Pediatric Precision Laboratory Advanced Neuroblastoma Therapy



Status:Recruiting
Conditions:Brain Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:Any - 21
Updated:2/17/2018
Start Date:September 2015
End Date:September 2026
Contact:Genevieve Bergendahl, MSN
Email:genevieve.bergendahl@helendevoschildrens.org
Phone:616-267-0334

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A Study Using Molecular Guided Therapy With Induction Chemotherapy Followed by a Randomized Controlled Trial of Standard Immunotherapy With or Without DFMO Followed by DFMO Maintenance for Subjects With Newly Diagnosed High-Risk Neuroblastoma.

A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of
using molecularly guided therapy in combination with standard therapy followed by a
Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO
maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.


Inclusion Criteria:

1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma
(nodular or intermixed) verified by histology or demonstration of clumps of tumor
cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with
the following disease stages at diagnosis are eligible, if they meet the other
specified criteria:

a) Subjects with newly diagnosed neuroblastoma with INSS Stage 4 are eligible with the
following: i. Age > 18 months (> 547 days) regardless of biologic features or ii. Age
12-18 months (365-547 days) with any of the following 3 unfavorable biologic features
(MYCN amplification, unfavorable pathology and/or DNA index = 1) or iii. MYCN
amplification (> 4-fold increase in MYCN signals as compared to reference signals),
regardless of age or additional biologic features.

b) Subjects with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the
following: i. MYCN amplification (> 4-fold increase in MYCN signals as compared to
reference signals), regardless of age or additional biologic features or ii. Age > 18
months (> 547 days) with unfavorable pathology, regardless of MYCN status.

c) Subjects with newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN
amplification (> 4-fold increase in MYCN signals as compared to reference signals),
regardless of age or additional biologic features.

2. Subjects must be age ≤ 21 years at initial diagnosis

3. Subjects must not have had prior systemic therapy except for localized emergency
radiation to sites of life-threatening or function-threatening disease and/or no more
than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as
per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification
status and histology.

4. Specimens will be obtained only in a non-significant risk manner and not solely for
the purpose of investigational testing.

5. Adequate liver function must be demonstrated, defined as:

1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND

2. ALT (SGPT) < 10 x upper limit of normal (ULN) for age

6. Subjects must have adequate renal function defined as a serum creatinine based on
age/gender

7. Adequate Cardiac Function defined as: Shortening fraction of ≥ 27% by echocardiogram,
or Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.

8. Ability to tolerate PBSC collection: No known contraindication to PBSC collection.
Examples of contraindications would include a weight or size less than that determined
to be feasible at the collecting institution, or a physical condition that would limit
the ability of the child to undergo apheresis catheter placement (if necessary) and/or
the apheresis procedure.

9. A negative serum pregnancy test is required for female participants of child bearing
potential (≥13 years of age or after onset of menses)

10. 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), levonorgestrol implants (Norplant), or
medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be
used, contraceptive foam with a condom is recommended.

11. 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:

1. Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with
favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index
> 1) are not eligible.

2. Lactating females are not eligible unless they have agreed not to breastfeed their
infants.

3. Subjects receiving any investigational drug concurrently.

4. Subjects with any other medical condition, including but not limited to malabsorption
syndromes, mental illness or substance abuse, deemed by the Investigator to be likely
to interfere with the interpretation of the results or which would interfere with a
subject's ability to sign or the legal guardian's ability to sign the informed
consent, and subject's ability to cooperate and participate in the study
We found this trial at
23
sites
Orlando, Florida 32806
Principal Investigator: Don Eslin, MD
Phone: 321-841-8588
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601 Children's Lane
Norfolk, Virginia 23507
(757) 668-7000
Principal Investigator: Eric Lowe, MD
Children's Hospital of The King's Daughters Children
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1120 15th Street
Augusta, Georgia 30912
Principal Investigator: Coleen McDonough, MD
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Austin, Texas 78723
Principal Investigator: Virginia Harrod, MD
Phone: 512-628-1902
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1208 3rd Avenue South
Birmingham, Alabama 35233
Principal Investigator: Elizabeth Alva, MD
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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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836 West Wellington Avenue
Chicago, Illinois 60657
Principal Investigator: Jason Canner, MD
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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: 616-267-0334
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30 Prospect Ave
Hackensack, New Jersey 07601
(201) 996-2000
Principal Investigator: Derek Hanson, MD
Hackensack University Medical Center Hackensack University Medical Center, part of the Hackensack University Health Network,...
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Hartford, Connecticut 06106
Principal Investigator: Michael Isakoff, MD
Phone: 860-545-9337
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Hershey, Pennsylvania 17033
Principal Investigator: Valerie Brown, MD
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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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Kansas City, Missouri 64108
Principal Investigator: Keith August, MD
Phone: 816-302-6893
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1 Children's Way
Little Rock, Arkansas 72202
(501) 364-1100
Principal Investigator: Kathleen Neville, MD
Phone: 501-364-2760
Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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500 S Preston St
Louisville, Kentucky
(502) 852-5555
Principal Investigator: Ashok Raj, MD
University of Louisville The University of Louisville is a state supported research university located in...
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Minneapolis, Minnesota 55404
Principal Investigator: Jawhar Rawwas, MD
Phone: 612-813-5913
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Oakland, California 94609
Principal Investigator: Anurag Agrawal, MD
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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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Portland, Oregon 97227
Principal Investigator: Jason Glover, MD
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Saint Louis, Missouri 63104
Principal Investigator: William Ferguson, MD
Phone: 314-268-4000
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San Diego, California 92123
Principal Investigator: William Roberts, MD
Phone: 858-966-8155
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