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Tolerability Study of Xerecept® in Pediatric Patients

Primary Purpose

Brain Edema, Brain Tumor

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

XERECEPT

About this trial

This is an interventional treatment trial for Brain Edema focused on measuring peritumoral brain edema, edema, malignant brain tumor, brain tumor, dexamethasone, Decadron

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept 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

10. 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).

Sites / Locations

Children's Memorial Hospital, Northwestern University Feinberg School of Medicine
Dana-Farber Cancer Institute Pediatric Oncology

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Xerecept

Arm Description

All patients will receive hCRF (XERECEPT)

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Dexamethasone Dosing

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

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

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

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

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

Full Information

NCT ID

NCT01369121

First Posted

June 7, 2011

Last Updated

July 29, 2013

Sponsor

Celtic Pharma Development Services

1. Study Identification

Unique Protocol Identification Number

NCT01369121

Brief Title

Tolerability Study of Xerecept® in Pediatric Patients

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 Type

Interventional

2. Study Status

Record Verification Date

July 2013

Overall Recruitment Status

Terminated

Why Stopped

Single subject remained on study - transferred to compassionate use

Study Start Date

June 2010 (undefined)

Primary Completion Date

October 2012 (Actual)

Study Completion Date

March 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Celtic Pharma Development Services

4. Oversight

Data Monitoring Committee

5. 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Brain Edema, Brain Tumor

Keywords

peritumoral brain edema, edema, malignant brain tumor, brain tumor, dexamethasone, Decadron

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Xerecept

Arm Type

Experimental

Arm Description

All patients will receive hCRF (XERECEPT)

Intervention Type

Drug

Intervention Name(s)

XERECEPT

Other Intervention Name(s)

hCRF, Corticorelin acetate injection

Intervention Description

BID dosing, subcutaneous for 1 year

Primary Outcome Measure Information:

Title

Maximum Tolerated Dose

Description

Time Frame

2 months

Secondary Outcome Measure Information:

Title

Dexamethasone Dosing

Description

To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing

Time Frame

1 Year

Title

Incidence and severity of specified Steroid-Related Side Effects

Description

Time Frame

1 year

Title

Number of patients with adverse events

Description

Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients

Time Frame

1 Year

Title

Change from baseline in clinical chemistry, hematology and urinalysis measures

Description

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

Time Frame

1 Year

Title

PedsQL™ Quality of Life Inventory Scores

Description

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

Time Frame

1 Year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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 10. 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).

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Stewart Goldman, MD

Organizational Affiliation

Ann & Robert H Lurie Children's Hospital of Chicago

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Memorial Hospital, Northwestern University Feinberg School of Medicine

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60614

Country

United States

Facility Name

Dana-Farber Cancer Institute Pediatric Oncology

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Tolerability Study of Xerecept® in Pediatric Patients

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