Trial record 1 of 1 for:    NMTRC008
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Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Spectrum Health Hospitals
Sponsor:
Collaborators:
Van Andel Research Institute
Dell, Inc.
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT01802567
First received: February 26, 2013
Last updated: February 5, 2014
Last verified: February 2014
  Purpose

The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.


Condition Intervention
Neuroblastoma
Medulloblastoma
Brain Tumors
Rare Tumors
Device: Guided Therapy- Pediatric Gene Analysis Platform

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancer

Resource links provided by NLM:


Further study details as provided by Spectrum Health Hospitals:

Primary Outcome Measures:
  • Days to treatment will be used in order to determine feasibility of using tumor samples to assess genomic mRNA expression arrays and DNA Mutation Panels using predictive modeling [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."


Secondary Outcome Measures:
  • Number of Participants with Adverse Events as a Measure of Safety [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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: 2 years ] [ Designated as safety issue: No ]
    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, flow cytometry of tumor burden in bone marrow and biomarker development [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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


Estimated Enrollment: 48
Study Start Date: February 2013
Estimated Study Completion Date: February 2016
Estimated Primary Completion Date: February 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Guided Therapy- Pediatric Gene Analysis Platform
A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Device: Guided Therapy- Pediatric Gene Analysis Platform
A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
Other Name: Molecular Guided Therapy

  Eligibility

Ages Eligible for Study:   13 Months and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects must have histologically proven neuroblastoma, brain tumor, or rare tumor and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
  • Subjects must be age >12 months at enrollment.
  • Subjects must be age ≤ 21 years at initial diagnosis.
  • Subjects must have measurable disease as 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% tumor are eligible to enroll.
  • Current disease state must be one for which there is currently no known curative therapy
  • Lansky or Karnofsky Score must be more than 50
  • Subjects without bone marrow metastases must have an ANC > 750/μl
  • Adequate liver function must be demonstrated, defined as:

    1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
    2. SGPT (ALT) < 10 x upper limit of normal (ULN) for age
  • 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 subjects 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:

  • Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
  • Subjects who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).
  • Subjects receiving anti-tumor therapy for their disease or any investigational drug concurrently
  • Subjects 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.
  • Subjects 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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01802567

Contacts
Contact: Genevieve Bergendahl, RN 616-267-0335 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa VanderWerff (616) 267-0327 alyssa.vanderwerff@helendevoschildrens.org

Locations
United States, Arizona
Phoenix Children's Hospital Not yet recruiting
Phoenix, Arizona, United States, 85016
Contact: Samuel Chimienti    602-546-0211    schimienti@phoenixchildrens.com   
Principal Investigator: Francis Eshun, MD         
United States, California
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: Mehrzad Milburn    858-966-8155    mmilburn@rchsd.org   
Principal Investigator: William Roberts, MD         
United States, Connecticut
Connecticut Children's Hospital Recruiting
Hartford, Connecticut, United States, 06106
Contact: Jennifer Hylton    860-545-9337    Jhylton02@connecticutchildrens.org   
Principal Investigator: Nehal Parikh, MD         
United States, Florida
Arnold Palmer Hospital for Children- MD Anderson Recruiting
Orlando, Florida, United States, 32806
Contact: Michelle Pope, RN    321-841-8588      
Principal Investigator: Don Eslin, MD         
United States, Hawaii
Kapiolani Medical Center for Women and Children Recruiting
Honolulu, Hawaii, United States, 96813
Contact: Andrea Siu, MPH    808-535-7169    andrea.siu@kapiolani.org   
Principal Investigator: Randal Wada, MD         
United States, Michigan
Helen DeVos Children's Hospital Recruiting
Grand Rapids, Michigan, United States, 49503
Contact: Shannon Mackeigan    616-267-1162    shannon.mackeigan@helendevoschildrens.org   
Principal Investigator: Giselle Sholler, MD         
Principal Investigator: Deanna Mitchell, MD         
United States, Missouri
Children's Mercy Hospitals and Clinics Recruiting
Kansas City, Missouri, United States, 64108
Contact: Sara Soliman, RN    816-855-1977    sjsoliman@cmh.edu   
Principal Investigator: Kathleen Neville, MD         
Cardinal Glennon Children's Medical Center Recruiting
St. Louis, Missouri, United States, 63104
Contact: Katherine Maxwell, RN    314-268-4000      
Principal Investigator: William Ferguson, MD         
United States, North Carolina
Levine Children's Hospital Recruiting
Charlotte, North Carolina, United States, 28204
Contact: Nicole Turner, RN    980-442-2355    felisha.turner@carolinashealthcare.org   
Principal Investigator: Javier Oesterheld, MD         
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Kate McCormack, RN    843-792-3379    mccormk@musc.edu   
Principal Investigator: Jaqueline Kraveka, MD         
United States, Tennessee
Vanderbilt-Ingram Cancer Center Not yet recruiting
Nashville, Tennessee, United States, 37232
Contact: Natalie Resczenko    615-936-1522    natalie.resczenko@Vanderbilt.Edu   
Principal Investigator: Valerie Brown, MD         
United States, Texas
Dell Children's Blood and Cancer Center Recruiting
Austin, Texas, United States, 78723
Contact: Letitia Holden, RN    512-628-1902    lholden@sfcaustin.com   
Principal Investigator: Sharon Lockhart, MD         
United States, Utah
Primary Children's Hospital Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Lisa Smith    801-662-4710    Lisa.Smith@imail.org   
Principal Investigator: Mark Fluchel, MD         
Sponsors and Collaborators
Giselle Sholler
Van Andel Research Institute
Dell, Inc.
Investigators
Study Chair: Giselle Sholler, MD The Spectrum Health Group
  More Information

Additional Information:
No publications provided

Responsible Party: Giselle Sholler, Director of Pediatric Oncology Translational Research Program, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01802567     History of Changes
Other Study ID Numbers: NMTRC008
Study First Received: February 26, 2013
Last Updated: February 5, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Medulloblastoma
Neoplasms
Neuroblastoma
Glioma
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neuroectodermal Tumors, Primitive
Neuroectodermal Tumors, Primitive, Peripheral

ClinicalTrials.gov processed this record on October 22, 2014