Pediatric Solid TumorsMASTNCT01050296
Molecular Analysis Of Solid Tumors
An observational study of Pediatric Solid Tumors, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
This study will prospectively characterize the molecular, cellular and genetic properties of primary and metastatic neuroblastoma, osteosarcoma, retinoblastoma, Ewing sarcoma family of tumors, soft tissue sarcomas, adrenocortical tumors and liver malignancies. Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Pediatric Solid Tumors
- How many people
- 1,000 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Feb 2010to 30 Sep 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients identified at St. Jude Children's Research Hospital and collaborating institutions with a suspected or known diagnosis of neuroblastoma, osteosarcoma, retinoblastoma, Ewing sarcoma or soft tissue sarcoma based initial diagnostic workup and evidence of gross disease amenable to excision.
Who may be able to join
- Must have a suspected or known diagnosis of neuroblastoma, osteosarcoma, Ewing sarcoma family of tumor or soft tissue sarcoma based on the initial diagnostic workup and evidence of gross disease amenable to excision. Specimens may be collected at some or all of the following time points: initial biopsy, bone marrow aspiration procedures, tumor resection, and at time of possible relapse.
- Patients with a diagnosis of retinoblastoma based on initial diagnostic workup and who require enucleation may be enrolled if there is no active therapeutic or biologic protocol for retinoblastoma.
- The patient or his/her legal guardian, as appropriate, must provide written informed consent within 30 days of the removal of the first collection of tissue/bone marrow/blood sample for this protocol.
- The patient is being seen at St. Jude Children's Research Hospital or at a collaborating institution.
- Patients must be less than or equal to 25 years old at the time of enrollment.
Who cannot join
- Patient is known to be Hepatitis B, Hepatitis C and/or HIV positive.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Sara M. Federico, MD, 866-278-5833, referralinfo@stjude.org
Sara M. Federico, MD
About this study
This study will prospectively characterize the molecular, cellular and genetic properties of primary and metastatic neuroblastoma, osteosarcoma, retinoblastoma, Ewing sarcoma family of tumors, soft tissue sarcomas, adrenocortical tumors and liver malignancies. These cell isolates will be used for gene expression array analysis, genomic analysis by [SNP] single nucleotide polymorphism chip, array [CGH] comparative genomic hybridization and next generation sequencing, and [TEM] transmission electron microscopy analysis. Additionally cell lines and orthotopic xenografts will be created from the obtained tumor specimens.
The specificity of TCRs will be examined by comparing paired TCR from peripheral blood and tumor infiltrating CD4+ and CD8+ T cells. Epigenetic studies will be performed looking at the methylation profile of these cells and to investigate the anti-tumor T cell response both pre- and post-PD1 inhibition.
Read the detailed description
Each year approximately 2,200 children in the United States are diagnosed with neuroblastoma, osteosarcoma, Ewing sarcoma family of tumors (ESFT), retinoblastoma, soft tissue sarcomas, adrenocortical tumors and liver malignancies. These aggressive pediatric solid tumors are developmental tumors that initiate during periods of tissue growth and morphogenesis in the neural crest, bone and soft tissues. The overall survival rate of these tumors in the advanced stage is less than 30%. Despite intensive efforts over the past three decades using multiple therapeutic modalities including chemotherapy, surgery, radiation, autologous bone marrow transplant and biological agents there has been modest improvement in the long-term survival of these advanced stage pediatric solid tumors. A better understanding of the molecular, cellular and genetic changes that occur in the developing tissues as tumors form could improve the treatment of these devastating cancers. In particular, chemotherapeutic agents may be more effectively targeted to key regulatory enzymes or proteins if the study had a better understanding of the pathways that are disrupted as cells progress from preneoplastic lesions to metastatic disease. The specific aim of this proposal is to identify the changes in gene expression that occur in neuroblastoma, retinoblastoma, osteosarcoma, Ewing sarcoma family of tumors [ESFT] and soft tissue sarcoma cells and to correlate these changes with genetic and cellular changes in the tumor cells. [RNA] ribonucleic acid and genomic [DNA] deoxyribonucleic acid will be isolated from neuroblastoma, retinoblastoma, osteosarcoma, ESFT [Ewing sarcoma family of tumors] and soft tissue sarcomas (both primary and metastatic lesions) following surgery or bone marrow aspiration of previously untreated patients. Additional testing will be conducted on tumor samples at any point during or following therapy in which a surgical specimen is obtained. When there is sufficient tumor sample remaining after pathological analysis and banking, fresh primary tumor cells will be used to prepare orthotopic xenografts and to establish models of each disease that recapitulate the advanced forms of neuroblastoma, osteosarcoma, Ewing sarcoma family of tumors [ESFT], retinoblastoma and soft tissue sarcomas. For a small group of these excess samples, this study will perform fixation for electron microscopy and process the samples for [TEM] transmission electron microscopy analysis. These studies will complement our active research program characterizing the molecular, cellular and genetic features of genetically engineered mouse models of each of these diseases. Biological samples from the cohort of patients treated at St. Jude Children's Research Hospital will be complemented with samples collected and processed by collaborating institutions around the world. Samples collected from international collaborators will be used for analysis of [DNA] deoxyribonucleic acid and [RNA] ribonucleic acid to complement the St. Jude Children's Research Hospital cohort. Through this collaboration the study anticipates that they will be able to obtain enough fresh tumor samples to improve their understanding of multistage tumorigenesis in pediatric solid malignancies.
- Conditions
- Pediatric Solid Tumors
- Keywords
- Neuroblastoma, Retinoblastoma, Osteosarcoma, Rhabdomyosarcoma, Ewing sarcoma, Non-rhabdomyosarcoma soft tissue sarcoma, Adrenocortical tumors, Liver malignancies
- Medical subject headings
- Neuroblastoma, Retinoblastoma, Osteosarcoma, Rhabdomyosarcoma, Sarcoma, Ewing, Sarcoma
What is being measured
The primary outcome is the main question the study is designed to answer.
Perform analysis of gene expression profiles. [RNA] ribonucleic acid will be isolated from fresh frozen tumor specimens and hybridized to Affymetrix gene expression arrays.
Measured: 5 years
Secondary outcomes (13)
Perform analysis of large chromosomal alterations such as amplifications and deletions. [DNA] deoxyribonucleic acid isolated from the tumor and blood samples will be hybridized to array [CGH] comparative genomic hybridization chips.
Measured: 5 years
Perform analysis of focal alterations in the genome including amplification, deletion and loss of heterozygosity (LOH). DNA isolated from the tumor and blood samples will be hybridized to [SNP] single nucleotide polymorphism chips.
Measured: 5 years
Perform analysis of point mutations. DNA isolated from the tumor and blood samples will be sequenced using next-generation sequencing technology to determine the sequence of the entire genome.
Measured: 5 years
Perform analysis of cell morphology. Tissue samples will be fixed in a buffer suitable for transmission electron microscopy and processed for [TEM] transmission electron microscopy analysis.
Measured: 5 years
Compare micro RNAs in tumors. Micro RNAs will be isolated from the total [RNA] ribonucleic acid left over from the gene expression analysis described above. These samples will be used for micro [RNA] chip analysis or deep sequencing.
Measured: 5 years
Compare epigenetic modifications of [DNA] deoxyribonucleic acid. Genomic DNA will be isolated and DNA methylation will be analyzed across the genome to determine if this epigenetic process is altered in the tumor samples.
Measured: 5 years
Compare epigenetic modifications of chromatin. Cells will be rapidly fixed and chromatin will be prepared in order to directly probe protein-DNA complexes by [ChIP-seq]chromatin immunoprecipitation or [ChIP-chip analysis] chromatin immunoprecipitation
Measured: 5 years
Perform protein analysis. Tumor cells will be rapidly frozen for subsequent isolation of total protein or specific fraction of cellular proteins (i.e. nuclear, cytoplasmic, membrane proteins).
Measured: 5 years
Perform immunohistochemical and FISH analysis of neuroblastoma, osteosarcoma, retinoblastoma, ESFT and soft tissue sarcomas. Tissue microarrays will be prepared from samples collected in this study.
Measured: 5 years
Cell isolates obtained from primary and metastatic tumor specimens will be used to establish cell lines and orthotopic xenografts in immunocompromised mice.
Measured: 5 years
Perform analysis of cell growth, metastasis and sensitivity to chemotherapy.
Measured: 5 years
Evaluate the extent of immune cell infiltration and their functional status in pediatric solid tumors utilizing flow cytometric analysis of fresh tumor samples.
