A Phase I Study of the Safety, Pharmacokinetics and Pharmacodynamics of Escalating Doses of the Selective Inhibitor of Nuclear Export (SINE) KPT-330 in Patients with Advanced Hematological Malignancies

Study Status

Open to Enrollment

Study Description

This clinical trial is for men and women with relapsed/refractory advance hematological malignancies. The study is evaluating an experimental drug called KPT-330.

KPT-330 is a Selective Inhibitor of Nuclear Export (SINE) that irreversibly binds and inactivates CRM1, thereby forcing the nuclear retention of key tumor suppressor (TSP) and growth regulatory (GRP) proteins. Transient retention of TSP/GRP in the nucleus at high levels via CRM1 blockade activates their cell cycle checkpoint and genome surveying actions. This leads to the death of nearly all types of malignant cells, whereas normal cells undergo transient cell cycle arrest and recovery when the export block is released. 

The purposes of this research study are to find out more information such as: the highest dose of KPT-330 that can be given safely, the side effects it may cause, to examine how the body affects the study drug concentrations in the blood (pharmacokinetics or PK), to examine the effects of this study drug on the body (pharmacodynamics or PDn) and to gain some information on its effectiveness in treating cancer.

Disease Status and/or Stage

Advanced hematologic malignancies

Sponsor

NPM Pharma

Key Eligibility

  • Men and women age 18 and older
  • Patients with malignancies that are refractory to or who are intolerant of established therapy known to provide clinical benefit for their condition
  • Histologically confirmed diagnosis of the following conditions: Non-Hodgkin's Lymphoma (NHL); Chronic Lymphocytic Leukemia (CLL); Waldensrom's Macroglobulinemia (WM)

Principal Investigator

Dr. Peter Martin

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For general inquiries, or if you need assistance finding a study, please contact:

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Tel: (646) 962-8232
[email protected]

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