Unique Aspects of the Design of Phase I/II Clinical Trials of Stem Cell Therapy

Ivonne Schulman, Russell Saltzman, Daniel DaFonseca, Lina Caceres, Cindy Delgado, Marietsy Pujol, Kevin Ramdas, Jairo Tovar, Mayra Vidro-Casiano, Joshua Hare and Wayne Balkan

A chapter in The Management of Clinical Trials from IntechOpen

Abstract: This chapter will review the unique aspects and limitations of the design of phase I/II (safety and efficacy) clinical trials of stem cell therapy. Although the classical pharmacologic principles applicable to drugs are not applicable to biologic (live cell) therapeutic agents, an important stage in the development of any new therapeutic agent is the establishment of an optimal dosage and delivery route. This can be particularly challenging when the treatment is a biologic agent, such as stem cells, that may exert its therapeutic effects via complex or poorly understood mechanisms. To date, clinical studies have shown inconsistent findings regarding the relationship between cell dose and clinical outcomes. This can be at least partially attributed to variations in donor cell type, source, characteristics, dosing/concentration, delivery route, underlying mechanisms of action, and efficacy endpoints tested. The current recommendations will be reviewed herein to give new investigators a general understanding of the unique issues that need to be considered and addressed when designing a stem cell therapy phase I/II clinical trial.

Keywords: phase I/II clinical trial; regenerative medicine; stem cells (search for similar items in EconPapers)
JEL-codes: I18 (search for similar items in EconPapers)
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Persistent link: https://EconPapers.repec.org/RePEc:ito:pchaps:128886

DOI: 10.5772/intechopen.72949

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