Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide

Conditions:Brain Cancer
Therapuetic Areas:Oncology
Age Range:Any
Start Date:February 2010
End Date:May 2015

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

The purpose of this research study is to evaluate a new investigational drug to treat
neuroblastoma. This study drug is called DFMO. The objectives of this study will be to
monitor for safety and to find a maximum tolerated dose in this population. A secondary
objective will be to look at efficacy of DFMO.

The safety of the proposed dosing regimen in this trial will be tested by an on-going
risk/benefit assessment during the study. A patient benefiting from treatment, not
progressing on therapy, and in the absence of any safety issues associated with DFMO and/or
etoposide may continue on treatment with the expectation that there will be an overall
clinical benefit.

The procedures involved in this study include Medical history, Physical exam, Vital signs
(blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the
tumor(s), MIBG scans, and Bone marrow aspirations. All of these tests and procedures are
considered standard of care for this population. Drug administration is also part of this
protocol, including an investigational new drug called DFMO, and later combined with an
already approved drug, etoposide.

The proposed dosing regimen is an oral dose of DFMO two times a day for each day while on
study. There will be 5 cycles. Each cycle will be 21 days in length. The first cycle will be
DFMO alone. In the second cycle etoposide will be added in and will be given orally once a
day for the first 14 days of each cycle (cycles 2-5).

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.
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