RecruitingPhase 1Phase 2NCT04396899

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


Sponsor

University Medical Center Goettingen

Enrollment

53 participants

Start Date

Feb 3, 2020

Study Type

INTERVENTIONAL

Conditions

Summary

The BioVAT-HF trial will test the hypothesis that cardiomyocyte implantation via engineered heart muscle (EHM), the proposed investigational medicinal product (IMP; designated "Biological Ventricular Assist Tissue" or BioVAT), results in sustainable remuscularization and biological enhancement of myocardial performance in the failing heart. EHM are constructed from defined mixtures of induced pluripotent stem cell (iPSC)-derived cardiomyocytes and stromal cells in a bovine collagen type I hydrogel. Comprehensive preclinical testing confirmed the rationale for the clinical translation of the myocardial remuscularization strategy by EHM implantation. The patient target population for EHM therapy is patients suffering from advanced heart failure with reduced ejection fraction (HFrEF; EF: ≤35%) and no realistic option for heart transplantation.


Eligibility

Min Age: 18 YearsMax Age: 80 Years

Inclusion Criteria9

  • Symptomatic heart failure (NYHA II-IV) with reduced ejection fraction (HFrEF with LVEF ≤35%) as assessed by echocardiography.
  • Patients on guideline-directed medical therapy
  • NT-proBNP \>300 pg/mL for patients in sinus rhythm or \>900 pg/mL if in atrial fibrillation
  • History of previous heart failure hospitalization in the past 12 months
  • At least one hypo- or dyskinetic segment or dilated heart chamber to demark the implant target area
  • (A) Stable disease condition allowing for an elective left-lateral mini-thoracotomy (for LV applications) or (B) 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).
  • years of age
  • 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 Criteria11

  • Contraindication to immunosuppressive drugs (e.g. known history of unresolved cancer, hepatitis B/C, HIV, HTLV1)
  • Contraindication to TachoSil® (e.g. hypersensitivity 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
  • 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)
  • Simultaneous participation in another interventional trial
  • Pregnant or breastfeeding females
  • Known or suspected alcohol and/or drug abuse

Interventions

BIOLOGICALEHM implantation

Implantation of EHM on dysfunctional left or right ventricular myocardium in patients with HFrEF (EF \<35%).


Locations(3)

University Medical Center Göttingen

Göttingen, Lower Saxony, Germany

Herz- und Diabeteszentrum Nordrhein-Westfalen

Bad Oeynhausen, North Rhine-Westphalia, Germany

University Medical Center Schleswig-Holstein

Lübeck, Schleswig-Holstein, Germany

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NCT04396899


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