NCT02485535

Selinexor in Treating Patients With Intermediate- and High-Risk Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome After Transplant

Phase I Study to Assess the Tolerability and Efficacy of Selinexor (KPT-330) as Single Agent to Eliminate Minimal Residual Disease and Maintain Remission in Patients With AML and High Risk MDS After Allogeneic Stem Cell Transplantation

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: University of Chicago · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This phase I trial studies the side effects and best dose of selinexor when given after stem cell transplant in treating patients with acute myeloid leukemia that is at intermediate or high risk of spreading or coming back (intermediate- or high-risk), or myelodysplastic syndrome that is at high risk of spreading or coming back (high-risk). Selinexor works to stop cancer growth by blocking an enzyme, which may cause cancer cells to die and also kill cells that cause the cancer to grow, which commonly do not respond to regular chemotherapy.

Detailed description

PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) of selinexor in patients with hematologic malignancies, especially acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS), after allogeneic (allo)-stem cell transplant (SCT). SECONDARY OBJECTIVES: I. To evaluate the toxicities of selinexor as maintenance treatment after allo-SCT. II. To determine the incidence of non-relapse mortality. III. To determine 2 years post SCT progression-free survival (PFS) and overall survival rates. IV. To determine the incidence of acute and chronic graft-versus-host disease (GVHD). V. To assess lymphoid and myeloid chimerism post transplantation. TERTIARY OBJECTIVES: I. To analyze donor immune re-constitution after allo-SCT with selinexor maintenance. II. To monitor minimal residual disease (MRD) by Wilms tumor 1 (WT1) polymerase chain reaction (PCR) during selinexor treatment in AML/MDS patients. III. To characterize the physiopathology of the leukemia initiating cells (LIC) at the time of disease relapse on selinexor maintenance and compare that at initial diagnosis of the disease. OUTLINE: This is a dose-escalation study. Beginning on day 60-100 after allo-SCT without evidence of GVHD above grade 1 and disease relapse with stable hematopoietic recovery, patients receive selinexor orally (PO) on day 1 of each week or on days 1 and 3 of weeks 1-3. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 1 year.

Conditions

• Acute Myeloid Leukemia
• de Novo Myelodysplastic Syndrome
• Myelodysplastic Syndrome
• Secondary Acute Myeloid Leukemia
• Secondary Myelodysplastic Syndrome

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Selinexor	Given PO

Timeline

• Start date: 2015-09-04
• Primary completion: 2018-02-28
• Completion: 2020-07-01
• First posted: 2015-06-30
• Last updated: 2021-05-13

Locations

1 site across 1 country: United States