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

Information source: Spectrum Health Hospitals
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Neuroblastoma

Intervention: DFMO (Drug); Etoposide (Drug)

Phase: Phase 1

Status: Active, not recruiting

Sponsored by: Giselle Sholler

Official(s) and/or principal investigator(s):
Giselle Sholler, MD, Study Chair, Affiliation: The Spectrum Health Group

Summary

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).

Clinical Details

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

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

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

Secondary outcome:

To evaluate the activity of DFMO as a single agent and in combination with etoposide in these tumor types based on: Progression free survival (PFS)

Determine the Overall Response Rate (ORR) of Participants using RECIST criteria Determine the Overall Response Rate (ORR) of Participants using RECIST criteria

To evaluate the pharmacokinetics (PK) of DFMO as single agent

Biology studies to include: effect on polyamine depletion, ODC activity, genomic analysis of cells pre- and post- treatment, correlation of in vitro response to in vivo response, flow cytometry of tumor burden in bone marrow and biomarker development

Eligibility

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

Criteria:

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

therapy.

- 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

>50,000/μl

- 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 guidelines 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.

Locations and Contacts

Children's Hospital of Orange County, Orange, California 92868, United States

Connecticut Children's Hospital, Hartford, Connecticut 06106, United States

Arnold Palmer Hospital for Children- MD Anderson, Orlando, Florida 32806, United States

Helen DeVos Children's Hospital, Grand Rapids, Michigan 49503, United States

Children's Mercy Hospitals and Clinics, Kansas City, Missouri 64108, United States

Levine Children's Hospital, Charlotte, North Carolina 28204, United States

UVM/FAHC, Burlington, Vermont 05401, United States

Additional Information

Starting date: February 2010
Last updated: November 17, 2014

Page last updated: August 23, 2015

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