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 : July 18, 2019
|Condition or disease||Intervention/treatment||Phase|
|Malignant Glioma Recurrent Brain Neoplasm Recurrent Childhood Central Nervous System Neoplasm Recurrent Childhood Glioblastoma Recurrent Lymphoma Recurrent Malignant Solid Neoplasm Refractory Central Nervous System Neoplasm Refractory Lymphoma Refractory Malignant Solid Neoplasm WHO Grade III Glioma||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|
|Actual Study Start Date :||October 5, 2015|
|Estimated Primary Completion Date :||March 31, 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
- Recommended phase 2 dose (RP2D) or maximum tolerated dose (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 (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE]) version 4.0.
- Incidence of adverse events [ Time Frame: Up to 30 days after completion of study treatment ]Will be graded according to NCI CTCAE version 4.0.
- 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).
- Antitumor effect of selinexor [ Time Frame: Up to 30 days after completion of study treatment ]Will be measured.
- Pharmacodynamics of selinexor [ Time Frame: Pre-dose and 4 hours after dose on day 1 of course 1 ]Will be measured.
- 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 ]Will be measured.
- 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.
- Progression free survival (surgical patients) [ Time Frame: Six months from the start of treatment ]Will be measured.
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
Hide Study Locations
|United States, Alabama|
|Children's Hospital of Alabama||Recruiting|
|Birmingham, Alabama, United States, 35233|
|Contact: Site Public Contact 205-638-9285 email@example.com|
|Principal Investigator: Gregory K. Friedman|
|United States, California|
|Children's Hospital Los Angeles||Recruiting|
|Los Angeles, California, United States, 90027|
|Contact: Site Public Contact 323-361-4110|
|Principal Investigator: Leo Mascarenhas|
|Children's Hospital of Orange County||Recruiting|
|Orange, California, United States, 92868|
|Contact: Site Public Contact 714-997-3000|
|Principal Investigator: Josephine H. Haduong|
|UCSF Medical Center-Mission Bay||Recruiting|
|San Francisco, California, United States, 94158|
|Contact: Site Public Contact 877-827-3222|
|Principal Investigator: Kieuhoa T. Vo|
|United States, Colorado|
|Children's Hospital Colorado||Recruiting|
|Aurora, Colorado, United States, 80045|
|Contact: Site Public Contact 303-764-5056 firstname.lastname@example.org|
|Principal Investigator: Margaret E. Macy|
|United States, District of Columbia|
|Children's National Medical Center||Recruiting|
|Washington, District of Columbia, United States, 20010|
|Contact: Site Public Contact 202-884-2549|
|Principal Investigator: Jeffrey S. Dome|
|United States, Georgia|
|Children's Healthcare of Atlanta - Egleston||Recruiting|
|Atlanta, Georgia, United States, 30322|
|Contact: Site Public Contact 404-785-2025 Leann.Schilling@choa.org|
|Principal Investigator: Jason R. Fangusaro|
|United States, Illinois|
|Lurie Children's Hospital-Chicago||Recruiting|
|Chicago, Illinois, United States, 60611|
|Contact: Site Public Contact 773-880-4562|
|Principal Investigator: Stewart Goldman|
|United States, Indiana|
|Riley Hospital for Children||Recruiting|
|Indianapolis, Indiana, United States, 46202|
|Contact: Site Public Contact 800-248-1199|
|Principal Investigator: James M. Croop|
|United States, Massachusetts|
|Dana-Farber Cancer Institute||Recruiting|
|Boston, Massachusetts, United States, 02215|
|Contact: Site Public Contact 877-442-3324|
|Principal Investigator: Suzanne Shusterman|
|United States, Michigan|
|C S Mott Children's Hospital||Recruiting|
|Ann Arbor, Michigan, United States, 48109|
|Contact: Site Public Contact 800-865-1125|
|Principal Investigator: Rajen Mody|
|United States, Minnesota|
|University of Minnesota/Masonic Cancer Center||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Site Public Contact 612-624-2620|
|Principal Investigator: Emily G. Greengard|
|United States, Missouri|
|Washington University School of Medicine||Recruiting|
|Saint Louis, Missouri, United States, 63110|
|Contact: Site Public Contact 800-600-3606 email@example.com|
|Principal Investigator: Robert J. Hayashi|
|United States, New York|
|NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center||Recruiting|
|New York, New York, United States, 10032|
|Contact: Site Public Contact 212-305-6361 firstname.lastname@example.org|
|Principal Investigator: Alice Lee|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center||Recruiting|
|Cincinnati, Ohio, United States, 45229|
|Contact: Site Public Contact 513-636-2799 email@example.com|
|Principal Investigator: James I. Geller|
|United States, Oregon|
|Oregon Health and Science University||Suspended|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia||Recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Site Public Contact 267-425-5544 CancerTrials@email.chop.edu|
|Principal Investigator: Elizabeth Fox|
|Children's Hospital of Pittsburgh of UPMC||Recruiting|
|Pittsburgh, Pennsylvania, United States, 15224|
|Contact: Site Public Contact 412-692-8570 firstname.lastname@example.org|
|Principal Investigator: Jean M. Tersak|
|United States, Tennessee|
|St. Jude Children's Research Hospital||Recruiting|
|Memphis, Tennessee, United States, 38105|
|Contact: Site Public Contact 866-278-5833 email@example.com|
|Principal Investigator: Wayne L. Furman|
|United States, Texas|
|Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Site Public Contact 713-798-1354 firstname.lastname@example.org|
|Principal Investigator: Jodi Muscal|
|United States, Washington|
|Seattle Children's Hospital||Recruiting|
|Seattle, Washington, United States, 98105|
|Contact: Site Public Contact 866-987-2000|
|Principal Investigator: Julie R. Park|
|United States, Wisconsin|
|Children's Hospital of Wisconsin||Recruiting|
|Milwaukee, Wisconsin, United States, 53226|
|Contact: Site Public Contact 414-955-4727 MACCCTO@mcw.edu|
|Principal Investigator: Paul D. Harker-Murray|
|Principal Investigator:||Julia Glade-Bender||COG Phase I Consortium|