Measured: 5 years
Evaluate tumor-associated macrophages (TAMSs) post-therapy to locate gene expression patterns, markers and functional correlates of the tissue repair properties of macrophages in the post-therapy microenvironment.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2010 (estimated)
- Study start10 Feb 2010
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Solid TumorsStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Alex's Lemonade Stand Foundation
- Responsible party
- Sponsor
- Investigators
- Sara M. Federico, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01050296
- Sponsor's protocol ID
- MAST
- Other IDs
- DOD-W81XWH-14-1-0103(CA130396)
- Acronym
- MAST
- Status last verified
- Nov 2025
- First submitted
- 14 Jan 2010
- Last updated
- 12 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Molecular Analysis Of Solid Tumors
- ClinicalTrials.gov ID
- NCT01050296
- Acronym / sponsor ID
- MAST / MAST
- Conditions
- Pediatric Solid Tumors
- Keywords
- Neuroblastoma, Retinoblastoma, Osteosarcoma, Rhabdomyosarcoma, Ewing sarcoma, Non-rhabdomyosarcoma soft tissue sarcoma, Adrenocortical tumors, Liver malignancies
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Alex's Lemonade Stand Foundation
- Study start
- 10 Feb 2010 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 15 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will prospectively characterize the molecular, cellular and genetic properties of primary and metastatic neuroblastoma, osteosarcoma, retinoblastoma, Ewing sarcoma family of tumors, soft tissue sarcomas, adrenocortical tumors and liver malignancies. These cell isolates will be used for gene expression array analysis, genomic analysis by [SNP] single nucleotide polymorphism chip, array [CGH] comparative genomic hybridization and next generation sequencing, and [TEM] transmission electron microscopy analysis. Additionally cell lines and orthotopic xenografts will be created from the obtained tumor specimens.
The specificity of TCRs will be examined by comparing paired TCR from peripheral blood and tumor infiltrating CD4+ and CD8+ T cells. Epigenetic studies will be performed looking at the methylation profile of these cells and to investigate the anti-tumor T cell response both pre- and post-PD1 inhibition.
Detailed description
Each year approximately 2,200 children in the United States are diagnosed with neuroblastoma, osteosarcoma, Ewing sarcoma family of tumors (ESFT), retinoblastoma, soft tissue sarcomas, adrenocortical tumors and liver malignancies. These aggressive pediatric solid tumors are developmental tumors that initiate during periods of tissue growth and morphogenesis in the neural crest, bone and soft tissues. The overall survival rate of these tumors in the advanced stage is less than 30%. Despite intensive efforts over the past three decades using multiple therapeutic modalities including chemotherapy, surgery, radiation, autologous bone marrow transplant and biological agents there has been modest improvement in the long-term survival of these advanced stage pediatric solid tumors. A better understanding of the molecular, cellular and genetic changes that occur in the developing tissues as tumors form could improve the treatment of these devastating cancers. In particular, chemotherapeutic agents may be more effectively targeted to key regulatory enzymes or proteins if the study had a better understanding of the pathways that are disrupted as cells progress from preneoplastic lesions to metastatic disease. The specific aim of this proposal is to identify the changes in gene expression that occur in neuroblastoma, retinoblastoma, osteosarcoma, Ewing sarcoma family of tumors [ESFT] and soft tissue sarcoma cells and to correlate these changes with genetic and cellular changes in the tumor cells. [RNA] ribonucleic acid and genomic [DNA] deoxyribonucleic acid will be isolated from neuroblastoma, retinoblastoma, osteosarcoma, ESFT [Ewing sarcoma family of tumors] and soft tissue sarcomas (both primary and metastatic lesions) following surgery or bone marrow aspiration of previously untreated patients. Additional testing will be conducted on tumor samples at any point during or following therapy in which a surgical specimen is obtained. When there is sufficient tumor sample remaining after pathological analysis and banking, fresh primary tumor cells will be used to prepare orthotopic xenografts and to establish models of each disease that recapitulate the advanced forms of neuroblastoma, osteosarcoma, Ewing sarcoma family of tumors [ESFT], retinoblastoma and soft tissue sarcomas. For a small group of these excess samples, this study will perform fixation for electron microscopy and process the samples for [TEM] transmission electron microscopy analysis. These studies will complement our active research program characterizing the molecular, cellular and genetic features of genetically engineered mouse models of each of these diseases. Biological samples from the cohort of patients treated at St. Jude Children's Research Hospital will be complemented with samples collected and processed by collaborating institutions around the world. Samples collected from international collaborators will be used for analysis of [DNA] deoxyribonucleic acid and [RNA] ribonucleic acid to complement the St. Jude Children's Research Hospital cohort. Through this collaboration the study anticipates that they will be able to obtain enough fresh tumor samples to improve their understanding of multistage tumorigenesis in pediatric solid malignancies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients identified at St. Jude Children's Research Hospital and collaborating institutions with a suspected or known diagnosis of neuroblastoma, osteosarcoma, retinoblastoma, Ewing sarcoma or soft tissue sarcoma based initial diagnostic workup and evidence of gross disease amenable to excision.
