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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

Tracking Information
First Submitted Date  ICMJE May 11, 2020
First Posted Date  ICMJE May 21, 2020
Last Update Posted Date February 21, 2021
Actual Study Start Date  ICMJE February 3, 2020
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 15, 2020)
  • 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.
  • 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure
Official Title  ICMJE Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Heart Failure
Intervention  ICMJE Biological: EHM implantation
Implantation of EHM on dysfunctional left or right ventricular myocardium in patients with HFrEF (EF <35%).
Study Arms  ICMJE EHM Implantation
All patients will receive EHM implant
Intervention: Biological: EHM implantation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 15, 2020)
53
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2024
Estimated Primary Completion Date March 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04396899
Other Study ID Numbers  ICMJE 02289
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Karsten Gavenis, University Medical Center Goettingen
Study Sponsor  ICMJE University Medical Center Goettingen
Collaborators  ICMJE
  • Deutsches Zentrum für Herz-Kreislauf-Forschung (DZHK)
  • University Medical Center Freiburg
Investigators  ICMJE
Principal Investigator: Tim Seidler, Prof. University Medical Center Goettingen
PRS Account University Medical Center Goettingen
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP