Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT01355679
First received: May 13, 2011
Last updated: June 16, 2016
Last verified: June 2016
  Purpose

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.


Condition Intervention
Neuroblastoma
Device: Guided Therapy

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Spectrum Health Hospitals:

Primary Outcome Measures:
  • Percentage of Participants That Are Able to Meet Feasibility Parameters. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Number of Participants With Adverse Events as a Measure of Safety [ Time Frame: 1 year ] [ 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 Using RECIST Criteria [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

  • Activity of Treatments Chosen Based on Progression Free Survival (PFS) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions


Enrollment: 16
Study Start Date: May 2011
Study Completion Date: January 2015
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Guided therapy
A total of 14 eligible neuroblastoma 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
A total of 14 eligible neuroblastoma 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 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).

  Eligibility

Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  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: NCT01355679

Locations
United States, Connecticut
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
United States, Florida
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, United States, 32806
United States, Maryland
National Cancer Institute
Bethesda, Maryland, United States, 20877
United States, Michigan
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
United States, Missouri
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Medical Center
St. Louis, Missouri, United States, 63104
United States, North Carolina
Levine Children's Hospital
Charlotte, North Carolina, United States, 28204
Sponsors and Collaborators
Giselle Sholler
Investigators
Study Chair: Giselle Sholler, MD The Spectrum Health Group
  More Information

Additional Information:
Responsible Party: Giselle Sholler, Study Chair, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01355679     History of Changes
Obsolete Identifiers: NCT01375517
Other Study ID Numbers: NMTRC 001 
Study First Received: May 13, 2011
Results First Received: May 18, 2015
Last Updated: June 16, 2016
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on September 23, 2016