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Trial Testing the Feasibility of Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma

12 Months
21 Years
Not Enrolling
Refractory Neuroblastoma, Relapsed Neuroblastoma

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Trial Information

Trial Testing the Feasibility of Using Molecular-Guided Therapy in Patients With Refractory or Recurrent Neuroblastoma

Inclusion Criteria:

- Patients must have histologically proven neuroblastoma and confirmation of refractory
or recurrent disease with histologic confirmation at diagnosis or at the time of

- Patients must be age >12 months and diagnosed before the age of 21

- Life expectancy must be more than 3 months

- If measurable disease, this must be demonstrated by residual abnormal tissue at a
primary or metastatic site measuring more than 1 cm in any dimension by standardized
imaging (CT or MRI). For patients with only skeletal sites of disease, there must be
at least one persisting focus with increased activity on a pre-treatment
meta-iodobenzylguanidine (MIBG) scan.

- Current disease state must be one for which there is currently no known curative

- Lansky Play Score must be more than 30

- Patients without bone marrow metastases must have an ANC > 750/μl and platelet count

- Adequate liver function must be demonstrated, defined as:

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

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

- No other significant organ toxicity defined as >Grade 2 by National Cancer Institute
Common Toxicity Criteria for Adverse Events version 3 (NCI-CTCAE V3.0

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

- 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 can not be used, contraceptive foam with a condom is recommended.

- Informed Consent: All patients and/or legal guardians must sign informed written
consent. Assent, when appropriate, will be obtained according to institutional
guidelines. Voluntary consent for optional biology studies will be included.

Exclusion Criteria:

- Patients who have received any chemotherapy within the last 21 days.

- Patients receiving anti-tumor therapy for their disease or any investigational drug

- Patients with serious infection or a life-threatening illness (unrelated to tumor)
that is > Grade 2 (NCI CTCAE V3.0), or active, serious infections requiring
parenteral antibiotic therapy within 2 weeks prior to screening

- Patients with any other medical condition, including 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 patient's ability
to sign or the legal guardian's ability to sign the informed consent, and patient's
ability to cooperate and participate in the study

Type of Study:


Study Design:

Observational Model: Case-Only, Time Perspective: Prospective

Outcome Measure:

Feasibility of using protocol process to make real time treatment decisions

Outcome Description:

To pilot 5 patients to evaluate the feasibility of using predictive modeling based on genome-wide mRNA expression profiles of bone marrow derived neuroblastoma cells or tumor biopsies to make real-time treatment decisions.

Outcome Time Frame:

4 months

Safety Issue:


Principal Investigator

Giselle Sholler, MD

Investigator Role:

Study Chair

Investigator Affiliation:

University of Vermont


United States: Institutional Review Board

Study ID:




Start Date:

April 2010

Completion Date:

July 2010

Related Keywords:

  • Refractory Neuroblastoma
  • Relapsed Neuroblastoma
  • Refractory Neuroblastoma
  • Relapsed Neuroblastoma
  • Neuroblastoma



UVM/FAHCBurlington, Vermont  05401