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

A Phase I, Randomized Pilot Study of Human Embryonic Stem Cell-Derived Cardiomyocytes (hESC-CMs) in PaTients With ChrOnic Ischemic Left VentRicular Dysfunction Secondary to Myocardial Infarction (HECTOR)

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).

Study Overview

• Status: Recruiting
• Conditions: Chronic Ischemic Left Ventricular Dysfunction
• Intervention / Treatment: Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 50M cells; Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 150 cells; Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 300M cells

Detailed Description

The phase I dose-escalation pilot study is intended as an initial safety assessment to establish the MTD prior to the phase II randomized, double-blinded, placebo-controlled study. An estimated eighteen (18) patients in phase I who are scheduled to undergo cardiac catheterization and have met all inclusion/exclusion criteria will be enrolled.

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Joseph C. Wu, MD, PhD; Phone Number: (650) 736-2246; Email: joewu@stanford.edu
• Study Contact Backup: Name: Evgenios Neofytou, MD; Phone Number: (650) 736-2246; Email: neofytou@stanford.edu

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94305
      • Recruiting
      • Stanford Hospital and Clinics

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.
• Have a non-cardiac condition that limits lifespan to < 1 year.
• Be on chronic therapy with immunosuppressant medication, such as corticosteroids or TNFα antagonists.
• Be serum-positive for HIV, hepatitis BsAg, or viremic hepatitis C.
• Be currently participating (or participated within the previous 30 days) in an investigational therapeutic or device trial.
• Be a female patient who is pregnant, nursing, or have child-bearing potential but is not using effective birth control.
• Tested positive for SARS-CoV-2 within the last 30 days

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Cohort 1; Low dose (50M cells)	Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 50M cells; 50 million (M) cells delivered in a dose of 5M cells per injection over 10 injections.; Other Names: Human ESC-CMs
Active Comparator: Cohort 2; Medium dose (150M cells)	Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 150 cells; 150M cells delivered in a dose of 15M cells per injection over 10 injections; Other Names: Human ESC-CMs
Active Comparator: Cohort 3; High dose (300M cells)	Drug: Human Embryonic Stem Cell-Derived Cardiomyocyte 300M cells; 300M cells delivered in a dose of 30M per injection over 10 injections; Other Names: Human ESC-CMs

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Joseph C. Wu
• Collaborators: California Institute for Regenerative Medicine (CIRM)

Study record dates

Study Major Dates

• Study Start (Actual): March 22, 2022
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 1, 2028

Study Registration Dates

• First Submitted: September 24, 2021
• First Submitted That Met QC Criteria: September 24, 2021
• First Posted (Actual): October 6, 2021

Study Record Updates

• Last Update Posted (Estimated): September 29, 2025
• Last Update Submitted That Met QC Criteria: September 25, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Ischemia; Ventricular Dysfunction; Ventricular Dysfunction, Left; Investigative Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Cytological Techniques; Cell Physiological Phenomena; Cell Count
• Other Study ID Numbers: 60978

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No