Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission



Status:Active, not recruiting
Conditions:Brain Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:Any - 21
Updated:7/12/2018
Start Date:March 2015
End Date:March 2021

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A Phase II Preventative Trial of DFMO (Eflornithine HCl) as a Single Agent in Patients With High Risk Neuroblastoma in Remission

The purpose of this research study is to evaluate a new investigational drug to prevent
reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The
objectives of this study will be to monitor for safety and 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 may
continue on treatment up to 27 cycles 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), meta-iodobenzylguanidine (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.

The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day
while on study. There will be 27 cycles. Each cycle will be 28 days in length.


Inclusion Criteria:

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

- Diagnosis: histologic verification at either the time of original diagnosis or a
previous relapse of high risk neuroblastoma.

- Disease Status: Neuroblastoma that is in remission

- First dose of study medication must be greater than 30 days from completion of
cytotoxic and antibody therapy and less than 120 days from previous therapy

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

- Both male and female post-pubertal study subjects need to agree to use one of the more
effective birth control methods during treatment and for six months after treatment is
stopped. These methods include total abstinence (no sex), oral contraceptives ("the
pill"), an intrauterine device (IUD), levonorgestrel implants (Norplant), or
medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be
used, contraceptive foam with a condom is recommended.

- Absolute Neutrophil Count (ANC) > 500/μl and platelet count >50,000/μl

- Organ Function Requirements: Subjects must have adequate liver function as defined by:

- Aspartate Aminotransferase (AST) and Alanine transaminase (ALT) <10x upper limit
of normal

- Serum bilirubin must be ≤ 2.0 mg/dl

- Serum creatinine based on age/gender

- 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:

- Lansky score < 60%

- Body Surface Area (BSA) (m2) of <0.25

- 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.
We found this trial at
21
sites
1319 Punahou St
Honolulu, Hawaii 96826
(808) 983-6000
Principal Investigator: Randal Wada, MD
Phone: 808-535-7169
Kapiolani Medical Center for Women and Children Hawai‘i Pacific Health is an integrated health care...
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800 Washington St
Boston, Massachusetts 02111
(617) 636-5000
Principal Investigator: Michael Kelly, MD
Phone: 617-636-8885
Tufts Medical Center Tufts Medical Center is an internationally-respected academic medical center – a teaching...
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Boston, MA
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Keri Streby, MD
Phone: 614-722-6039
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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Austin, Texas 78723
Principal Investigator: Virginia Harrod, MD
Phone: 512-628-1902
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Bronx, New York 10467
Principal Investigator: Daniel Weiser, MD
Phone: 718-741-2356
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Principal Investigator: Jaqueline Kraveka, MD
Phone: 843-792-2957
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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Charlotte, North Carolina 28204
Principal Investigator: Javier Oesterheld, MD
Phone: 980-442-2312
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Dallas, Texas 75235
Principal Investigator: Ted Laetsch, MD
Phone: 214-456-0004
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Grand Rapids, Michigan 49503
Principal Investigator: Deanna Mitchell, MD
Phone: 616-267-1162
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Hartford, Connecticut 06106
Principal Investigator: Nehal Parikh, MD
Phone: 860-545-9337
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Hershey, Pennsylvania 17033
Principal Investigator: Valerie Brown, MD
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Houston, Texas 77030
Principal Investigator: Peter Zage, MD, PhD
Phone: 832-824-4615
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Kansas City, Missouri 64108
Principal Investigator: Kathleen Neville, MD
Phone: 816-855-1977
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Minneapolis, Minnesota 55404
Principal Investigator: Jawhar Rawwas, MD
Phone: 612-813-5913
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Nashville, Tennessee 37232
Principal Investigator: Devang Pastakia, MD
Phone: 615-936-1522
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Orlando, Florida 32806
Principal Investigator: Don Eslin, MD
Phone: 321-841-8588
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Francis Eshun, MD
Phone: 602-546-0211
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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Saint Louis, Missouri 63104
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Saint Petersburg, Florida 33701
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Salt Lake City, Utah 84143
Principal Investigator: Mark Fluchel, MD
Phone: 801-662-4710
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San Diego, California 92123
Principal Investigator: William Roberts, MD
Phone: 858-966-8155
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