araag2/TREC_Precision-Medicine huggingface dataset:
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Isotretinoin in Treating Young Patients With High-Risk Neuroblastoma

Summary: RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors plan better treatment for patients receiving isotretinoin.
  PURPOSE: This clinical trial is studying the side effects and best dose of isotretinoin in treating young patients with high-risk neuroblastoma.

Detailed description: OBJECTIVES:
  Primary
  -  To investigate the feasibility of implementing individualized dosing of isotretinoin in patients with high-risk neuroblastoma after course 1 of treatment, based on isotretinoin pharmacokinetics and toxicity.
  -  To minimize the large inter-patient variation in plasma concentrations of isotretinoin.
  -  To ensure that patients are not exposed to potentially sub-optimal plasma concentrations of isotretinoin during long-term treatment, particularly for patients who are not able to swallow isotretinoin capsules.
  Secondary
  -  To obtain preliminary data on the potential impact of isotretinoin therapeutic monitoring on clinical response and toxicity in these patients.
  OUTLINE: This is a multicenter study.
  Patients receive oral isotretinoin twice daily on days 1-14. Courses repeat every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
  Blood samples are collected at baseline and during courses 1-3 for pharmacokinetic studies and determination of each patient's individual therapeutic-dose level requirement. Genotyping to identify genes that metabolize enzymes is conducted via PCR.
  After completion of study therapy, patients are followed up periodically for up to 3 years.

Eligibility: DISEASE CHARACTERISTICS:
  -  Histologically confirmed high-risk neuroblastoma
  -  Concurrent isotretinoin as part of clinical treatment
  PATIENT CHARACTERISTICS:
  -  Not pregnant
  -  Fertile patients must use effective contraception
  -  Has a single- or double-lumen central venous catheter in place
  PRIOR CONCURRENT THERAPY:
  -  See Disease Characteristics

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Impact of Helmet Use in Preschool Children

Summary: -  To test the hypothesis if provision of helmet education and free helmet distribution will significantly increase helmet use in preschool children of low-income families compared to those children receiving helmet education without free helmet distribution;
  -  To test the hypothesis if helmet users in preschool children will significantly reduce head and facial injuries compared to those non-helmet users

Detailed description: Current literature review shows that there were no definite prospective research studies that indicate the effectiveness of helmet promotion and wearing in injury prevention/reduction particularly to head and facial injuries among preschool children of low-income families.
  The magnitude and seriousness of the injury problem in these children in the community could be high. Many injuries may occur that are not treated in emergency departments. Even the occurrence of a minor injury in the face is undesirable. These ages are learning periods to ride, are liable to fall, and are prone to injuries by using, not only, bicycles but also tricycles, scooters, roller skates/roller blades and other riding toys (vehicles).

Eligibility: Inclusion Criteria:
  -  Age 3-5 year olds and their siblings, registered at the Community Action of Northeast Indiana (CANI) Head Start Program
  -  The child must possess a bicycle or a riding toy to ride
  -  The child's caregiver speaks English or Spanish
  Exclusion Criteria:
  -  Age less than two or greater than 5-year olds
  -  3-5 year old children that do not own a bicycle or a riding toy to ride
  -  The child's caregiver does not speak English or Spanish.

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Genetic Biomarkers in Children With Neuroblastoma (Also Known as Neuroblastoma Epidemiology in North America [NENA])

Summary: This research trial studies the genes biomarkers in children with neuroblastoma. Studying the genes in a child's cancer cells may help doctors improve ways to diagnose and treat children with neuroblastoma.

