Experimental Design:
toxicities, tumor, DLT, hematologic malignancies
DISEASE PROGRESSION, TUMOR, SOLID TUMORS, HEMATOLOGIC MALIGNANCIES, ADENOID CYSTIC CARCINOMA
Patients ≥18 years of age with selected advanced solid tumors [namely, adenoid cystic carcinoma (ACC)] and hematologic malignancies were eligible. CB-103 was dosed orally in cycles of 28 days at escalating doses until disease progression. Notch-activating mutations were required in a dose confirmatory cohort. Endpoints included dose-limiting toxicities (DLT), safety, tumor response, pharmacokinetics, and pharmacodynamics. Exploratory analyses focused on correlates of Notch and target gene expression.