Key inclusion criteria (5 listed)
- Must have a suspected or known diagnosis of neuroblastoma, osteosarcoma, Ewing sarcoma family of tumor or soft tissue sarcoma based on the initial diagnostic workup and evidence of gross disease amenable to excision. Specimens may be collected at some or all of the following time points: initial biopsy, bone marrow aspiration procedures, tumor resection, and at time of possible relapse.
- Patients with a diagnosis of retinoblastoma based on initial diagnostic workup and who require enucleation may be enrolled if there is no active therapeutic or biologic protocol for retinoblastoma.
- The patient or his/her legal guardian, as appropriate, must provide written informed consent within 30 days of the removal of the first collection of tissue/bone marrow/blood sample for this protocol.
- The patient is being seen at St. Jude Children's Research Hospital or at a collaborating institution.
- Patients must be less than or equal to 25 years old at the time of enrollment.
Key exclusion criteria (1 listed)
- Patient is known to be Hepatitis B, Hepatitis C and/or HIV positive.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Perform analysis of gene expression profiles. [RNA] ribonucleic acid will be isolated from fresh frozen tumor specimens and hybridized to Affymetrix gene expression arrays.
Time frame: 5 years
13 secondary outcomes
Perform analysis of large chromosomal alterations such as amplifications and deletions. [DNA] deoxyribonucleic acid isolated from the tumor and blood samples will be hybridized to array [CGH] comparative genomic hybridization chips.
Time frame: 5 years
Perform analysis of focal alterations in the genome including amplification, deletion and loss of heterozygosity (LOH). DNA isolated from the tumor and blood samples will be hybridized to [SNP] single nucleotide polymorphism chips.
Time frame: 5 years
Perform analysis of point mutations. DNA isolated from the tumor and blood samples will be sequenced using next-generation sequencing technology to determine the sequence of the entire genome.
Time frame: 5 years
Perform analysis of cell morphology. Tissue samples will be fixed in a buffer suitable for transmission electron microscopy and processed for [TEM] transmission electron microscopy analysis.
Time frame: 5 years
Compare micro RNAs in tumors. Micro RNAs will be isolated from the total [RNA] ribonucleic acid left over from the gene expression analysis described above. These samples will be used for micro [RNA] chip analysis or deep sequencing.
Time frame: 5 years
Compare epigenetic modifications of [DNA] deoxyribonucleic acid. Genomic DNA will be isolated and DNA methylation will be analyzed across the genome to determine if this epigenetic process is altered in the tumor samples.
Time frame: 5 years
Compare epigenetic modifications of chromatin. Cells will be rapidly fixed and chromatin will be prepared in order to directly probe protein-DNA complexes by [ChIP-seq]chromatin immunoprecipitation or [ChIP-chip analysis] chromatin immunoprecipitation
Time frame: 5 years
Perform protein analysis. Tumor cells will be rapidly frozen for subsequent isolation of total protein or specific fraction of cellular proteins (i.e. nuclear, cytoplasmic, membrane proteins).
Time frame: 5 years
Perform immunohistochemical and FISH analysis of neuroblastoma, osteosarcoma, retinoblastoma, ESFT and soft tissue sarcomas. Tissue microarrays will be prepared from samples collected in this study.
Time frame: 5 years
Cell isolates obtained from primary and metastatic tumor specimens will be used to establish cell lines and orthotopic xenografts in immunocompromised mice.
Time frame: 5 years
Perform analysis of cell growth, metastasis and sensitivity to chemotherapy.
Time frame: 5 years
Evaluate the extent of immune cell infiltration and their functional status in pediatric solid tumors utilizing flow cytometric analysis of fresh tumor samples.
Time frame: 5 years
Evaluate tumor-associated macrophages (TAMSs) post-therapy to locate gene expression patterns, markers and functional correlates of the tissue repair properties of macrophages in the post-therapy microenvironment.
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Nov 2025.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sara M. Federico, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Solid Tumors, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 12 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sara M. Federico, MD (1-866-278-5833, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Perform analysis of gene expression profiles. [RNA] ribonucleic acid will be isolated from fresh frozen tumor specimens and hybridized to Affymetrix gene expression arrays. (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2025.