Tolerability Study of Xerecept® in Pediatric Patients

Sponsor

Celtic Pharma Development Services (Industry)

Overall Status

Terminated

CT.gov ID

NCT01369121

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

7.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Brain Edema Brain Tumor	Drug: XERECEPT	Phase 1/Phase 2

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

Study Design

Study Type:

Interventional

Actual Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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

Study Start Date :

Jun 1, 2010

Actual Primary Completion Date :

Oct 1, 2012

Actual Study Completion Date :

Mar 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Xerecept All patients will receive hCRF (XERECEPT)	Drug: XERECEPT BID dosing, subcutaneous for 1 year Other Names: hCRF Corticorelin acetate injection

Arm

Intervention/Treatment

Experimental: Xerecept

All patients will receive hCRF (XERECEPT)

Drug: XERECEPT

BID dosing, subcutaneous for 1 year

Other Names:

hCRF

Corticorelin acetate injection

Outcome Measures

Primary Outcome Measures

Maximum Tolerated Dose [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.

Secondary Outcome Measures

Dexamethasone Dosing [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 [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 [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 [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 [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

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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)
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.
Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)
Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.
Karnofsky/Lansky performance status ≥ 40
Life expectancy of at least 6 months
Female subjects must not be pregnant or breast-feeding and, if of childbearing potential, must use adequate methods of contraception during the study
Ability to provide written informed consent or, if unable to provide, have a legal guardian or representative provide written informed

Exclusion Criteria:

Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
Subject and/or parent/guardian is unwilling or unable to comply with this protocol
Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Memorial Hospital, Northwestern University Feinberg School of Medicine	Chicago	Illinois	United States	60614
2	Dana-Farber Cancer Institute Pediatric Oncology	Boston	Massachusetts	United States	02115