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Shifts in neuroendocrine cancer clinical trial design

Nov. 2, 2021, 8:00 AM

Neuroendocrine neoplasms (NENs) — heterogeneous tumors that can occur anywhere in the body — have been increasing in incidence. Despite NEN heterogeneity, clinical trials have historically grouped them together. 

With recent advances in pathology classification and approval of targeted therapies, Satya Das, MD, MSCI, and colleagues sought to determine if NEN clinical trial designs have changed. 

The researchers conducted a systematic survey of completed studies since 2000 and compared studies in the first decade (2000-2009) to the second (2010-2020). 

They reported Oct. 27 in JAMA Network Open that of 117 evaluated studies, those that began enrollment after 2010 were more likely to focus on select types of NENs, specify tumor differentiation, and use progression-free survival rather than objective response rate as an end point. 

Although this quality improvement study noted shifts in trial design, more than 20% of studies still included all NENs. The authors assert that studying novel agents in specific NENs may enhance drug development in this field.

Other Vanderbilt investigators on the study include Liping Du, PhD, Cody Lee, MD, Nina Arhin, MD, Jordan Berlin, MD, and Heather LaFerriere, MLIS. 

This work was supported by a Neuroendocrine Tumor Research Foundation Investigator award and a Vanderbilt-Ingram Cancer Center GI SPORE Career Enhancement grant (CA236733), both to Das.

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