Tolerability Study of Xerecept® in Pediatric Patients
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.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/II Open Label Individual Dose Titration Trial of the Human Corticotropin- Releasing Factor (hCRF), Corticorelin Acetate Injection (Xerecept®), to Determine the Tolerability of Xerecept® in a Pediatric Population|
- Maximum Tolerated Dose [ Time Frame: 2 months ] [ Designated as safety issue: Yes ]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.
- Dexamethasone Dosing [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
- Incidence and severity of specified Steroid-Related Side Effects [ Time Frame: 1 year ] [ Designated as safety issue: No ]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.
- Number of patients with adverse events [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients
- Change from baseline in clinical chemistry, hematology and urinalysis measures [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]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
- PedsQL™ Quality of Life Inventory Scores [ Time Frame: 1 Year ] [ Designated as safety issue: No ]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
|Study Start Date:||June 2010|
|Study Completion Date:||March 2013|
|Primary Completion Date:||October 2012 (Final data collection date for primary outcome measure)|
All patients will receive hCRF (XERECEPT)
BID dosing, subcutaneous for 1 year
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
|United States, Illinois|
|Children's Memorial Hospital, Northwestern University Feinberg School of Medicine|
|Chicago, Illinois, United States, 60614|
|United States, Massachusetts|
|Dana-Farber Cancer Institute Pediatric Oncology|
|Boston, Massachusetts, United States, 02115|
|Principal Investigator:||Stewart Goldman, MD||Ann & Robert H Lurie Children's Hospital of Chicago|