A Phase 1 Study of KTX-1001 in People With Multiple Myeloma


Full Title

A Phase 1 Study of KTX-1001, an Oral, First-In-Class, Selective, and Potent MMSET Catalytic Inhibitor that Suppresses H3K36me2 in Patients with Relapsed and Refractory Multiple Myeloma


Multiple myeloma that keeps growing after treatment can cause cancer cells to build up inside the bones. The cancer cells crowd out healthy blood cells and make abnormal proteins that cause discomfort. Researchers in this study want to find the best dose of KTX-1001 to treat multiple myeloma that grows after treatment.

KTX-1001 attacks the cells that make these abnormal proteins. It targets the activity of a mutated (changed) or overexpressed (too much is made) gene called MMSET. This gene helps turn healthy blood cells into cancer cells. KTX-1001 is taken orally (by mouth).

Who Can Join

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

  • Have multiple myeloma that came back or keeps growing after at least 3 regimens of treatment.
  • Have recovered from the serious side effects of prior therapies before taking KTX-1001.
  • 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 or to see if you can join this study, please call Dr. Saad Usmani’s office at 646-608-4165.




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID