Summary

for people ages 12 months to 21 years (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

Summary

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.

Official Title

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.

Keywords

Neuroblastoma Sorafenib Lapatinib Vorinostat Bortezomib Dasatinib Eflornithine Crizotinib Niacinamide

Eligibility

You can join if…

Open to people ages 12 months to 21 years

  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.

  1. Subjects must be age ≤ 21 years at initial diagnosis
  2. 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.
  3. Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
  4. Adequate liver function must be demonstrated, defined as:
  5. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
  6. ALT (SGPT) < 10 x upper limit of normal (ULN) for age
  7. Subjects must have adequate renal function defined as a serum creatinine based on age/gender
  8. Adequate Cardiac Function defined as: Shortening fraction of ≥ 27% by echocardiogram,or Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.
  9. 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.
  10. A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
  11. . 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.
  12. . Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines.

You CAN'T join if...

  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

Locations

  • UCSF Benioff Children's Hospital Oakland- not yet accepting patients
    Oakland, California, United States
  • Rady Children's Hospital accepting new patients
    San Diego, California, 92123, United States
  • Phoenix Children's Hospital not yet accepting patients
    Phoenix, Arizona, 85016, United States
  • Randall Children's Hospital accepting new patients
    Portland, Oregon, United States
  • Children's Medical Center accepting new patients
    Dallas, Texas, 75235, United States
  • Dell Children's Blood and Cancer Center accepting new patients
    Austin, Texas, 78723, United States
  • Children's Mercy Hospitals and Clinics not yet accepting patients
    Kansas City, Missouri, 64108, United States
  • Children's Hospital and Clinics on Minnesota accepting new patients
    Minneapolis, Minnesota, 55404, United States
  • Arkansas Children's Hospital accepting new patients
    Little Rock, Arkansas, 72202, United States
  • Cardinal Glennon Children's Medical Center accepting new patients
    Saint Louis, Missouri, 63104, United States
  • Advocate Children's Medical Group accepting new patients
    Chicago, Illinois, United States
  • Helen DeVos Children's Hospital accepting new patients
    Grand Rapids, Michigan, 49503, United States
  • University of Louisville not yet accepting patients
    Louisville, Kentucky, United States
  • University of Alabama, Children's of Alabama accepting new patients
    Birmingham, Alabama, United States
  • Augusta University Health not yet accepting patients
    Augusta, Georgia, United States
  • Levine Children's Hospital accepting new patients
    Charlotte, North Carolina, 28204, United States
  • Medical University of South Carolina accepting new patients
    Charleston, South Carolina, 29425, United States
  • Arnold Palmer Hospital for Children not yet accepting patients
    Orlando, Florida, 32806, United States
  • Penn State Milton S. Hershey Medical Center and Children's Hospital accepting new patients
    Hershey, Pennsylvania, 17033, United States
  • Children's Hospital of The King's Daughters not yet accepting patients
    Norfolk, Virginia, United States
  • Kapiolani Medical Center for Women and Children accepting new patients
    Honolulu, Hawaii, 96813, United States
  • Hackensack University Medical Center accepting new patients
    Hackensack, New Jersey, United States
  • Connecticut Children's Hospital accepting new patients
    Hartford, Connecticut, 06106, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Giselle Sholler
Links
Beat Childhood Cancer Main Website
ID
NCT02559778
Phase
Phase 2
Lead Scientist
Anurag Agrawal
Study Type
Interventional
Last Updated
February 13, 2018