This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials.
Steroid-related side effects are much more severe in a pediatric population than they are in adults. The side effects appear more quickly and are often apparent in as few as 14 days. Dexamethasone side effects in children include rounding of the face, acne, increased body hair, muscle weakness, osteoporosis, mood changes, weight gain, fluid retention, glucose instability, high blood pressure, increased susceptibility to infection, stunted growth and aseptic necrosis of the hip joints14-16. Although extensive studies have been performed with Xerecept® in adults, no studies have been performed with Xerecept® in pediatric subjects. Therefore, this study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. The study will also explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
BID dosing, subcutaneous for 1 year
Children's Memorial Hospital, Northwestern University Feinberg School of Medicine
Chicago, Illinois, United States
Dana-Farber Cancer Institute Pediatric Oncology
Boston, Massachusetts, United States
Maximum Tolerated Dose
To define the maximum tolerated dose (MTD) of Xerecept® in pediatric patients; the MTD will be defined as the dose at which 1 or fewer of six patients experiences a dose limiting adverse event of any kind.
Time frame: 2 months
Dexamethasone Dosing
To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
Time frame: 1 Year
Incidence and severity of specified Steroid-Related Side Effects
To explore the clinical benefit associated with a reduction in steroid dosing over the course of the study by comparing the incidence and severity of pre-specified steroid-related side effects in all patients.
Time frame: 1 year
Number of patients with adverse events
Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients
Time frame: 1 Year
Change from baseline in clinical chemistry, hematology and urinalysis measures
Standard clinical chemistry, hematology and urinalysis measures will be analyzed at baseline and monthly during the study; changes from baseline will be measured over the course of the study
Time frame: 1 Year
PedsQL™ Quality of Life Inventory Scores
Child self report and parent proxy reports conducted at each study visit to assess changes in health-related quality of life over the course of the study
Time frame: 1 Year
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