This study has been terminated.

(Single subject remained on study - transferred to compassionate use)

Sponsor:
Celtic Pharma Development Services
Information provided by (Responsible Party):
Celtic Pharma Development Services
ClinicalTrials.gov Identifier:
NCT01369121
First received: June 7, 2011
Last updated: July 29, 2013
Last verified: July 2013
History of Changes
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Tolerability Study of Xerecept in Pediatric Patients
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 Phase 1
Phase 2
Study Type: Interventional
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
Resource links provided by NLM:
MedlinePlus related topics: Brain Cancer Cancer Childhood Brain Tumors Edema
Drug Information available for: Corticotropin
U.S. FDA Resources
Further study details as provided by Celtic Pharma Development Services:
Primary Outcome Measures:
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.
Secondary Outcome Measures:
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
Enrollment: 15
Study Start Date: June 2010
Study Completion Date: March 2013
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Xerecept
All patients will receive hCRF (XERECEPT)
Drug: XERECEPT
BID dosing, subcutaneous for 1 year
Other Names:
hCRF
Corticorelin acetate injection
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a
study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general
information, see Learn About Clinical Studies.
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 29, 2013
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 June 05, 2014