A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Neuroblastoma
- Sponsor
- Giselle Sholler
- Enrollment
- 16
- Locations
- 7
- Primary Endpoint
- Percentage of Participants That Are Able to Meet Feasibility Parameters.
- Status
- Completed
- Last Updated
- last year
Overview
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.
Investigators
Giselle Sholler
Study Chair
Milton S. Hershey Medical Center
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
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
Outcomes
Primary Outcomes
Percentage of Participants That Are Able to Meet Feasibility Parameters.
Time Frame: 1 year
Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
Secondary Outcomes
- Overall Response Rate (ORR) of Participants Using RECIST Criteria(1 year)
- Activity of Treatments Chosen Based on Progression Free Survival (PFS)(1 year)
- Number of Participants With Adverse Events as a Measure of Safety(1 year)