Clinical Trial: Tolerability Study of Xerecept® in Pediatric Patients

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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&

Brief Summary: 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.

Detailed Summary:

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


Sponsor: Celtic Pharma Development Services

Current Primary Outcome: Maximum Tolerated Dose [ Time Frame: 2 months ]

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.


Original Primary Outcome: Maximum Tolerated Dose [ Time Frame: 6 months ]

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.


Current Secondary Outcome:

  • Dexamethasone Dosing [ Time Frame: 1 Year ]
    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 ]
    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 ]
    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 ]
    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 ]
    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


Original Secondary Outcome:

  • Dexamethasone Dosing [ Time Frame: 6 months ]
    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: 6 months ]
    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: 6 months ]
    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: 6 months ]
    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: 6 months ]
    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


Information By: Celtic Pharma Development Services

Dates:
Date Received: June 7, 2011
Date Started: June 2010
Date Completion:
Last Updated: July 29, 2013
Last Verified: July 2013