Clinical trial NCT01355679: Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma
Official title: A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Neuroblastoma
Lead sponsor: Milton S. Hershey Medical Center
Sponsor class: OTHER
First posted: 2011-05-18
Enrollment: 16
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing.

This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration.


Eligibility criteria: 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 recurrence/progression
* Patients must be age \> 12 months and ≤ 21 at initial diagnosis.
* 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); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be \> 75% are eligible to enroll.
* Current disease state must be one for which there is currently no known curative therapy
* Lansky or KarnofskyScore must be more than 50
* Patients without bone marrow metastases must have an ANC \> 750/μl and platelet count \> 50,000/μl
* 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 NCI-CTCAE V4.0
* 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 cannot 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 7 days prior to enrollment and 14 days prior to study treatment start date.
* Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow.
* Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently
* Patients with serious infection or a life-threatening illness (unrelated to tumor) that is \> Grade 2 (NCI CTCAE V4.0), or active, serious infections requiring parenteral antibiotic therapy.
* 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

Primary outcomes:
measure: Percentage of Participants That Are Able to Meet Feasibility Parameters.
timeFrame: 1 year
Secondary outcomes:
measure: Number of Participants With Adverse Events as a Measure of Safety
timeFrame: 1 year
measure: Overall Response Rate (ORR) of Participants Using RECIST Criteria
timeFrame: 1 year
measure: Activity of Treatments Chosen Based on Progression Free Survival (PFS)
timeFrame: 1 year
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Connecticut Children's Hospital
state: Connecticut
city: Orlando
country: United States
facility: Arnold Palmer Hospital for Children- MD Anderson
state: Florida
city: Bethesda
country: United States
facility: National Cancer Institute
state: Maryland
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospitals and Clinics
state: Missouri
city: Saint Louis
country: United States
facility: Cardinal Glennon Children's Medical Center
state: Missouri
city: Charlotte
country: United States
facility: Levine Children's Hospital
state: North Carolina
Study officials:
affiliation: Beat Childhood Cancer at Atrium Health
name: Giselle Sholler, MD
role: STUDY_CHAIR
References: