Can a Child's tumor DNA guide better cancer treatment?
NCT ID NCT02162732
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether analyzing the DNA and RNA of a child's tumor could help doctors choose personalized treatments. It included 186 children with relapsed or hard-to-treat cancers like neuroblastoma and brain tumors. The goal was to see if this approach was feasible and safe, not to cure the cancer directly.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this approach could help doctors select more effective treatments for children with hard-to-treat cancers based on their tumor's unique genetic makeup.
- What could go wrong
- This was an early feasibility study, not a treatment trial. It tested whether the technology works, not whether it improves outcomes. Results may not lead to better survival.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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186 people
The number who actually took part.
- Started
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Jul 2014
- Finished
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Jan 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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13 months to 21 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Subjects must have proven pediatric cancer with confirmation at diagnosis or at the time of recurrence/progression and clinical determination of disease for which there is no known effective curative therapy or disease that is refractory to established proven therapies fitting into one of the following categories: * 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: 1. Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to biopsy 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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American University of Beirut Medical Center
Beirut, Lebanon
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
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Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
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Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, 55404, United States
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Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
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Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
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Dell Children's Blood and Cancer Center
Austin, Texas, 78723, United States
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813, United States
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Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
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Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Rady Children's Hospital
San Diego, California, 92123, United States
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Randall Children's Hospital
Portland, Oregon, United States
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Texas Children's Cancer and Hematology Centers
Houston, Texas, 77030, United States
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The Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
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