Selinexor in Treating Younger Patients With Recurrent or Refractory Solid Tumors or High-Grade Gliomas
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|ClinicalTrials.gov Identifier: NCT02323880|
Recruitment Status : Recruiting
First Posted : December 24, 2014
Last Update Posted : September 12, 2017
|Condition or disease||Intervention/treatment||Phase|
|Childhood Central Nervous System Neoplasm Childhood Lymphoma Childhood Solid Neoplasm Malignant Glioma Recurrent Brain Neoplasm||Other: Pharmacological Study Drug: Selinexor||Phase 1|
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 central nervous system (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.
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) twice weekly (days 1, 3, 8, 10, 15, and 17). Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||81 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Study of Selinexor (KPT-330), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors|
|Study Start Date :||October 2015|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||March 2021|
Experimental: Treatment (selinexor)
Patients receive selinexor PO twice weekly (days 1, 3, 8, 10, 15, and 17). Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.
Other: Pharmacological Study
- Incidence of adverse events graded according to NCI CTCAE version 4.0 [ Time Frame: Up to 30 days after completion of study treatment ]
- Pharmacokinetics (PK) of selinexor [ Time Frame: Pre-dose and 30 minutes and 1, 2, 3, 4, 6, 8, and 24 hours after the first dose on day 1 of course 1 ]The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).
- RP2D or MTD of selinexor [ Time Frame: Up to 28 days ]The MTD will be the maximum dose at which fewer than one-third of patients experience dose limiting toxicity, graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0.
- Antitumor effect of selinexor [ Time Frame: Up to 30 days after completion of study treatment ]
- Penetration, pharmacodynamic effects, and biologic effects of selinexor in tumor tissue of patients with recurrent/refractory HGG requiring resection [ Time Frame: Up to time of surgical resection during course 1 ]
- PFS (surgical patients) [ Time Frame: Six months from the start of treatment ]
- Pharmacodynamics of selinexor [ Time Frame: Pre-dose and 4 hours after dose on day 1 of course 1 ]
- Rate of objective radiographic response (medical patients) [ Time Frame: Up to 30 days after completion of study treatment ]Response will be defined as either partial response or complete response on two consecutive 2-dimensional measurements on standard imaging done 4 weeks apart.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02323880
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|Principal Investigator:||Julia Glade-Bender||COG Phase I Consortium|