Human Embryonic Stem Cell-Derived Cardiomyocyte Therapy for Chronic Ischemic Left Ventricular Dysfunction

NCT05068674 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 60978
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical study will utilize a new cell therapy approach (Human embryonic stem cells derived cardiomyocytes or hESC-CMs) to improve survival and cardiac function in patients with chronic left ventricular dysfunction secondary to MI (Myocardial Infarction).

Conditions

Chronic Ischemic Left Ventricular Dysfunction

Outcome Measures

Primary Outcomes (1)

• The maximum tolerated dose (MTD) among 3 dose levels of allogeneic human embryonic stem cell-derived cardiomyocytes (hESC-CMs)

  The primary endpoints are safety and feasibility. The feasibility of preparing and delivering the study product, as well as collecting cardiac MRI variables in subjects will be assessed.

  3 Years

Study Arms (3)

Cohort 1

ACTIVE COMPARATOR

Low dose (50M cells)

Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 50M cells

Cohort 2

ACTIVE COMPARATOR

Medium dose (150M cells)

Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 150 cells

Cohort 3

ACTIVE COMPARATOR

High dose (300M cells)

Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 300M cells

Interventions

Human Embryonic Stem Cell-Derived Cardiomyocyte 50M cells DRUG

50 million (M) cells delivered in a dose of 5M cells per injection over 10 injections.

Also known as: Human ESC-CMs

Cohort 1

Human Embryonic Stem Cell-Derived Cardiomyocyte 150 cells DRUG

150M cells delivered in a dose of 15M cells per injection over 10 injections

Also known as: Human ESC-CMs

Cohort 2

Human Embryonic Stem Cell-Derived Cardiomyocyte 300M cells DRUG

300M cells delivered in a dose of 30M per injection over 10 injections

Also known as: Human ESC-CMs

Cohort 3

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Be ≥ 21 and \< 80 years of age.
• Provide written informed consent.
• Have a diagnosis of chronic ischemic left ventricular dysfunction secondary to MI as defined by previous myocardial infarction documented by an imaging study demonstrating coronary artery disease with corresponding areas of akinesis, dyskinesis, or severe hypokinesis.
• Be a candidate for cardiac catheterization within 5 to 10 weeks of screening.
• Have been treated with appropriate maximal medical therapy for heart failure or postinfarction left ventricular dysfunction. For beta-blockade, the patient must have been on a stable dose of a clinically appropriate beta-blocker for 3 months. For angiotensinconverting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) or angiotensin receptor neprilysin inhibitors (ARNIs) or have appropriate medical indication precluding use of one or both of these agents, the patient must have been on a stable dose of a clinically appropriate agent for 1 month or within no more than doubling the dose of any of ARB, ACE inhibitors, and ARNIs over the last 3 months.
• Left ventricular ejection fraction below 40%.
• Class II/III NYHA symptoms of heart failure within the 6 months prior to baseline testing.
• Hospitalization in the past 6 months or NT pro-BNP \> 1200 pg/mL, or \>1600 pg/mL if atrial fibrillation was present.
• Automated implantable cardioverter-defibrillator (AICD) in place.

You may not qualify if:

• Have a baseline glomerular filtration rate \< 35 ml/min/1.73 m2
• Have a known, serious radiographic contrast allergy.
• Have a prosthetic aortic valve or heart constrictive device.
• Have a documented presence of aortic stenosis (aortic stenosis graded as 1.5 cm2 or less).
• Have a documented presence of moderate to severe aortic insufficiency (echocardiographic assessment of aortic insufficiency graded as ≥+2).
• Have evidence of a life-threatening arrhythmia in the absence of a defibrillator (nonsustained ventricular tachycardia ≥ 20 consecutive beats or complete second- or third-degree heart block in the absence of a functioning pacemaker) or QTc interval \> 550 ms on screening ECG.
• AICD firing in the past 60 days prior to enrollment.
• Be eligible for or require coronary artery revascularization.
• Have a hematologic abnormality as evidenced by hematocrit \< 25%, white blood cell \< 2,500/µl, or platelet values \< 100,000/µl without another explanation.
• Have liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times the ULN.
• Have a coagulopathy (INR \> 1.3) not due to a reversible cause (i.e., Coumadin). Patients on Coumadin will be withdrawn 5 days before the procedure and confirmed to have an INR \< 1.3. Patients who cannot be withdrawn from Coumadin will be excluded from enrollment.
• Have known allergies to penicillin or streptomycin.
• Be an organ transplant recipient.
• Have a history of organ or cell transplant rejection.
• Have a clinical history of malignancy within 5 years (i.e., patients with prior malignancy must be disease-free for 5 years), except curatively-treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma.

Sponsors & Collaborators

• Joseph C. Wu: lead
• California Institute for Regenerative Medicine (CIRM): collaborator

Study Sites (1)

Stanford Hospital and Clinics

Palo Alto, California, 94305, United States

RECRUITING

MeSH Terms

Interventions

Cell Count

Intervention Hierarchy (Ancestors)

Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Cell Physiological Phenomena

Central Study Contacts

Joseph C. Wu, MD, PhD

CONTACT

(650) 736-2246; joewu@stanford.edu

Evgenios Neofytou, MD

CONTACT

(650) 736-2246; neofytou@stanford.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine and Radiology.

Model Details: Phase I will be a standard 3+3 dose-escalation study to evaluate 3 doses of allogeneic hESC-CMs

Study Record Dates

• First Submitted: September 24, 2021
• First Posted: October 6, 2021
• Study Start: March 22, 2022
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 1, 2028
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)