Detailed description: Study Subtype: Ancillary/Correlative Observational Study Model: Case-only Time Perspective: Prospective Biospecimen Retention: Samples With DNA Biospecimen Description: Saliva and Tissue Study Population Description: Patients already registered in ACCRN07 Sampling Method: Probability Sample
  PRIMARY OBJECTIVES:
  I. Evaluate the independent association of common genetic polymorphisms involved in folate, vitamin A, and related metabolic and transport pathways and the risk of neuroblastoma (NB).
  II. Evaluate the joint effects of multiple genes on the risk of NB. III. Evaluate the effects of gene-exposure interactions on the risk of NB. IV. Evaluate genetic effects within NB subgroups defined by age at diagnosis and a Children's Oncology group classification schema based on age, MYCN oncogene status, histology, and DNA ploidy.
  V. Recontact mothers of participating NENA case children, conduct brief web-based screen to ascertain whether the pregnancy including the index NENA child was a multiple (twin, triplet, etc), whether the mother knew if the children were MZ or DZ, and obtain a complete pregnancy history.
  VI. Request a saliva sample from the other twin/multiple sibling of the NENA child.
  VII. Extract DNA from saliva samples and securely store. VIII. Clean new survey data and merge with main NENA study database.
  OUTLINE:
  The biologic mother of the patient is asked to complete a Diet History Questionnaire about diet during pregnancy, and information on demographics, lifestyle factors, medication used during pregnancy, history of breast feeding, and family history of cancer or birth defects. Parents are given ORAgene saliva collection kits for self-collection. Saliva bio-specimen samples are collected from both biologic parents and the patient. Tissue samples previously stored in a tissue bank are obtained for deceased patients, if available. DNA is extracted from samples, amplified and analyzed using real-time polymerase chain reaction (PCR) quantitation assay, and genotyped using single nucleotide polymorphisms.

Eligibility: Inclusion Criteria:
  -  Primary diagnosis of neuroblastoma made at a North American COG institution
  -  Diagnosed between 12/24/2007- 7/31/2013
  -  Diagnosed at < 6 years of age
  -  Biological mother is alive and willing to participate
  -  Questionnaire respondents must understand English or Spanish
  -  Are only those participating in CCRN who have agreed to be contacted for future studies

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: An Investigational Drug, Crizotinib (PF-02341066), Is Being Studied In Tumors, Except Non-Small Cell Lung Cancer, That Are Positive For Anaplastic Lymphoma Kinase (ALK)

Summary: This is a Phase 1 trial evaluating the safety and efficacy of crizotinib in patients with tumors except non-small cell lung cancer that are positive for ALK.


Eligibility: Inclusion Criteria:
  -  histologically or cytologically proven diagnosis of malignancy other than NSCLC
  -  positive for translocation or inversion event involving the ALK gene locus
  -  positive for ALK amplification events
  -  positive for ALK activating point mutations
  Exclusion Criteria:
  -  mutations of amplifications involving the c-Met gene but not the ALK gene
  -  concurrent treatment on another therapeutic clinical trial
  -  prior therapy specifically directed against ALK

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Prognostic Biomarkers in Tumor Tissue Samples From Young Patients With Neuroblastoma

Summary: This research study is studying biomarkers in tumor tissue samples from young patients with neuroblastoma. Studying samples of tissue from patients with cancer in the laboratory may help doctors identify biomarkers and learn more about biomarkers related to cancer

Detailed description: PRIMARY OBJECTIVES:
  I. Determine whether ubiquitin carboxyl-terminal hydrolase 1 (UCHL1) expression is an indirect cancer stem cell marker in pediatric patients with neuroblastoma. Correlate UCHL1 expression with staging information and patient's outcome.
  Study Subtype: Observational Observational Study Model: Case-control Time Perspective: Retrospective Biospecimen Retention: None Retained Biospecimen Description: Tissue Study Population Description: Tissue microarray samples from patients with Neuroblastoma supplied by the COG Biopathology Center Sampling Method: Probability
  OUTLINE:
  Archived tissue microarray samples are analyzed for ubiquitin carboxyl-terminal hydrolase 1 (UCHL1) expression by UCHL1 monoclonal antibody and peroxidase technique. Results are then compared with patients' clinical data, including age at diagnosis, race, date of diagnosis, histology, stage and location at diagnosis, presence of cancer predisposition, date of completion of treatment, surgery performed, type of chemotherapy received, history of recurrence, and status (alive with disease, alive without disease, dead), if available.

