NCT02323880

Selinexor in Treating Younger Patients With Recurrent or Refractory Solid Tumors or High-Grade Gliomas

A Phase 1 Study of Selinexor (KPT-330), A Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 12 Months – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase I trial studies the side effects and best dose of selinexor in treating younger patients with solid tumors or central nervous system (CNS) tumors that have come back (recurrent) or do not respond to treatment (refractory). Drugs used in chemotherapy, such as selinexor, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Detailed description

PRIMARY OBJECTIVES: I. To determine the recommended phase 2 dose (RP2D) or the maximum tolerated dose (MTD) of the tablet formulation of selinexor in children with recurrent/refractory solid and CNS tumors. II. To describe the toxicities of selinexor in children with recurrent/refractory solid and CNS tumors. III. To characterize the pharmacokinetics of the tablet formulation of selinexor in children with recurrent/refractory solid and CNS tumors. SECONDARY OBJECTIVES: I. To determine the antitumor effect of selinexor in a preliminary manner in children with recurrent/refractory solid and CNS tumors. II. To determine the pharmacodynamic properties of selinexor in children and adolescents with refractory solid tumors in plasma proteins and whole blood ribonucleic acid (RNA). III. To explore the penetration, pharmacodynamic effects, and biologic effects of selinexor in tumor tissue of patients with recurrent/refractory high-grade gliomas (HGG) requiring resection. IV. To further assess the toxicity and antitumor effects of selinexor in children with recurrent/refractory HGG in expanded cohorts following dose-escalation by measuring rate of objective radiographic response (medical patients) and rate of progression-free survival (PFS) six months from the start of treatment (surgical patients). OUTLINE: This is a dose escalation study. Patients receive selinexor orally (PO) on either a twice weekly (days 1, 3, 8, 10, 15, 17) or once weekly (days 1, 8, 15, and 22) schedule. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 30 days.

Conditions

• Malignant Glioma
• Recurrent Brain Neoplasm
• Recurrent Childhood Central Nervous System Neoplasm
• Recurrent Childhood Glioblastoma
• Recurrent Lymphoma
• Recurrent Malignant Solid Neoplasm
• Refractory Lymphoma
• Refractory Malignant Solid Neoplasm
• Refractory Primary Central Nervous System Neoplasm
• WHO Grade 3 Glioma

Interventions

Type	Name	Description
OTHER	Pharmacological Study	Correlative studies
DRUG	Selinexor	Given PO

Timeline

• Start date: 2015-10-30
• Primary completion: 2022-09-30
• Completion: 2024-12-31
• First posted: 2014-12-24
• Last updated: 2025-01-14
• Results posted: 2024-01-02

Locations

23 sites across 1 country: United States

Regulatory

• FDA-regulated drug study