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

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ClinicalTrials.gov Identifier: NCT04396899
Recruitment Status : Recruiting
First Posted : May 21, 2020
Last Update Posted : February 21, 2021
Sponsor:
Collaborators:
Deutsches Zentrum für Herz-Kreislauf-Forschung (DZHK)
University Medical Center Freiburg
Information provided by (Responsible Party):
Karsten Gavenis, University Medical Center Goettingen

Brief 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.

Condition or disease Intervention/treatment Phase
Heart Failure Biological: EHM implantation Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 53 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure
Actual Study Start Date : February 3, 2020
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Failure

Arm Intervention/treatment
EHM Implantation
All patients will receive EHM implant
Biological: EHM implantation
Implantation of EHM on dysfunctional left or right ventricular myocardium in patients with HFrEF (EF <35%).




Primary Outcome Measures :
  1. Target heart wall thickness [ Time Frame: 12 months ]
    Target heart wall thickness (HWT) as determined by high resolution echocardiography and/or CINE-mode MRI (17-segment model). Primary efficacy analyses are based on the changes in HWT between baseline and 2 weeks, 1 month, 3 months, 6 months and 12 months after implantation. To test for a time effect a one-way Repeated Measures ANOVA will be performed for the primary endpoint. In case of detecting a time effect this is followed by Dunnett-type pairwise comparisons to baseline using paired t-Tests. Due to the explorative character of the efficacy analysis testing will be performed at a 10% two-sided significance level. Mean differences will be reported along with 90% confidence intervals.

  2. Heart wall thickening fraction [ Time Frame: 12 months ]
    Heart wall thickening fraction (HWTF) as determined by high resolution echocardiography and/or CINE-mode MRI (17-segment model). Primary efficacy analyses are based on the changes in HWTF between baseline and 2 weeks, 1 month, 3 months, 6 months and 12 months after implantation. To test for a time effect a one-way Repeated Measures ANOVA will be performed for the primary endpoint. In case of detecting a time effect this is followed by Dunnett-type pairwise comparisons to baseline using paired t-Tests. Due to the explorative character of the efficacy analysis testing will be performed at a 10% two-sided significance level. Mean differences will be reported along with 90% confidence intervals.



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04396899


Contacts
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Contact: Wolfram-Hubertus Zimmermann, Prof. +49 551 / 3965781 sekretariat.pharma@med.uni-goettingen.de
Contact: Florian Walker, Dr. +49 551 / 3960825 florian.walker@med.uni-goettingen.de

Locations
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Germany
University Medical Center Göttingen Recruiting
Göttingen, Lower Saxony, Germany, 37075
Contact: Tim Seidler, Prof.         
Herz- und Diabeteszentrum Nordrhein-Westfalen Not yet recruiting
Bad Oeynhausen, North Rhine-Westphalia, Germany, 32545
Contact: Jan Gummert, Prof.         
University Medical Center Schleswig-Holstein Not yet recruiting
Lübeck, Schleswig-Holstein, Germany, 23562
Contact: Stephan Ensminger, Prof.         
Sponsors and Collaborators
University Medical Center Goettingen
Deutsches Zentrum für Herz-Kreislauf-Forschung (DZHK)
University Medical Center Freiburg
Investigators
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Principal Investigator: Tim Seidler, Prof. University Medical Center Goettingen
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Responsible Party: Karsten Gavenis, on behalf of Principal Investigator Prof. Seidler, University Medical Center Goettingen
ClinicalTrials.gov Identifier: NCT04396899    
Other Study ID Numbers: 02289
First Posted: May 21, 2020    Key Record Dates
Last Update Posted: February 21, 2021
Last Verified: February 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Heart Failure
Heart Diseases
Cardiovascular Diseases