Tolerability Study of Xerecept® in Pediatric Patients

Brain Tumor Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01369121
• Other study ID #: CPDS 1001
• Status: Terminated
• Phase: Phase 1/Phase 2
• First received: June 7, 2011
• Last updated: July 29, 2013
• Start date: June 2010
• Est. completion date: March 2013

Study information

• Verified date: July 2013
• Source: Celtic Pharma Development Services
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: March 2013
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 18 Years

Eligibility

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).

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Brain Edema
• Brain Neoplasms
• Brain Tumor

Intervention

Drug:
• XERECEPT
BID dosing, subcutaneous for 1 year

Locations

Country	Name	City	State
United States	Dana-Farber Cancer Institute Pediatric Oncology	Boston	Massachusetts
United States	Children's Memorial Hospital, Northwestern University Feinberg School of Medicine	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Celtic Pharma Development Services

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	2 months	Yes
Secondary	Dexamethasone Dosing	To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing	1 Year	Yes
Secondary	Incidence and severity of specified Steroid-Related Side Effects	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.	1 year	No
Secondary	Number of patients with adverse events	Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients	1 Year	Yes
Secondary	Change from baseline in clinical chemistry, hematology and urinalysis measures	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	1 Year	Yes
Secondary	PedsQL™ Quality of Life Inventory Scores	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	1 Year	No