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

Clinical trial: Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

Summary: The purpose of this study is to evaluate the feasibility of giving four weekly doses of Rituximab (anti-CD20 antibody) in the treatment of children with refractory neuroblastoma associated opsoclonus-myoclonus. Patients must have continued symptoms of opsoclonus, myoclonus and or ataxia despite surgical resection and a minimum of one month of steroid therapy. Evaluations include clinical symptoms of opsoclonus-myoclonus and ataxia as well as detailed evaluation of learning and development.

Detailed description: Opsoclonus-myoclonus ataxia syndrome (OMS) is a rare immune mediated paraneoplastic syndrome that occurs in approximately 2 to 3% of children with neuroblastoma. Children with neuroblastoma associated opsoclonus-myoclonus tend to have a favorable prognosis from the standpoint of the cure of their cancer. Unfortunately,approximately two-thirds of this subgroup of patients are left with long term sequellae of the syndrome, including residual symptoms of opsoclonus, myoclonus, ataxia, learning difficulties and disturbance of sleep and mood.
  Multiple lines of evidence indicate an immune mechanism to this rare disorder. This includes occurence of OMS in the post-infectious state, aggressive lymphocytic infiltration of the tumor in children with OMS, and documented responses to therapries that act through suppression of the immune system.
  The current study utilizes four weekly doses of anti-CD 20 antibody (rituximab) to treat children with refractory OMS. Refractory disease is defined as continued symptoms of OMS despite surgical resection of the tumor and a minimum of one month of steroid therapy.
  All patients have baseline OMS evaluation and detailed neurocognitive testing with all studies being repeated at the completion of the four weekly infusions. OMS testing is repeated at Month 3. OMS testing and detailed neurocognitive testing is conducted at 6 months intervals until 2 years from the initial infusion.
  The goal of the study is to utilize this novel therapy to improve long term neurologic and neurodevelopmental outcome in children with refratory neuroblastoma associated opsoclonus-myoclonus.

Eligibility: Inclusion Criteria:
  Pathologic confirmation of diagnosis of neuroblastoma Surgical resection of primary tumor Symptoms of OMS despite a minimum of one month of steroid therapy Must meet all laboratory criteria to demonstrate adequate organ function -
  Exclusion Criteria:
  Patients currently receiving systemic chemotherapy for treatment of neuroblastoma Patients with documented active infection Patients who are HIV, Hep B or Hep C positive Organ toxicity from any prior therapy or surgical intervention must be resolved prior to study entry

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

Clinical trial: The MOM Program: 5 Year Follow-up Study of a Home Visiting Program at The Children's Hospital of Philadelphia

Summary: The purpose of the MOM Program Continuation is to promote child development by helping families become more competent in accessing and using available health, developmental and educational resources. The program focuses are childhood immunizations, Early Intervention services, lead screening, Early Head Start and Head Start enrollment. The Intervention consists of frequent phone calls and home visits to encourage mothers to have their babies immunized on schedule and to participate in needed developmental and educational services. The program seeks to fill the gap between children's need for services and mothers' ability to assure their children's participation in those services.

Detailed description: The MOM Program Continuation builds on the work of The MOM Program, a 3 year randomized intervention. The premise of the MOM Program was that professional support through home visits and frequent phone contacts would improve children's cognitive and behavioral development, ultimately improving their early educational success. The children were enrolled in the MOM Program at birth. The MOM Program Continuation provides continued intervention for the children and mothers of the original cohort to assure that the children are prepared for kindergarten entry at 5 years of age and to evaluate the children's cognitive and behavioral outcomes at age 5 years. The program will also create a longitudinal database to study the linkages between maternal cognitive ability and child cognitive, educational, and behavioral outcomes through entry into school.

Eligibility: Inclusion Criteria: Previous enrollment in The MOM Program; child age of 5 years (plus or minus 3 months); willingness to come to office for follow-up assessment visit; parental consent and child assent.
  Exclusion Criteria: None.

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

Clinical trial: Topotecan, Vincristine, and Doxorubicin in Treating Young Patients With Refractory Stage 4 Neuroblastoma

Summary: RATIONALE: Drugs used in chemotherapy, such as topotecan, vincristine, and doxorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells.
  PURPOSE: This phase II trial is studying how well giving topotecan together with vincristine and doxorubicin works in treating young patients with refractory stage 4 neuroblastoma.

Detailed description: OBJECTIVES:
  Primary
  -  Assess whether treatment with topotecan hydrochloride, vincristine, and doxorubicin hydrochloride can achieve a satisfactory response rate in pediatric patients with stage 4 neuroblastoma that failed to respond to rapid first-line treatment.
  Secondary
  -  Determine time to progression in these patients.
  -  Determine the toxicity of this regimen in these patients.
  OUTLINE: This is a multicenter, open-label study.
  Patients receive topotecan hydrochloride IV over 30 minutes on days 1-5 and vincristine IV continuously and doxorubicin hydrochloride IV continuously over 48 hours on days 5 and 6. Patients also receive filgrastim (G-CSF) subcutaneously beginning on day 9 and continuing until blood counts recover. Treatment repeats every 21-28 days for 2 courses in the absence of disease progression or unacceptable toxicity. Patients who achieve partial response (PR) after two courses of treatment receive an additional two courses. Patients who achieve complete response or very good PR are treated according to the standard therapy in protocol SIOP-EUROPE- HR-NBL-1. Patients who fail to achieve PR after 2 courses receive further treatment at the physician's discretion.
  Patients are followed periodically for at least 3 years.
  PROJECTED ACCRUAL: A total of 63 patients will be accrued for this study.