Eligibility: Inclusion Criteria:
  -  Diagnosed with neuroblastoma
  -  Tissue microarrays containing normal and neuroblastoma samples provided by the Children's Oncology Group (COG) Biopathology Center

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Biomarker Expression in Samples From Young Patients With Anaplastic Large Cell Lymphoma

Summary: RATIONALE: Studying samples of tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer. it may also help doctors predict how patients will respond to treatment.
  PURPOSE: This research trial studies biomarker expression in tissue samples from young patients with anaplastic large cell lymphoma.

Detailed description: OBJECTIVES:
  -  To determine the expression of immunoregulatory proteins including CD4, FOXP3 and CD276 in anaplastic large cell lymphoma (ALCL).
  -  To correlate the expression of these proteins with the levels of anaplastic lymphoma kinase (ALK) autoantibodies in patient of ANHL0131.
  OUTLINE: Archived tissue microarray samples are analyzed by mass spectrometry and validated by Western blotting, flow cytometry, and IHC.

Eligibility: DISEASE CHARACTERISTICS:
  -  Diagnosis of anaplastic large cell lymphoma
  -  Tissue microarray samples available from the Cooperative Human Tissue Network (CHTN)
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  Not specified

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Biomarkers in Blood and Tissue Samples From Patients With Newly Diagnosed Neuroblastoma

Summary: RATIONALE: Studying samples of blood and tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
  PURPOSE: This research trial studies biomarkers in blood and tissue samples from patients with newly diagnosed neuroblastoma.

Detailed description: OBJECTIVES:
  -  Determine the expression of neuropeptide Y (NPY) and its receptors (Rs) in human neuroblastoma (NB) tissues.
  -  Determine whether BDNF/TrkB and TrkAIII stimulate expression of NPY and its Rs.
  -  Determine whether NPY mediates BDNF- and TrkAIII-induced NB proliferation and survival.
  -  Determine neurotrophins' angiogenic actions.
  -  Identify factors released from NB cells upon NPY stimulation (proteomics).
  -  Determine whether NPY upregulates expression of the identified proteins in NB and their Rs in endothelial cells (ECs).
  -  Test whether inhibition of the identified pathways reduces angiogenic activity of NB-conditioned media.
  -  Determine the mechanisms of NYP actions and signaling pathways.
  -  Test whether blocking NPY-Y2/Y5 pathway reduces NB growth and vascularization in vivo.
  OUTLINE: Archived tumor tissue and serum samples are analyzed for neuropeptide Y and its receptors (Y1, Y2, and Y5) expression, neuroblastoma prognostic factors (MYCN, TrkA, TrkAIII, TrkB, BDNF, and NGF), and angiogenic markers by real-time PCR, IHC, ELISA, radioimmunoassay (RIA), mitogenic assay, caspase 3/7 activity assay, western blots, liquid chromatography, tandem mass spectrometry, proteomic assays, and other assays. Results are then analyzed and compared with patients' clinical data, including stage of disease, its phenotype, prognostic markers, age and gender, and response to treatment.

Eligibility: DISEASE CHARACTERISTICS:
  -  Children with newly diagnosed neuroblastoma or ganglioneuroblastoma
  -  Samples from the Children's Oncology Group (COG) from patients enrolled in clinical trials in institutions in the USA, Canada, and Australia
  -  Paraffin-embedded tumor specimens, RNA isolated from tumor tissues, and patient serum must be available
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  Not specified

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Studying Biomarkers in Samples From Patients With High-Risk Neuroblastoma

Summary: RATIONALE: Studying samples of blood and tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
  PURPOSE: This research trial studies biomarkers in samples from patients with high-risk neuroblastoma.

Detailed description: OBJECTIVES:
  -  To establish telomere length measurement by quantitative polymerase chain reaction (qPCR) as an alternative lengthening of telomeres (ALT) detection method in neuroblastoma (NB).
  -  To determine the frequency of ALT in high-risk NB and the characteristics of ALT+ NB.
  -  To establish C-circle (extra-chromosomal telomeric DNA circles) level as a marker of ALT activity in NB.
  -  To evaluate the prognostic significance of ALT in NB.
  -  To evaluate the utility of the C-circle assay for the detection of circulating tumor DNA in NB patients with an ALT+ tumor.
  OUTLINE: Archived tumor tissue and serum samples are analyzed for telomere length measurement, frequency, and C-circle levels by PCR. Results are then compared with patients' age at diagnosis and outcomes including survival data (event-free and overall survival).

