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Results summary of St. Jude clinical trial: 



Testing the addition of a new drug, selinexor (KPT-330), to chemotherapy in patients with advanced leukemia or myelodysplastic syndrome

Why was this study done?

Cancer is the uncontrolled growth of human cells. The growth of normal human cells is controlled by multiple processes. When cancer cells escape one or more of these processes, it leads to rapid growth.

One way cancer cells continue to grow is by getting rid of certain proteins called “tumor suppressor proteins” that normally cause cancer cells to die. Selinexor (KPT-330) works by trapping these proteins within the cell. That causes the cancer cells to die or stop growing.

This study was done to test the safety of selinexor and to find the highest dose of the drug that can be given safely when it is combined with two other chemotherapy (chemo) drugs. The study tested different doses of selinexor to see which dose is safer in children with advanced leukemia or myelodysplastic syndrome (MDS).

The study was done in two parts: Phase I and Phase II. The Phase I study aimed to find the highest safe dose of selinexor that could be given when combined with the other two drugs. The Phase II portion looked at how active was the drug combination in making the leukemia go away in patients with leukemia or MDS.

The study’s main goals were to find:

  • the highest safe dose of selinexor that could be given when combined with fludarabine and cytarabine
  • what side effects resulted when the drug combination was given
  • how the body absorbs, processes and removes the drug
  • how treatment with the drug affects the body

When was this study done?

The study opened August 2014 and closed in July 2019.

What did the study consist of?

  • Patients took the drug selinexor by mouth along with the intravenous drugs fludarabine and cytarabine.
  • Different doses of selinexor were given to groups of patients.
  • The doses increased for each group in the study until side effects required the dose to be lowered. This final dose was used in Phase II.

What did we learn from this study?

The study found that six doses of selinexor, given at 55 milligrams/m2 per dose, was well tolerated in combination with the two chemo drugs in children with acute leukemia that had come back or was hard to treat. The most common side effect was a lowering of sodium in the blood (not causing any symptoms). Two patients treated at higher doses also had transient side effects of difficulty talking, walking or coordination of movement.

In almost half (47%) of the patients treated we also observed that the leukemia cells responded to the treatment. This study and other promising studies show the combo is effective in high-risk leukemia and leukemia cells. Scientists will conduct more research to test the usefulness of selinexor-based therapy in children.

What are the next research steps as a result of this study?

The Phase II portion of this study further explored the combination of selinexor, fludarabine and cytarabine and its effects.

How does this study affect my child?

Although survival rates are near 90% for children with acute lymphoblastic leukemia and 60% for those with acute myeloid leukemia, patients with relapsed or hard-to-treat disease have dismal outcomes. Response rates to selinexor are promising and will be further studied.

Your child will receive information and guidance for care. Please speak with your St. Jude doctor about specific guidelines that apply to your child.

For more information

Please talk with your child’s doctor about questions or concerns you have as a result of this study.

Publication generated from this study:

Phase I Study of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination With Fludarabine and Cytarabine, in Pediatric Relapsed or Refractory Acute Leukemia. Thomas B. Alexander, John K. Choi, Raul C. Ribeiro, Ching-Hon Pui, and Jeffrey E. Rubnitz, St Jude Children's Research Hospital; Raul C. Ribeiro, Ching-Hon Pui, and Jeffrey E. Rubnitz, University of Tennessee College of Medicine, Memphis, TN; and Norman J. Lacayo, Lucile Packard Children's Hospital Stanford, Stanford, CA. J Clin Oncol. 2016 Dec;34(34):4094-4101.