A Phase I Study of ABL001 in Patients with Chronic Myelogenous Leukemia or Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia


Full Title

A Phase I, Multicenter, Open-label Study of Oral ABL001 in Patients with Chronic Myelogenous Leukemia or Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia


Tyrosine kinase inhibitors (TKIs) are medicines used to treat Philadelphia chromosome-positive (Ph+) leukemias by targeting Bcr-Abl, a protein which drives leukemia growth. However, it is not uncommon for Ph+ leukemias not to respond, to respond only partially, or to become resistant to these drugs. ABL001 is an investigational drug designed to deactivate Bcr-Abl by targeting it at a different location on the protein than currently available medicines (TKIs).

The purpose of this study is to find the highest doses of ABL001 that can be given safely to patients with chronic or accelerated phase chronic myelogenous leukemia (and later in the study, Ph+ acute lymphoblastic leukemia) that is not responding to other therapies. ABL001 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 chronic myelogenous leukemia or Ph+ acute lymphoblastic leukemia that is not responding to other therapies.
  • Patients must recover from the serious side effects of prior therapies before entering the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is open to patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Michael Mauro at 212-639-3107.