Eligibility: DISEASE CHARACTERISTICS:
  -  Snap-frozen neuroblastoma (NB) tumors collected at diagnosis (Objectives 1 to 3)
  -  High-risk stage 3 or 4 NB AND MYCN non-amplified, i.e., exclude stage 4 infants who are not high-risk
  -  Up to five high-risk (> 18 months, unfavorable histology) stage 3/MYCN non-amplified tumors
  -  Patients preferably treated on protocol COG-A3973 or similar protocols with myeloablative therapy
  -  At least 3 years of follow-up for those with no event (current evidence suggests that ALT+ NBs often relapse late, i.e., 2 years or longer from diagnosis)
  -  NB tumor DNA collected at diagnosis (Objectives 2 & 3)
  -  High-risk stage 3 or 4 NB as for Objective 1, except for MYCN status
  -  Stage 4 tumors are preferred; may include up to seven high-risk stage 3 tumors with similar distribution of MYCN-amplified and non-amplified tumors
  -  Frozen serum from NB patients (Objective 5; 2nd stage of project)
  -  Paired serum obtained at diagnosis from patients with ALT+ or ALT- tumors identified in Objective 2
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  See Disease Characteristics

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Studying Gene Expression in Samples From Younger Patients With Neuroblastoma

Summary: RATIONALE: Studying samples of blood and tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer. It may also help doctors find better ways to treat cancer.
  PURPOSE: This research trial studies gene expression in samples from younger patients with neuroblastoma.

Detailed description: OBJECTIVES:
  -  Determine whether TβRIII expression and TGF-β signaling decrease in advanced-stage neuroblastoma (NBL) and whether these changes are confined to the Schwannian stroma.
  -  Determine whether sTβRIII levels and TGF-β signaling correlate with NBL stage, tumor stroma content, surface TβRIII expression, and TGF-β signaling.
  OUTLINE: Archived paraffin-embedded tissue and plasma samples are analyzed for TβRIII expression, TGF-β signaling, and SMAD3 expression and phosphorylation by immunohistochemistry, enzyme-linked immunosorbent assay (ELISA), and other assays. Surface expression of TβRIII in the neuroblastic and stromal tumor components are correlated with matched circulating levels of soluble TβRIII. Results are then correlated with each patient's outcome data, including stage.

Eligibility: DISEASE CHARACTERISTICS:
  -  Tissue samples and matched plasma samples available from 1 of the following groups:
  -  Patients with low-stage (International Neuroblastoma Staging System [INSS] stage 1 or 2) neuroblastoma (NBL)
  -  Patients with advanced-stage (INSS stage 3 or 4) NBL
  -  Patients with stage 4S NBL
  -  Clinical and/or outcome data associated with the tissue and plasma samples including INSS stage, age, MYCN amplification status, chromosomal alterations, and 5-year survival, if known
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  Not specified

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Blood and Cerebrospinal Fluid for Pediatric Brain Tumor Research

Summary: In normal patients, blood and cerebrospinal fluid (CSF) contain circulating cells and other molecules such as proteins and nucleic acids. In patients with central nervous system (CNS) and other conditions, the levels of these molecules may be altered. In several other studies at our institution, the investigators are investigating such molecules in tumor specimens as well as the blood and cerebrospinal fluid of pediatric patients with CNS tumors. However, these levels are difficult to interpret without comparing them to levels in patients without CNS tumors. The investigators propose a study to collect small amounts of blood and cerebrospinal fluid from pediatric patients without CNS tumors who are undergoing a diagnostic or therapeutic neurosurgical procedure aimed at addressing altered CSF dynamics.


