Clinical trial NCT04945018: A Study of iPS Cell-derived Cardiomyocyte Spheroids (HS-001) in Patients With Heart Failure (LAPiS Study)
Official title: A Phase I/II Study of Human Induced Pluripotent Stem (iPS) Cell-derived Cardiomyocyte Spheroids (HS-001) in Patients With Severe Heart Failure, Secondary to Ischemic Heart Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Heart Failure
Ischemic Heart Disease
Keywords: Heart Failure
Ischemic Heart Disease
Lead sponsor: Heartseed Inc.
Sponsor class: INDUSTRY
First posted: 2021-06-30
Enrollment: 10
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Safety and Tolerability
timeFrame: 26 weeks post-transplant
Secondary outcomes:
measure: Left Ventricular Ejection Fraction in Cardiac MRI scan & Echocardiography
timeFrame: 26 weeks and 52 weeks post-transplant
measure: Myocardial wall motion evaluation in Echocardiography
timeFrame: 26 weeks and 52 weeks post-transplant
measure: Myocardial blood flow in SPECT
timeFrame: 26 weeks and 52 weeks post-transplant
measure: Myocardial viability in SPECT
timeFrame: 26 weeks and 52 weeks post-transplant
measure: 6-minute walk distance
timeFrame: 26 weeks and 52 weeks post-transplant
measure: Kansas City Cardiomyopathy Questionnaire (KCCQ)
timeFrame: 26 weeks and 52 weeks post-transplant
measure: 5-level EQ-5D version (EQ-5D-5L)
timeFrame: 26 weeks and 52 weeks post-transplant
measure: N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)
timeFrame: 26 weeks and 52 weeks post-transplant
Other outcomes:
Locations:
city: Kawasaki
country: Japan
facility: St. Marianna University Hospital
city: Saitama
country: Japan
facility: Saitama Medical University International Medical Center
city: Tokyo
country: Japan
facility: Juntendo University Hospital
city: Tokyo
country: Japan
facility: Nihon University Itabashi Hospital
city: Tokyo
country: Japan
facility: The University of Tokyo Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical and Dental University Medical Hospital
city: Tokyo
country: Japan
facility: Tokyo Metropolitan Geriatric Medical Center
city: Tokyo
country: Japan
facility: Tokyo Women's Medical University
Study officials:
References: