Clinical trial design methodologies for advanced sarcoma therapy

Bradley J. Petek, Seth M. Pollack, Anastasia Constantinidou, Robin L. Jones*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review


Sarcomas are rare and heterogeneous mesenchymal tumors affecting connective tissue. For many years, doxorubicin-based chemotherapy has been the main systemic therapy option for patients with metastatic soft tissue sarcoma. This 'one size fits all' approach has led to marginal benefit, but the introduction of tyrosine kinase inhibitors in gastrointestinal stromal tumors has shown that sub-type-specific, molecularly targeted therapy can lead to significant advances. Next generation sequencing has led to the discovery of the biologic nature of many histological sub-types of sarcomas, and now an emphasis has been placed on assessment of novel therapies for each sub-group. However, the transition into the clinic is both challenging and protracted due to the rarity and heterogeneity of these tumors. The high failure rate of phase III trials in oncology has shown that clinical trials can be difficult to run, especially in rare forms of cancer like sarcomas. In this review, the authors provide an evaluation of the potential approaches toward better clinical trial design in sarcoma studies.

Original languageEnglish (US)
Pages (from-to)72-75
Number of pages4
JournalClinical Research and Regulatory Affairs
Issue number2
StatePublished - Jun 1 2015
Externally publishedYes


  • Cancer treatment
  • Clinical trial design
  • Histological sub-type
  • Sarcoma
  • Systemic therapy

ASJC Scopus subject areas

  • Pharmacology
  • Pharmaceutical Science
  • Pharmacology (medical)


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