Data Studio

Li Ka Shing Learning Center (LKSC)
Rm 209

DATE: October 18, 2017
TIME: 1:30 - 3:00 pm
TITLE: Basket Trials for Oncology Studies

Shivaani Kummar, Professor, Medicine & Radiology

Jason C. Paik, Fellow in Hematology and Oncology, Stanford Cancer Institute

Teresa Purzner, Bio-X SIGF Graduate Student Fellow, Developmental Biology



Many new cancer therapies target molecular alterations in cancer cells by inhibiting a signaling pathway, and the target may occur in multiple types of cancer. Ideally, the Phase II clinical trials can evaluate which cancers are sensitive to the therapy. This leads to a basket design for the clinical trial, in which each basket consists of patients with a specific cancer and whose cancer harbors the target. This consultation will focus on a two-stage design for a therapy that targets the Hedgehog signaling pathway, which is linked to basal cell carcinoma and some medulloblastoma. In the first stage, efficacy is evaluated in each basket. At the interim analysis, the homogeneity of the efficacy measure is evaluated to determine whether the baskets are to be aggregated in the second stage. The investigators have three primary questions about this study design. How do we achieve adequate power given that both the cancers and molecular alterations are rare? How do we combine baskets to ensure adequate power? How do we decide when to conduct the interim analysis given that the baskets have different recruitment rates?

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Suggested readings:

Stephan Teglund and Rune Toftgård. Hedgehog beyond medulloblastoma and basal cell carcinoma. Biochimica et Biophysica Acta (BBA)-Reviews on Cancer 2010; 1805:181-208.

Richard Simon. Critical Review of Umbrella, Basket and Platform Designs for Oncology Clinical Trials. Clinical Pharmacology & Therapeutics 2017.

Kristen M. Cunanan et al. An efficient basket trial design. Statistics in medicine 2017; 36:1568-1579.