A Phase I/II Study of GSK525762 in Patients with Persistent Hematologic Cancers

Full Title

A Phase I/II Open-label, Dose Escalation Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK525762 in Subjects with Relapsed, Refractory Hematologic Malignancies


Standard treatments for leukemia and myeloproliferative cancers include radiation therapy, chemotherapy, or both. Despite these therapies, the risk of the cancer coming back is high. Doctors are therefore seeking better treatments.

GSK525762 is an investigational drug that targets a family of proteins involved in leukemia growth called BET. It is hoped that targeting these proteins with GSK525762 can prevent leukemia cells from dividing.

The purpose of this study is to find the highest dose of GSK525762 that can be given safely in patients with leukemia and myeloproliferative cancers that continue to grow despite standard therapy. GSK525762 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 leukemia, myeloproliferative cancer, lymphoma, or myeloma which has continued to grow despite standard therapy, or patients must not have been candidates for standard therapy (such as some older patients).
  • Patients should have recovered from the serious side effects of prior treatment before entering the study.
  • Patients who have had an autologous stem cell transplant may participate if at least 3 months have passed since the transplant. Patients who had an allogeneic stem cell transplant may not participate.
  • Patients must be physically well enough that they are fully ambulatory, capable of all self care, and are capable of all but physically strenuous activities. As an example, patients must be well enough that they would be able to carry out office work or light housework.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.