A Study of iPS Cell-derived Cardiomyocyte Spheroids (HS-001) in Patients With Heart Failure (LAPiS Study)

Phase 1

Active, not recruiting

• Conditions: Ischemic Heart Disease; Heart Failure
• Interventions: Biological: HS-001 CS; Device: HS-001-D needle, HS-001-D Adaptor

• Registration Number: NCT04945018
• Lead Sponsor: Heartseed Inc.

Brief Summary

The purpose of this clinical study is to evaluate the safety and efficacy of HS-001 CS transplanted into severe heart failure patients with underlying ischemic heart disease for 26 weeks after transplantation.

Detailed Description

This is a multicenter, open-label, two-group dose-escalation, phase I/II study in 10 severe heart failure patients (five in the low-dose group and five in the high-dose group) with underlying ischemic heart disease.

After screening period is completed, subjects undergo HS-001 CS transplantation. After transplantation, subjects take immunosuppressant and have efficacy/safety assessments.

Study Timeline

• Study First Submitted: 2021-06-22
• Study First Submitted QC: 2021-06-22
• Study First Posted: 2021-06-30
• Study Start: 2022-04-19
• Status Verified: 2025-01
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-13
• Primary Completion: 2025-07-31
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Patients with resting left ventricular ejection fraction (LVEF) ≦40% based on institutional assessment on either screening cardiac MRI or echocardiographic assessment
• New York Heart Association (NYHA) cardiac function classification of grade II or higher at screening
• Other Criteria apply, please contact the investigator

Exclusion Criteria

• Patients screened less than 1 month after the onset of myocardial infarction
• Patients with congenital heart disease, or cardiac sarcoidosis
• Other Criteria apply, please contact the investigator

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
HS-001 High dose	HS-001 CS	HS-001 High dose Administration
HS-001 High dose	HS-001-D needle, HS-001-D Adaptor	HS-001 High dose Administration
HS-001 Low dose	HS-001 CS	HS-001 Low dose Administration
HS-001 Low dose	HS-001-D needle, HS-001-D Adaptor	HS-001 Low dose Administration

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	26 weeks post-transplant	Adverse events and safety in the 26 weeks after HS-001 CS transplantation

Secondary Outcome Measures

Name	Time	Method
Left Ventricular Ejection Fraction in Cardiac MRI scan & Echocardiography	26 weeks and 52 weeks post-transplant	Left Ventricular Ejection Fraction in the 26 weeks and 52 weeks after HS-001 CS transplantation
Myocardial wall motion evaluation in Echocardiography	26 weeks and 52 weeks post-transplant	Myocardial wall motion evaluation (Index of myocardial strain) in the 26 weeks and 52 weeks after HS-001 CS transplantation
Kansas City Cardiomyopathy Questionnaire (KCCQ)	26 weeks and 52 weeks post-transplant	Kansas City Cardiomyopathy Questionnaire in the 26 weeks and 52 weeks after HS-001 CS transplantation
Myocardial blood flow in SPECT	26 weeks and 52 weeks post-transplant	Myocardial blood flow in the 26 weeks and 52 weeks after HS-001 CS transplantation
Myocardial viability in SPECT	26 weeks and 52 weeks post-transplant	Myocardial viability in the 26 weeks and 52 weeks after HS-001 CS transplantation
N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)	26 weeks and 52 weeks post-transplant	N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) in the 26 weeks and 52 weeks after HS-001 CS transplantation
6-minute walk distance	26 weeks and 52 weeks post-transplant	6-minute walk distance in the 26 weeks and 52 weeks after HS-001 CS transplantation
5-level EQ-5D version (EQ-5D-5L)	26 weeks and 52 weeks post-transplant	5-level EQ-5D version (EQ-5D-5L) in the 26 weeks and 52 weeks after HS-001 CS transplantation

Trial Locations

Locations (8)

Nihon University Itabashi Hospital; 🇯🇵 Tokyo, Japan

St. Marianna University Hospital; 🇯🇵 Kawasaki, Japan

Saitama Medical University International Medical Center; 🇯🇵 Saitama, Japan

Juntendo University Hospital; 🇯🇵 Tokyo, Japan

The University of Tokyo Hospital; 🇯🇵 Tokyo, Japan

Tokyo Medical and Dental University Medical Hospital; 🇯🇵 Tokyo, Japan

Tokyo Metropolitan Geriatric Medical Center; 🇯🇵 Tokyo, Japan

Tokyo Women's Medical University; 🇯🇵 Tokyo, Japan