Study Results
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View full resultsBasic Information
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COMPLETED
NA
186 participants
INTERVENTIONAL
2014-07-08
2024-01-18
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Guided Therapy
A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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).
Guided Therapy
A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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).
Interventions
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Guided Therapy
A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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).
Eligibility Criteria
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Inclusion Criteria
* Neuroblastoma- Patients that have relapsed following standard of care therapy (such as high risk patients, patient presenting after age 15 months or MYCN amplified, and only following (for eligible patients) high-dose chemotherapy followed by hematopoietic stem cell transplantation and maintenance therapy with retinoic acid and antibody therapy) or having progressed during standard of care therapy and non-responsive/progressive to accepted curative chemotherapy.
* Brain Tumors
* Medulloblastomas (At relapse after standard of care therapy \[surgery, chemotherapy and/or radiation\] and/or non-responsive/progressive on accepted curative therapy)
* Gliomas (At relapse after standard of care therapy \[surgery and/or radiation and/or chemotherapy\] and/or non-responsive/progressive on accepted curative therapy)
* Ependymomas (At relapse after standard of care therapy \[surgery with or without radiation\] and/or non-responsive/progressive on accepted curative therapy)
* Choroid plexus tumors (At relapse after standard of care therapy \[surgery\] and/or non-responsive/progressive on accepted curative therapy)
* Craniopharyngiomas (At relapse after standard of care therapy \[surgery or suppressive therapy\] and/or non-responsive/progressive on accepted curative therapy)
* Dysembryoplastic neuroepithelial tumors (DNETs) (At relapse after standard of care therapy \[surgery\] and/or non-responsive/progressive on accepted curative therapy)
* Meningiomas (At relapse after standard of care therapy \[surgery\] and/or non-responsive/progressive on accepted curative therapy)
* Primitive Neuroectodermal Tumors (PNETs) (At relapse after standard of care therapy \[surgery, chemotherapy, and/or radiation\] and/or non-responsive/progressive on accepted curative therapy)
* Germ cell tumors (At relapse after standard of care therapy \[surgery, and/or radiation and/or chemotherapy\] and/or non-responsive/progressive on accepted curative therapy)
* Rare Tumors:
* Soft tissue sarcoma Rhabdomyosarcoma (At relapse after standard of care therapy \[surgery, and/or radiation, chemotherapy\] and/or non-responsive/progressive to accepted curative chemotherapy) Non-rhabdomyosarcoma (At relapse after standard of care therapy \[surgery, and/or radiation, chemotherapy\] and/or non-responsive/progressive to accepted curative chemotherapy)
* Bone Ewings sarcoma (At relapse after standard of care therapy \[surgery, and/or radiation, chemotherapy\] and/or non- responsive/progressive to accepted curative chemotherapy) Osteosarcoma (At relapse after standard of care therapy \[surgery, chemotherapy\] and/or non- responsive/progressive to accepted curative chemotherapy)
* Renal Wilms tumor (At relapse after standard of care therapy \[surgery, and/or radiation, chemotherapy\] and/or non- responsive/progressive to accepted chemotherapy) Renal cell carcinoma (At relapse after standard of care therapy \[surgery, chemotherapy\] and/or non- responsive/progressive to accepted curative chemotherapy) Malignant rhabdoid tumor (At diagnosis, as there is no known curative therapy) Clear Cell Sarcoma- (At relapse after standard of care therapy \[radiation, chemotherapy\] and/or non- responsive/progressive to accepted curative chemotherapy) Germ Cell tumors (At relapse after standard of care therapy \[surgery, chemotherapy\] and/or non-responsive/progressive to accepted curative chemotherapy)
* Liver Tumors (At relapse after standard of care therapy \[surgery, chemotherapy\] and/or non- responsive/progressive to accepted curative chemotherapy)
2. Subjects must be age \>12 months at enrollment
3. Subjects must be age ≤ 21 years at initial diagnosis
4. Subjects must have measurable disease as demonstrated by residual abnormal tissue at a primary or metastatic site (measurable on CT or MRI) at the time of biopsy; tumor must be accessible for biopsy. In addition, subjects with bone or bone marrow only disease expected to be \>75% tumor are eligible to enroll.
5. Current disease state must be one for which there is currently no known effective therapy
6. Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
7. Lansky or Karnofsky Score must be ≥ 50
8. Subjects without bone marrow metastases must have an ANC \> 750/μl to begin treatment.
9. Subjects with CNS disease must have been on a stable dose of steroids for 2 weeks prior to their biopsy and must not have progressive hydrocephalus at enrollment.
10. Adequate liver function must be demonstrated, defined as:
* Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
* ALT (SGPT) \< 10 x upper limit of normal (ULN) for age
11. A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
12. 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.
13. Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria
2. 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).
3. Subjects receiving any investigational drug concurrently.
4. Subjects with uncontrolled serious infections or a life-threatening illness (unrelated to tumor)
5. 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
13 Months
21 Years
ALL
No
Sponsors
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Translational Genomics Research Institute
OTHER
Dell, Inc.
INDUSTRY
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Study Chair
Principal Investigators
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Giselle Sholler, MD
Role: STUDY_CHAIR
Beat Childhood Cancer at Atrium Health
Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
Rady Children's Hospital
San Diego, California, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
Arnold Palmer Hospital for Children
Orlando, Florida, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States
The Children's Hospital at Montefiore
The Bronx, New York, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Randall Children's Hospital
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States
Dell Children's Blood and Cancer Center
Austin, Texas, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
American University of Beirut Medical Center
Beirut, , Lebanon
Countries
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References
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Sholler GLS, Bergendahl G, Lewis EC, Kraveka J, Ferguson W, Nagulapally AB, Dykema K, Brown VI, Isakoff MS, Junewick J, Mitchell D, Rawwas J, Roberts W, Eslin D, Oesterheld J, Wada RK, Pastakia D, Harrod V, Ginn K, Saab R, Bielamowicz K, Glover J, Chang E, Hanna GK, Enriquez D, Izatt T, Halperin RF, Moore A, Byron SA, Hendricks WPD, Trent JM. Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers: a Beat Childhood Cancer Research Consortium trial. Genome Med. 2024 Feb 12;16(1):28. doi: 10.1186/s13073-024-01297-5.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Document Type: Informed Consent Form
Related Links
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Beat Childhood Cancer
Other Identifiers
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NMTRC009
Identifier Type: -
Identifier Source: org_study_id
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