Molecular-Guided Therapy for Childhood Cancer
Molecular-guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancers
1 other identifier
interventional
186
2 countries
19
Brief Summary
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup. This technology includes a genomic report based on DNA exomes and RNA sequencing that will be used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started Jul 2014
Longer than P75 for not_applicable
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 11, 2014
CompletedFirst Posted
Study publicly available on registry
June 13, 2014
CompletedStudy Start
First participant enrolled
July 8, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 18, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
January 18, 2024
CompletedResults Posted
Study results publicly available
April 5, 2024
CompletedNovember 3, 2025
October 1, 2025
9.5 years
June 11, 2014
March 4, 2024
October 29, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers.
The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy.
2 years
Secondary Outcomes (3)
Number of Participants With an Unexpected Adverse Events as a Measure of Safety
Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 4 years.
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.
Followed until off therapy, generally 4 years
Progression Free Survival (PFS) Interval Will be Measured by Days and Compared to the PFS of Previous Chemotherapy Regimens Since Relapse for Each Patient.
From date of start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 4 years
Study Arms (1)
Guided Therapy
EXPERIMENTALA 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
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
You may qualify if:
- 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)
- +16 more criteria
You may not qualify if:
- Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to biopsy
- 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 any investigational drug concurrently.
- Subjects with uncontrolled serious infections or a life-threatening illness (unrelated to tumor)
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Giselle Shollerlead
- Translational Genomics Research Institutecollaborator
- Dell, Inc.collaborator
Study Sites (19)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
The Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
Randall Children's Hospital
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
Dell Children's Blood and Cancer Center
Austin, Texas, 78723, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
American University of Beirut Medical Center
Beirut, Lebanon
Related Publications (1)
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.
PMID: 38347552DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Giselle Sholler, MD
- Organization
- Penn State Health Children's Hospital
Study Officials
- STUDY CHAIR
Giselle Sholler, MD
Beat Childhood Cancer at Atrium Health
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Study Chair
Study Record Dates
First Submitted
June 11, 2014
First Posted
June 13, 2014
Study Start
July 8, 2014
Primary Completion
January 18, 2024
Study Completion
January 18, 2024
Last Updated
November 3, 2025
Results First Posted
April 5, 2024
Record last verified: 2025-10