A Phase 1 Study of Selinexor (KPT-330 IND#125052) a Selective XPO1 Inhibitor in Recurrent and Refractory Pediatric Solid Tumors Including CNS Tumors.
Research Study on an Investigational Medication in Children with Recurrent or Refractory Solid Tumors
Brief description of study.
The investigators will conduct a phase 1 trial of Selinexor in children with recurrent or refractory solid tumors, including CNS tumors using the Rolling Six design. The aims of the trial will be to establish the maximum tolerated pediatric doses of tablet formulation; to investigate the toxicities, pharmacokinetics, and pharmacodynamics of Selinexor in children with cancer; and to preliminarily explore efficacy in pediatric solid and CNS tumors, including medical and surgical expansion cohorts of HGG patients.
Detailed description of study
The investigators will conduct a phase 1 trial of Selinexor in children with recurrent or refractory solid tumors, including CNS tumors using the Rolling Six design. The aims of the trial will be to establish the maximum tolerated pediatric doses of tablet formulation; to investigate the toxicities, pharmacokinetics, and pharmacodynamics of Selinexor in children with cancer; and to preliminarily explore efficacy in pediatric solid and CNS tumors, including medical and surgical expansion cohorts of HGG patients.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Cancer,Solid Tumor
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Age: 1 years - 21 years
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Gender: All
Inclusion Criteria
Patients with recurrent or refractory solid tumors, including lymphoma and CNS tumors, are eligible.
Patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevations of CSF or serum tumor markers including alpha-fetoprotein or beta-HCG
Patients with recurrent or refractory high grade glioma (WHO Grade III/IV) including disseminated tumors (excluding DIPG), not requiring surgical resection
Patients with recurrent or refractory high grade glioma (WHO Grade III/IV) and requiring surgical resection (excluding DIPG and disseminated tumors)
Patients must have either measurable or evaluable disease on Part A
Parts B & C: Patients must have measurable disease on imaging
Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy
No evidence of active graft vs. host disease and at least 56 days must have elapsed after transplant or stem cell infusion
Patients must not have received prior exposure to Selinexor
Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
Patients must be able to swallow tablets whole
Exclusion Criteria
Pregnancy or Breast-Feeding Pregnant or breast-feeding women including a
Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible
Patients who are currently receiving another investigational drug are not eligible
Patients who are currently receiving other anti-cancer agents are not eligible
Patients who have an uncontrolled infection are not eligible
Patients who have received a prior solid organ transplantation are not eligible
Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible
Patients with known macular degeneration, uncontrolled glaucoma, or cataracts are not eligible
This study investigates an investigational medication in children with recurrent or refractory solid tumors, including tumors in the central nervous system (CNS). The purpose is to determine the highest dose that children can tolerate. The study will also look into how the medication affects the body, its side effects, and how it moves through the body.
Participants will take part in procedures that involve taking the investigational medication in tablet form. The study will monitor the effects of the medication, looking for any side effects and measuring how the body processes it. Participants will be grouped into different study arms based on their specific conditions.
- Who can participate: Children with recurrent or refractory solid tumors, including lymphoma and CNS tumors, may participate if they have verified malignancy and no prior exposure to the investigational medication.
- Study details: Participants will take the investigational medication in tablet form and will be monitored for side effects and how the medication is processed by their bodies. Participants will be grouped into different study arms based on their specific conditions. A placebo will not be used in this study.
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