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
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
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
Stewart Goldman, MD
Ann & Robert H Lurie Children's Hospital of Chicago
United States: Food and Drug Administration
|Children's Memorial Hospital, Northwestern University Feinberg School of Medicine||Chicago, Illinois 60614|
|Dana-Farber Cancer Institute Pediatric Oncology||Boston, Massachusetts 02115|