- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01802567
Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
January 10, 2024 updated by: Giselle Sholler
A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancer
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
48
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Arizona
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Phoenix, Arizona, United States, 85016
- Phoenix Children's Hospital
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California
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San Diego, California, United States, 92123
- Rady Children's Hospital
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Connecticut
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Hartford, Connecticut, United States, 06106
- Connecticut Children's Hospital
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Florida
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Orlando, Florida, United States, 32806
- Arnold Palmer Hospital for Children- MD Anderson
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Hawaii
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Honolulu, Hawaii, United States, 96813
- Kapiolani Medical Center for Women and Children
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Michigan
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Grand Rapids, Michigan, United States, 49503
- Helen DeVos Children's Hospital
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Missouri
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Kansas City, Missouri, United States, 64108
- Children's Mercy Hospitals and Clinics
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Saint Louis, Missouri, United States, 63104
- Cardinal Glennon Children's Medical Center
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North Carolina
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Charlotte, North Carolina, United States, 28204
- Levine Children's Hospital
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South Carolina
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Charleston, South Carolina, United States, 29425
- Medical University of South Carolina
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Tennessee
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Nashville, Tennessee, United States, 37232
- Vanderbilt-Ingram Cancer Center
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Texas
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Austin, Texas, United States, 78723
- Dell Children's Blood and Cancer Center
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Utah
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Salt Lake City, Utah, United States, 84113
- Primary Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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).
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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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."
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2 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Number of Participants with Adverse Events as a Measure of Safety
Time Frame: 2 years
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To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
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2 years
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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
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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.
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2 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Study Chair: Giselle Sholler, MD, Beat Childhood Cancer at Atrium Health
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2013
Primary Completion (Estimated)
February 1, 2024
Study Completion (Estimated)
December 1, 2025
Study Registration Dates
First Submitted
February 26, 2013
First Submitted That Met QC Criteria
February 27, 2013
First Posted (Estimated)
March 1, 2013
Study Record Updates
Last Update Posted (Actual)
January 12, 2024
Last Update Submitted That Met QC Criteria
January 10, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Neoplasms by Histologic Type
- Neoplasms
- Neoplasms, Glandular and Epithelial
- Glioma
- Neoplasms, Neuroepithelial
- Neuroectodermal Tumors
- Neoplasms, Germ Cell and Embryonal
- Neoplasms, Nerve Tissue
- Neuroectodermal Tumors, Primitive
- Neuroectodermal Tumors, Primitive, Peripheral
- Medulloblastoma
- Neuroblastoma
Other Study ID Numbers
- NMTRC008
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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