Eligibility: Inclusion Criteria:
  -  Children without central nervous system tumors who are undergoing a neurosurgical procedure to address hydrocephalus during which CSF will be obtained
  -  Between the ages of 1 year and 21 years
  -  Patients must be having blood draws, lumbar punctures or CSF sampling from Ommaya reservoirs or VPS as part of routine clinical care.
  Exclusion Criteria:
  -  Patients who do not require routine blood draws and/or CSF collection as part of their routine clinical care
  -  Patients who are considered too ill to participate as determined by their treating physician
  -  Patients with documented bacterial of viral infections of the CSF, brain parenchyma and/or neurosurgical devices and/or
  -  Patients with suspected de-myelinating conditions
  -  Patients who are pregnant or lactating.

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: An Extension Study to WA19977 in Patients With Active Polyarticular-Course Juvenile Idiopathic Arthritis

Summary: This long-term, interventional, open-label extension study will evaluate the safety and efficacy of RoActemra/Actemra (tocilizumab) in patients from Poland and Russia with polyarticular-course juvenile idiopathic arthritis who completed the WA19977 study. Patients will receive RoActemra/Actemra 8 mg/kg every 4 weeks. The anticipated time on study treatment is 104 weeks.


Eligibility: Inclusion Criteria:
  -  Patients who completed 104 weeks of study WA19977 with at least a JIA ACR30 clinical response to RoActemra/Actemra and no serious adverse event or adverse event
  -  Written informed consent obtained from patient if patient is 18 years of age and older, or if under the age of 18 years from parents or legal guardian
  Exclusion Criteria:
  -  Patient did not benefit from RoActemra/Actemra therapy in study WA19977
  -  Treatment with any investigational drug since the last administration of study drug in the core study WA19977
  -  Patients developed any other autoimmune rheumatic disease other than the permitted JIA subsets
  -  Any significant medical or surgical condition that would jeopardize patient's safety
  -  Current serious uncontrolled concomitant disease or infection

Relevance to patient case: not relevant
Patient case: 4-year-old female with neuroblastoma; genetic feature: ALK

Clinical trial: Neuroblastoma Biology Study

Summary: Medical scientists want to find better ways to treat neuroblastoma and to find ways to prevent the tumor from growing back. To do this, they need more information about the characteristics of neuroblastoma cells. Therefore, they want to study samples of neuroblastoma tissues and neuroblastoma and normal cells in the blood and bone marrow that may be related to the growth of neuroblastoma cells. Doctors and other medical scientists also want to find better ways to detect and measure neuroblastoma to improve the ability to follow the response of tumor cells to therapy.

Detailed description: The purposes of this study are:
  To establish a storage place or bank of samples of blood, bone marrow, and/or tumor, and molecular components isolated from these samples from children with neuroblastoma. The stored specimens will be shared with laboratory researchers studying high risk neuroblastoma.
  To evaluate a new test of blood and bone marrow specimens to find tumor cells. The results of this new test, called 5-gene TaqManÂ® Low Density Array or TLDA, will be compared between blood and bone marrow and imaging (radiology) studies. Radiology studies (CT scans, MRI scans, and MIBG scans) and the TLDA test will be compared for their ability to measure the amount of tumor present and how this changes with therapy.
  To collect clinical data (such as treatments received, date of diagnosis, tumor stage, etc) and radiology scans to provide this information as needed for the laboratory studies to be done on the specimens.
  To obtain neuroblastoma tumor cells from tumor tissue, bone marrow, and/or blood to use to start cell lines, or tumor cells that will keep growing in the laboratory. These cell lines will be shared with laboratory researchers studying high risk neuroblastoma.

Eligibility: Inclusion Criteria:
  -  Patients must be greater than or 31 days of age.
  -  Patients must have had a diagnosis of high risk neuroblastoma either by histological verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
  -  All patients with refractory or recurrent high risk neuroblastoma at NANT institutions are eligible regardless of disease status (including no measurable or evaluable tumor) as long as they undergo a disease evaluation and appropriate samples are submitted.
  -  Additionally, all patients with high risk neuroblastoma without relapse treated at a NANT institution are eligible if undergoing a disease evaluation, as long as Children's Oncology Group specimens are prioritized.
  Exclusion Criteria:
  -  There are no exclusion criteria on this study.

Relevance to patient case: not relevant