A Phase 1 Study of GLB-001 in People With Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome


Full Title

A First-in-human (FIH), Phase 1, Multicenter, Open-label, Dose Escalation and Expansion Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Orally Administered GLB-001 in Patients with Relapsed or Refractory Acute Myeloid Leukemia (RR AML) or Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes (RR HR-MDS)


In this study, researchers want to find the best dose of GLB-001 to treat leukemia. The people in this study have acute myeloid leukemia (AML) or higher-risk myelodysplastic syndrome (HR-MDS) that keeps growing after treatment.

GLB-001 breaks down the CK1-alpha protein, which plays an important role in cancer cell growth and survival. By breaking down this protein, GLB-001 may help slow or stop the growth of AML or HR-MDS. GLB-001 is taken orally (by mouth).

Who Can Join

To join this study, there are a few conditions. You must:

  • Have AML or HR-MDS that came back or keeps growing even after treatment.
  • Have recovered from the serious side effects of previous therapies before taking GLB-001.
  • Be able to walk and do routine activities for more than half the time you are awake.
  • Be age 18 or older.


For more information and to see if you can join this study, please call Dr. Eytan Stein’s office at 646-608-3749.




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID