Clinical Trial: Molecular-Guided Therapy for Childhood Cancer

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Molecular-guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancers

Brief Summary: The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup. This technology includes a genomic report based on DNA exomes and RNA sequencing that will be used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.

Detailed Summary:
Sponsor: Giselle Sholler

Current Primary Outcome: Days to treatment will be used in order to determine feasibility of using tumor samples to assess genomic sequencing using predictive modeling to make real-time treatment decisions for children with relapsed/refractory cancers. [ Time Frame: 2 years ]

The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of Participants with Adverse Events as a Measure of Safety [ Time Frame: 2 years ]
    To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
  • Overall Response Rate (ORR) of Participants by the presence of radiologically assessable disease by cross-sectional CT or MRI imaging and/or by MIBG or PET scans. [ Time Frame: 2 years ]
    To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle.
  • Duration of response will be objectively documented [ Time Frame: 5 years ]
    Duration of response, defined as the period of time from when measurement criteria are met for complete response (CR) or partial response (PR), whichever is first recorded, until the first date that recurrent or progressive disease (PD) is objectively documented (taking as reference for PD the smallest measurements recorded since the treatment started)
  • Biology studies to include: genomic analysis of cells pre- and post- treatment, correlation of in vitro response to in vivo response, sub analysis examination of disease types, and biomarker development. [ Time Frame: 2 years ]
    To explore the relationship between tumor phenotype and response by permitting use of tumor tissue in a correlative biologic study
  • Progression Free Survival (PFS) interval will be measured by days and compared to the PFS of previous chemotherapy regimens since relapse for each patient. [ Time Frame: 2 years ]
    Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements


Original Secondary Outcome: Same as current

Information By: Spectrum Health Hospitals

Dates:
Date Received: June 11, 2014
Date Started: June 2014
Date Completion: June 2021
Last Updated: April 25, 2017
Last Verified: April 2017