Clinical Trial Design for Investigational Cardio-Regenerative Therapy

Adv Exp Med Biol. 2018:1098:199-211. doi: 10.1007/978-3-319-97421-7_11.


Human trials of cardio-regenerative biologic therapies are being performed worldwide to address a growing, unmet need for durable treatments of cardiovascular disease. A well-constructed clinical trial design for these novel therapies requires careful attention to defining a clear hypothesis, a patient population, and anticipated outcomes. The scope of screening, method of randomization, blinding approach, data monitoring, and statistical analysis plan are the foundational elements that must be addressed in any clinical trial. Although the experience of human trials involving extracellular matrix constructs for cardiovascular disease treatment is limited, numerous lessons have been learned in the field of cell therapy that are translatable across all biologic treatment options. Future progress in this field may include testing combinations of cells, gene-transfer agents, and matrix and identifying treatment responders versus nonresponders.

Keywords: Cardiomyopathy; Clinical trial; Heart failure; Myocardial infarction; Regenerative medicine; Stem cell; Treatment.

Publication types

  • Review

MeSH terms

  • Cardiac Surgical Procedures / methods*
  • Cell Transplantation / methods*
  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / standards
  • Endpoint Determination
  • Extracellular Matrix*
  • Heart / physiology
  • Heart Diseases / surgery*
  • Heart Diseases / therapy
  • Humans
  • Regeneration
  • Regenerative Medicine / methods*
  • Research Design*
  • Sample Size
  • Tissue Engineering / methods*
  • Tissue Scaffolds
  • Treatment Outcome