Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure

Phase 1

• Conditions: Heart failure; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]; MedDRA version: 20.0Level: LLTClassification code: 10000803Term: Acute heart failure Class: 10007541

• Registration Number: CTIS2024-515708-38-01
• Lead Sponsor: niversitaetsmedizin Goettingen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-07-25
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 53

Inclusion Criteria

Heart failure with reduced ejection fraction (HFrEF with EF = 35%) as assessed by high-resolution echocardiography and MRI or CT., At least one hypo- or dyskinetic segment to demark the implant target area., Stable disease condition allowing for an elective left-lateral mini-thoracotomy (for LV applications) or open-chest surgery (for RV applications) for a clinically indicated intervention on the LV (e.g., coronary bypass surgery, valve repair, mechanical circulatory support device implantation) with concomitant RV dysfunction, diagnosed using the Tricuspid Annular Plane Systolic Excursion (TAPSE) index <16 mm (Rudski et al. 2010)., 18-80 years of age, Previous implantation of an ICD or CRT-D with event recorder, New York Heart Association (NYHA) Class III or IV under optimal medical therapy, Willingness and ability to give written informed consent, Female subjects of childbearing potential must agree to use acceptable method(s) of contraception for the full study duration.

Exclusion Criteria

Contraindication to immunosuppressive drugs (e.g. known history of unresolved cancer, hepatitis B/C, HIV, HTLV1), Simultaneous participation in another interventional trial, Pregnant or breastfeeding females, Known or suspected alcohol and/or drug abuse, Contraindication to TachoSil® (e.g. hypersenstitivity to human fibrinogen, human thrombin, horse collagen, human albumin, Riboflavin, Natriumchloride, Natriumcitrate, L-Arginin- Hydrochloride), Hypertrophic cardiomyopathy (HCM), Terminal kidney failure (stage 4; GFR <30 ml/min) at the time of enrolment, Terminal liver failure (Child-Pugh stage C; score >10) at the time of enrolment, Autoimmune disease, History of disabling stroke, Reduced life expectancy in the short term due to non-cardiac disease, Any condition that excludes adherence to study protocol (in particular lack of adherence to prescribed medication)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary objectiv is to assess safety and efficacy of Engineered Human Myocardium (EHM) in patients with terminal heart failure (HFrEF EF =35%) with or without RV dysfunction (TAPSE <16 mm);Secondary Objective: Secondary objective is to assess effects of EHM-grafts on disease-specific events and symptoms;Primary end point(s): Part A (Dose Escalation steps): Adverse events related to the procedure, including in particular arrhythmic events and worsening of disease progression within 28 days (based on a comparison of data obtained during visit 2 and visit 7), Part B: Adverse events related to the procedure, including in particular arrhythmic events and worsening of disease progression within the whole study duration, Evidence for structural and functional muscular augmentation of target myocardium determined as enhanced target heart wall thickness (HWT) and thickening fraction (HWTF)