A Phase I Study of HDM201 in Patients with Advanced Cancers and a Normal TP53 Gene

Full Title

A Phase I, Open Label, Multicenter, Dose-escalation Study of Oral HDM201 in Adult Patients with Advanced Solid and Hematological Tumors Characterized by Wild-type TP53 (Version Date: 19-SEP-2016)


The p53 protein normally acts to control cell growth. Many tumor cells find ways to prevent p53 from working, however. HDM201 is an investigational drug that may restore normal function to the p53 protein, causing tumor cells to weaken or die. In order for the drug to work, patients must have a normal (“wild-type”) version of the gene that governs the production of p53, called TP53.

The purpose of this study is to find the highest dose of HDM201 that can be given safely in patients with advanced solid tumors and blood cancers and normal TP53. HDM201 is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have advanced solid tumors and blood cancers and normal TP53. Examples include colorectal cancer, melanoma, soft-tissue sarcoma, osteosarcoma, testicular cancer, renal cell carcinoma, acute myeloid leukemia, myelodysplastic syndromes, and acute lymphoblastic leukemia.
  • A sufficient amount of time must have passed since completion of prior therapy and entry into the study, ranging from 4 to 6 weeks depending on the treatment, and patients must have recovered from any serious side effects of therapy.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. David Hyman at 646-888-4226.