ADV1414: Phase I Study of Selinexor to Treat Patients with Recurrent and Refractory Pediatric Solid Tumors

A Phase I Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

Categories:

Solid Tumor

Phase I/II

Diseases Treated:

Solid Tumors

Eligibility Overview:

  • Diagnosis of solid tumor that is resistant to other anticancer therapy or has come back after therapy. Includes lymphoma and central nervous system (CNS) tumors that may or may not require surgery.
  • Fully recovered from the acute toxic effects of all prior anticancer therapy
  • At least 1 but not more than 21 years of age
  • Able to swallow tablets whole
  1. Brief Summary

    This study will test the effects of selinexor (KPT-330) on children between the ages of 1 and 21 who have recurrent and refractory solid tumors, including lymphoma and CNS tumors. Children with these kinds of aggressive tumors have few treatment options and have a high rate of mortality. Selinexor is an experimental drug that has shown some promise in the lab as a treatment for acute myeloid leukemia, sarcoma, and other solid tumors. It has been administered to adults in Phase 1 trials with no severe, unmanageable side effects. This study will determine how much selinexor may be safely given to children who have recurrent and refractory brain and solid tumors. The trial will also examine selinexor’s impact on the rest of the body and its effects on tumor tissue of patients with recurrent or refractory high grade gliomas (HGG).

    Primary Objectives

    • To determine the recommended phase 2 dose or the maximum tolerated dose of the tablet form of selinexor in children with recurrent or refractory solid and CNS tumors
    • To describe the toxic effects of selinexor 
    • To characterize the pharmacokinetics of the tablet form of selinexor

    Eligibility Criteria

    Inclusion criteria include:

    • At least 1 but not more than 21 years old
    • Able to swallow tablets whole
    • Diagnosed with one of the following:
      • Recurrent or refractory solid tumor, including lymphoma and CNS tumors
      • Intrinsic brain stem tumor, optic pathway glioma, or a pineal tumor and elevations of CSF or serum tumor markers
      • Recurrent or refractory high grade glioma, including disseminated tumors (excluding DIPG), not requiring surgical resection
      • Recurrent or refractory HGG requiring surgical resection (excluding DIPG and disseminated tumors) and is medical stable to receive 3-4 doses of selinexor before undergoing surgery

    Exclusion criteria include:

    • Pregnant or breast-feeding
    • Receiving corticosteroids and has not been on a stable or decreasing dose for at least 7 days prior to enrollment
    • Currently taking another investigational drug
    • Currently receiving other anti-cancer agents
    • Has had a solid organ transplant
    • BMI less than 3rd percentile for age
    • Macular degeneration, uncontrolled glaucoma or cataracts
    • Body surface area less than 0.84m2

    Study Sites

    St. Jude Children’s Research Hospital
    Memphis, Tennessee

    Collaborating sites in the U.S.

  2. About this Clinical Trial

    There are very few, effective treatments for aggressive pediatric brain and solid tumors. These tumors include those that come back after treatment, also known as recurrent, and tumors that don’t respond to therapy, also known as refractive. 

    Researchers have tested new, experimental drugs to find a treatment that may fight these kinds of tumors better. One of those drugs is called selinexor, also known as KPT-330. Selinexor has shown promise in fighting cancer in test tubes and animals. It has also been used in adults with no severe side effects, but doctors don’t yet know if younger patients will respond in the same way. St. Jude is offering this Phase I study to see how selinexor can benefit children, adolescents and young adults with these kinds of tumors.

    Purpose of this Clinical Trial

    The main goal of this study is to find out how much selinexor may be given safely to children who have these kinds of aggressive tumors without causing severe side effects. Researchers also want to find out how well selinexor treats these tumors and want to learn more about the drug’s effects on the rest of the body.

    Eligibility Overview

    • Diagnosis of solid tumor that did not respond to treatment or came back after treatment, including lymphoma and central nervous system tumors
    • At least 1 but not more than 21 years of age
    • Able to swallow tablets whole
  3. ADV1414 Quick View
    Sponsor Children's Oncology Group
    ClinicalTrials.gov identifier NCT02323880
    Trial start date October 2015
    Estimated enrollment 75
    Study type Interventional
    Study phase Phase I
    Conditions Solid tumors
    Ages 1 to 21 years old
    Principal investigator Wayne L. Furman, MD
    Study sites St. Jude Children’s Research Hospital and collaborating sites in the U.S.
    For a consultation or to discuss ADV1414 St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

Contact

Wayne L. Furman, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.