Class Determination
Measured: Baseline, 1,3,6 and 12 months post-catheterization
Minnesota Living With Heart Failure Questionnaire (MLHFQ)
Evaluate Quality Of Life Changes via Minnesota Living with Heart Failure (MLHF). The Maximum possible scores being 105 and the minimum 0. Higher scores indicate a worse or worsening quality of life, while lower scores or decreasing scores indicate a better quality of life.
Measured: Baseline, 1,3,6 and 12 months post-catheterization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2021
- Study start21 Sep 2021
- Primary completion30 Dec 2022 (estimated)
- Study completion31 Jul 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cardiovascular DiseasesStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- HELP Therapeutics Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Xijing Hospital
- Responsible party
- Sponsor
- Investigators
- Ling Tao, MD, PhD, Principal investigator, Xijing Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04982081
- Sponsor's protocol ID
- LTao
- Status last verified
- Sep 2022
- First submitted
- 1 Jul 2021
- Last updated
- 23 Sep 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treating Congestive Heart Failure Patients With Human iPSC-derived Cardiomyocytes Through Catheter-based Endocardial Injection
- ClinicalTrials.gov ID
- NCT04982081
- Acronym / sponsor ID
- LTao
- Keywords
- induced pluripotent stem cell, Congestive heart failure, Regenerative medicine, dilated cardiomyopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- HELP Therapeutics Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Xijing Hospital
- Study start
- 21 Sep 2021 (actual)
- Primary completion
- 30 Dec 2022 (estimated)
- Study completion
- 31 Jul 2023 (estimated)
- First posted
- 29 Jul 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.
Heart failure is the primary cause of morbidity and mortality worldwide. Currently drug treatments for heart failure manage the symptoms, but not restore the loss cardiomyocytes due to the very limited regenerative capability in the adult heart. Novel reparative therapies that replace the cardiomyocytes loss are highly demanded to restore the cardiac function. The main purposes of this explanatory study is to investigate the safety and efficacy of the catheter-based endocardial delivery of human iPSC-derived cardiomyocytes in patients with congestive heart failure.
Detailed description
Patients with severe congestive heart failure will be treated with allogeneic human iPSC-derived cardiomyocytes (HiCM-188) through the catheter-based injections. HiCM188, produced by Help therapeutics with cGMP condition, will be transplanted into the myocardium through a transcatheter endocardial injection system with two dosage (100 million cells or 400 million cells). The safety and efficacy assessments will be conducted at1, 3, 6 and 12 months after the cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| hiPSC-CM therapy low dosage | Experimental | Biological: hiPSC-CM therapy |
| hiPSC-CM therapy high dosage | Experimental | Biological: hiPSC-CM therapy |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients aged 18-75 years (including 18 and 75).
- Signed the informed consent.
- Patients with congestive heart failure who have received regular treatment for heart failure.
- New York Heart Association (NYHA) Class III or IV despite optimal standard of care
- Left Ventricular Ejection Fraction (LVEF)<40% as assessed by echocardiography ( measure in the 3 months of recruit is included, excluding the measured values within 1 month of myocardial infarction ）
Key exclusion criteria (25 listed)
- PRA ≥ 20% or DSA positive.
- Patients received treatments such as pacemakers, ICD or CRT device.
- Patient with severe valvular disease or presence of a mechanical valve replacement, such as PCI implantation, or patients requiring simultaneous radiofrequency ablation of atrial fibrillation.
- Patient with any therapeutic traumatic heart surgery within 30 days.
- Hemodynamic instability or cardiogenic shock.
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 |
|---|---|---|
| hiPSC-CM therapy20 patients with congestive heart failure who met the inclusion and exclusion criteria will be recruited. After being fully informed and signed the informed consent, the patients will be randomly divided into two dosage groups: 100 million cells (10 patients) and 400 million cells (10 patients). Human iPSC-derived cardiomyocytes will be injected into the myocardium through a transcatheter endocardial injection system. | Biological | hiPSC-CM therapy high dosage, hiPSC-CM therapy low dosage |
Primary outcome
Incidence of major serious adverse events (SAEs)
Time frame: First month post-catheterization
Incidence of SAEs is defined as the composite of: death, fatal myocardial infarction, stroke, tamponade, cardiac perforation, ventricular arrhythmias affecting hemodynamics (> 15s), and tumorigenicity related to the hiPSC-CM .
9 secondary outcomes
Incidence of severe arrhythmia
Time frame: 1-6 months post-catheterization
Incidence of newly formed tumors
Time frame: Baseline, 1,3,6 and 12 months post-catheterization
Changes in penal reactive antibodies (PRA)
Time frame: Baseline, 1, 3 and 6 months post-catheterization
Changes in donor specific antibodies (DSA)
Time frame: Baseline, 1, 3 and 6 months post-catheterization
Overall Left Ventricular systolic performance as assessed by MRI
Time frame: Baseline, 1, 3, 6 and 12 months post-catheterization
Overall Left Ventricular systolic performance as assessed by PET/ECT Scan
Time frame: Baseline, 6 and 12 months post-catheterization
Functional status by 6 minute walk test
Time frame: Baseline, 1,3,6 and 12 months post-catheterization
Functional status by New York Heart Association (NYHA) Classification
Time frame: Baseline, 1,3,6 and 12 months post-catheterization
Minnesota Living With Heart Failure Questionnaire (MLHFQ)
Time frame: Baseline, 1,3,6 and 12 months post-catheterization
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2022.
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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.
Ling Tao, MD, PhD principal investigator
Xijing Hospital
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 hiPSC-CM therapy in people with Cardiovascular Diseases and Congestive Heart Failure, run by HELP Therapeutics Co., Ltd. at 1 site in China.
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 7 inclusion and 25 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 23 Sep 2022. 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 China. 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 Jiaxian Wang, MD, PhD (+86-18565616060, wangjx@helptx.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of major serious adverse events (SAEs) (time frame: First month post-catheterization). 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 23 Sep 2022.