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Tolerability Study of Xerecept® in Pediatric Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Celtic Pharma Development Services
ClinicalTrials.gov Identifier:
NCT01369121
First received: June 7, 2011
Last updated: July 31, 2012
Last verified: July 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Brain Edema
Brain Tumor
 Drug: XERECEPT (corticorelin acetate injection)
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Celtic Pharma Development Services:

Primary Outcome Measures:
  • Maximum Tolerated Dose [ Time Frame: 6 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.


Secondary Outcome Measures:
  • Dexamethasone Dosing [ Time Frame: 6 months ] [ 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: 6 months ] [ 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: 6 months ] [ 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: 6 months ] [ 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: 6 months ] [ 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


Enrollment: 15
Study Start Date: June 2010
Study Completion Date: May 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xerecept
All patients will receive hCRF (XERECEPT)
 Drug: XERECEPT (corticorelin acetate injection)
BID dosing, subcutaneous for 6 months
Other Name: hCRF

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have histologically confirmed primary, recurrent or metastatic cerebral tumors and require chronic steroid dosing to manage symptoms of peritumoral brain edema (No histological confirmation is required of optic pathway tumors or brain stem gliomas)
  2. Have been taking dexamethasone (or equivalent) at a dose of ≥1 mg/day for a minimum of 14 days and have had a stable dose for at least 7 days.
  3. Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)
  4. Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.
  5. Karnofsky/Lansky performance status ≥ 40
  6. Life expectancy of at least 6 months
  7. Female subjects must not be pregnant or breast-feeding and, if of childbearing potential, must use adequate methods of contraception during the study

10. Ability to provide written informed consent or, if unable to provide, have a legal guardian or representative provide written informed

Exclusion Criteria:

  1. Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
  2. Subject and/or parent/guardian is unwilling or unable to comply with this protocol
  3. Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
  4. Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01369121

Locations
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
Sponsors and Collaborators
Celtic Pharma Development Services
Investigators
Principal Investigator: Stewart Goldman, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

No publications provided

Responsible Party: Celtic Pharma Development Services
ClinicalTrials.gov Identifier: NCT01369121     History of Changes
Other Study ID Numbers: CPDS 1001
Study First Received: June 7, 2011
Last Updated: July 31, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Celtic Pharma Development Services:
peritumoral brain edema
edema
malignant brain tumor
 brain tumor
dexamethasone
Decadron

Additional relevant MeSH terms:
Brain Edema
Brain Neoplasms
Edema
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Central Nervous System Neoplasms
Nervous System Neoplasms
 Neoplasms by Site
Neoplasms
Signs and Symptoms
Corticotropin-Releasing Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 23, 2013