A Phase I/IIA, Multi-Centre, Open-Label, Dose-Escalation Study with Expansion Arms to Assess the Safety, Tolera-bility, Pharmacokinetics and Preliminary Efficacy of CB-103 Administered Orally in Adult Patients with Locally Ad-vanced or Metastatic Solid Tumour Malignancies Charac-terised by Alterations of the NOTCH Signalling Pathway
Ongoing - recruitment active · 2020 until 2030
This study is designed as an open label, non-randomised, uncontrolled Phase I/IIA dose escalation study with expan-sion cohorts of CB-103 administered orally on a once-daily schedule, based on a 28-day treatment cycle. The admin-istration schedule may be adapted during dose escalation (e.g. twice-daily, intermittent dosing schedule) depending on the PK and safety signals that occur. There will be two parts to this study. The aim of the Phase I part of the study (Part A) with the dose escalation is to determine the MTD/RP2D. An adaptive 2-parameter Bayesian logistic regression model (BLRM) for dose escalation with over-dose control (EWOC) will be used in Part A to guide de-termination of the MTD or the RP2D. Part A will be fol-lowed by the expansion Phase IIA (Part B of the study) to determine preliminary evidence of anti-tumour activity and to confirm the safety of the CB-103 MTD/RP2D in different expansion arms consisting of patients stratified into various pre-selected cancer indications at an advanced or meta-static stage of the disease.