Study Population
malignant glomus tumor, T-ALL/LBL, cardiac disease, tumor
GASTROINTESTINAL TUMORS, TUMOR, THROMBOEMBOLIC EVENT, BREAST CANCER, DISEASE, SOLID TUMORS, OSTEOSARCOMA, HEPATOCELLULAR CARCINOMA, CARDIAC DISEASE, ONCOLOGY
The study enrolled adults with histologically confirmed, locally advanced and/or metastatic solid tumors who had progressed on at least one line of prior systemic therapy (except for ACC) and relapsed/refractory T-ALL/LBL for whom no standard therapy was available. In dose escalation, participants with solid tumors with known or frequent Notch pathway–activating mutations were eligible (breast cancer, gastrointestinal tumors, hepatocellular carcinoma, osteosarcoma, malignant glomus tumor, and ACC), while the confirmatory cohort planned to enroll participants with selected tumor types (including T-ALL/LBL) and confirmed Notch pathway activation. Key eligibility criteria included patients ≥18 years of age with evaluable disease, Eastern Cooperative Oncology Group (ECOG) performance status 0–1, able to swallow capsules, and adequate organ function. Participants were excluded if they had clinically significant cardiac disease or thromboembolic events within the preceding 6 months, and drugs prolonging the QTc were avoided. A complete list of eligibility criteria is outlined in the Supplementary Materials and Methods.