CDK4/6 Inhibition in Locally Advanced/Metastatic Chordoma
Status:
Unknown status
Trial end date:
2021-07-31
Target enrollment:
Participant gender:
Summary
In chordoma cell lines and patient biopsies, the p16 (CDKN2A) tumor suppressor is
consistently deleted. Thus, chordomas are an example of a tumor with universal activation of
the cyclin-dependent kinases 4 and 6 (CDK4/6) pathway, and experiments with patient-derived
chordoma cell lines demonstrate aberrant CDK4/6 activity downstream of p16 loss can be
efficiently inhibited by the CDK4/6 inhibitor palbociclib, resulting in reduced proliferation
and growth of neoplastic cells. The investigators aim to conduct a phase II clinical trial to
evaluate the efficacy of the small-molecule CDK4/6 inhibitor palbociclib in patients with
locally advanced/metastatic chordoma who are not candidates for standard therapy. The primary
objective is disease control in patients with chordoma treated with palbociclib as single
agent. The study design of this phase II study is based on a Simon two-stage design.