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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


Phase 1/Phase 2
1 Year
18 Years
Not Enrolling
Both
Brain Edema, Brain Tumor

Thank you

Trial Information

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


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


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).

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Maximum Tolerated Dose

Outcome Description:

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.

Outcome Time Frame:

6 months

Safety Issue:

Yes

Principal Investigator

Stewart Goldman, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Ann & Robert H Lurie Children's Hospital of Chicago

Authority:

United States: Food and Drug Administration

Study ID:

CPDS 1001

NCT ID:

NCT01369121

Start Date:

June 2010

Completion Date:

May 2012

Related Keywords:

  • Brain Edema
  • Brain Tumor
  • peritumoral brain edema
  • edema
  • malignant brain tumor
  • brain tumor
  • dexamethasone
  • Decadron
  • Brain Edema
  • Brain Neoplasms
  • Edema

Name

Location

Children's Memorial Hospital, Northwestern University Feinberg School of Medicine Chicago, Illinois  60614
Dana-Farber Cancer Institute Pediatric Oncology Boston, Massachusetts  02115