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A Phase I Trial for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide

Phase 1
Open (Enrolling)

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Trial Information

A Phase I Trial for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide

Inclusion Criteria:

- Age: 0-21 years at the time of diagnosis.

- Diagnosis: Histologic verification at either the time of original diagnosis or
relapse of neuroblastoma.

- Disease Status: Refractory or relapsed neuroblastoma

- Measurable disease, including at least one of the following:

Measurable tumor >10mm by CT or MRI A positive MIBG and abnormal urinary catecholamine
levels Positive bone marrow biopsy/aspirate.

- Current disease state must be one for which there is currently no known curative

- A negative urine pregnancy test is required for female subjects of child bearing
potential (onset of menses or ≥13 years of age).

- Patients without bone marrow metastases must have an ANC > 500/μl and platelet count

- Organ Function Requirements Subjects must have adequate liver function as defined by
AST or ALT <10x normal Serum bilirubin must be ≤ 2.0 mg/dl Serum creatinine must be
≥ 1.5 mg/dl

- Informed Consent: All subjects and/or legal guardians must sign informed written
consent. Assent, when appropriate, will be obtained according to institutional

Exclusion Criteria:

- Life expectancy <2 months or Lansky score <30%

- Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation.

- Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
not eligible. Subjects must have fully recovered from the effects of prior
chemotherapy (hematological and bone marrow suppression effects)

- Infection: Subjects who have an uncontrolled infection are not eligible until the
infection is judged to be well controlled in the opinion of the investigator.

- Subjects who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study, or in whom compliance is likely to be
suboptimal, should be excluded.

Type of Study:


Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Outcome Description:

To determine the safety, tolerability and maximum tolerated dose (MTD) of DFMO as a single agent and in combination with etoposide in pediatric and young adult patients with refractory or recurrent neuroblastoma

Outcome Time Frame:

length of study plus 30 days

Safety Issue:


Principal Investigator

Giselle Sholler, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Van Andel Research Institute


United States: Food and Drug Administration

Study ID:




Start Date:

February 2010

Completion Date:

June 2016

Related Keywords:

  • Neuroblastoma
  • Refractory Neuroblastoma
  • Relapsed neuroblastoma
  • Neuroblastoma



Children's Hospital of Orange County Orange, California  92668
Children's Mercy Hospitals and Clinics Kansas City, Missouri  64108
Connecticut Children's Hospital Hartford, Connecticut  06106
Arnold Palmer Hospital for Children- MD Anderson Orlando, Florida  32806
Helen DeVos Children's Hospital Grand Rapids, Michigan  49503
Levine Children's Hospital Charlotte, North Carolina  28204
UVM/FAHC Burlington, Vermont  05401