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

NCT ID: NCT04396899

Last Updated: 2025-05-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-02-03

Study Completion Date

2027-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Heart Failure

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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EHM Implantation

All patients will receive EHM implant

Group Type OTHER

EHM implantation

Intervention Type BIOLOGICAL

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

Interventions

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EHM implantation

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. HFrEF (EF ≤ 35%) as assessed by high-resolution echocardiography or MRI
2. No realistic chance or not eligible for heart transplantation
3. At least one hypo- or dyskinetic segment to demark the implant target area
4. 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) with concomitant RV dysfunction, diagnosed using the Tricuspid Annular Plane Systolic Excursion (TAPSE) index \<16 mm (Rudski et al. 2010).
5. 18-80 years of age
6. Previous implantation of an ICD or CRT-D with event recorder
7. New York Heart Association (NYHA) Class III or IV under optimal medical therapy
8. Willingness and ability to give written informed consent
9. Female subjects of childbearing potential must agree to use acceptable method(s) of contraception for the full study duration.

Exclusion Criteria

1. Contraindication to immunosuppressive drugs (e.g. known history of unresolved cancer, hepatitis B/C, HIV, HTLV1)
2. Alloimmunisation against EHM implant cells
3. Hypertrophic cardiomyopathy (HCM)
4. Terminal kidney failure (stage 4; GFR \<30 ml/min)
5. Terminal liver failure
6. Autoimmune disease
7. History of stroke
8. Reduced life expectancy in the short term due to non-cardiac disease
9. Simultaneous participation in another interventional trial
10. Pregnant or breastfeeding females
11. Known or suspected alcohol and/or drug abuse
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Deutsches Zentrum für Herz-Kreislauf-Forschung (DZHK)

OTHER

Sponsor Role collaborator

University Medical Center Freiburg

OTHER

Sponsor Role collaborator

Repairon GmbH

UNKNOWN

Sponsor Role collaborator

University Medical Center Goettingen

OTHER

Sponsor Role lead

Responsible Party

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Karsten Gavenis

on behalf of Principal Investigator Prof. Seidler and Study Director Prof. Zimmermann

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Tim Seidler, Prof.

Role: PRINCIPAL_INVESTIGATOR

University Medical Center Goettingen

Wolfram-Hubertus Zimmermann, Prof.

Role: STUDY_DIRECTOR

University Medical Center Goettingen

Locations

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University Medical Center Göttingen

Göttingen, Lower Saxony, Germany

Site Status RECRUITING

Herz- und Diabeteszentrum Nordrhein-Westfalen

Bad Oeynhausen, North Rhine-Westphalia, Germany

Site Status NOT_YET_RECRUITING

University Medical Center Schleswig-Holstein

Lübeck, Schleswig-Holstein, Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Wolfram-Hubertus Zimmermann, Prof.

Role: CONTACT

+49 551 / 3965781

Florian Walker, Dr.

Role: CONTACT

+49 551 / 3960825

Facility Contacts

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Tim Seidler, Prof.

Role: primary

Jan Gummert, Prof.

Role: primary

Stephan Ensminger, Prof.

Role: primary

Other Identifiers

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02289

Identifier Type: -

Identifier Source: org_study_id

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