Eligibility: DISEASE CHARACTERISTICS:
  -  Diagnosis of stage 4 neuroblastoma
  -  Failed to achieve adequate metastatic partial response after first-line therapy while enrolled on protocol SIOP-EUROPE-HR-NBL-1
  PATIENT CHARACTERISTICS:
  -  Neutrophil count > 1,000/mm³
  -  Platelet count ≥ 100,000/mm³
  -  Creatinine ≤ 1.36 mg/dL
  -  Bilirubin ≤ 2.92 mg/dL
  -  AST and ALT < 2.5 times upper limit of normal
  -  Glomerular filtration rate ≥ 60 mL/min
  -  Normal cardiac function on echocardiography
  -  No severe organ dysfunction
  -  No active hepatitis C or hepatitis B virus positivity
  -  No HIV infection
  PRIOR CONCURRENT THERAPY:
  -  No anti-tumor chemotherapy within the past 10 days
  -  No radiotherapy within the past 30 days
  -  No other investigational drugs within the past 30 days
  -  No prior doxorubicin hydrochloride

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

Clinical trial: Observation of Young Patients With Localized Neuroblastoma Who Have Undergone Surgery Only

Summary: RATIONALE: Observation is closely monitoring a patient's condition and not giving treatment until symptoms appear or change. Observation may help doctors see how effective surgery is in treating neuroblastoma.
  PURPOSE: This phase II trial is studying how well surgery alone works in treating young patients with localized neuroblastoma.

Detailed description: OBJECTIVES:
  Primary
  -  Evaluate the safety and efficacy of surgical treatment alone in young patients with stage 2 neuroblastoma without N-myc amplification (NMA).
  Secondary
  -  Determine predictive factors of relapse and survival of patients with stage 1, 2A, or 2B neuroblastoma without NMA who have undergone surgery only.
  OUTLINE: This is a nonrandomized, multicenter study.
  Patients with stage 1 or 2 disease and no N-myc amplification (NMA) undergo observation comprising clinical evaluation, ultrasound or CT scan of the abdomen, and chest x-ray periodically for up to 5 years. All other patients undergo additional therapy and follow-up according to national standards. Patients who develop recurrent disease or disease progression may undergo surgical resection, preceded or followed by chemotherapy, according to national standards.
  PROJECTED ACCRUAL: A total of 140 patients will be accrued for this study.

Eligibility: DISEASE CHARACTERISTICS:
  -  Histologically confirmed neuroblastoma or ganglioneuroblastoma
  -  Meets 1 of the following International Neuroblastoma Staging System (INSS) criteria:
  -  Stage 1
  -  Stage 2A or 2B meeting 1 of the following criteria:
  -  With or without N-myc amplification
  -  No evaluation of NMA
  -  Symptomatic spinal cord compression
  -  Stage 3*
  -  Dumbbell syndrome with clinical signs of spinal cord compression*
  -  NOTE: *These patients are eligible for the study but do not undergo observation; instead they will undergo standard treatment
  -  Has undergone complete or gross surgical resection OR diagnostic surgical or needle biopsy
  -  No metastases within 1 month of diagnosis
  -  No skin metastases by clinical examination and MIBG scan
  -  Normal liver by CT scan or ultrasonography
  -  Normal chest X-ray (in case of nonthoracic primary site)
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  See Disease Characteristics
  -  Prior steroids allowed
  -  No prior chemotherapy

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

Clinical trial: A Study of Pemetrexed in Children With Recurrent Cancer

Summary: To determine the response rate of pemetrexed given every 21 days for the treatment of children with relapsed or refractory osteosarcoma, Ewing's sarcoma/peripheral primitive neuroectodermal tumors (PNET), rhabdomyosarcoma, neuroblastoma, ependymoma, medulloblastoma/supratentorial PNET or non-brain stem high-grade glioma.


Eligibility: Inclusion Criteria:
  -  Patients must have osteosarcoma, Ewing's sarcoma, medulloblastoma, neuroblastoma, rhabdomyosarcoma, ependymoma or high-grade non-brainstem glioma
  -  Measurable disease
  -  Eastern Cooperative Oncology Group (ECOG) performance 0,1,2
  -  Adequate renal, liver and bone marrow function
  -  Patient's current disease state must be one with no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
  Exclusion Criteria:
  -  Growth factors that support platelet or white cell number or function must not have been administered within the last 7 days prior to enrollment (14 days if Neulasta)
  -  Patients with central nervous system (CNS) tumors who have not been on a stable or decreasing dose of dexamethasone or other corticosteroid for 7 days prior to enrollment
  -  Patients with uncontrolled infection
  -  Patients who have received pemetrexed previously
  -  Patients with pleural effusions or ascites

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

Clinical trial: A Study Of Oral PF-02341066, A C-Met/Hepatocyte Growth Factor Tyrosine Kinase Inhibitor, In Patients With Advanced Cancer

Summary: PF-02341066 may work in cancer by blocking the cell growth, migration and invasion of tumor cells. PF-02341066 is a new class of drugs called c-Met/Hepatocyte growth factor receptor tyrosine kinase inhibitors. This compound is also an inhibitor of the anaplastic lymphoma kinase (called ALK) tyrosine kinase and ROS receptor tyrosine kinases. This research study is the first time PF-02341066 will be given to people. PF-02341066 is taken by mouth daily.


Eligibility: Inclusion Criteria:
  -  Advanced malignancies (except leukemias), histologically proven at diagnosis; Histologically confirmed advanced malignancies that are known to be sensitive to PF-03241066 inhibition, e.g. ALK, c-MET and ROS
  -  Solid tumors must have measurable disease (Recommended Phase 2 Dose Cohort patients with non-measurable disease may enter on a case-by-case basis); not required for DDI sub-studies.
  -  Adequate blood cell counts, kidney function, liver function and Eastern Cooperative Oncology Group (ECOG) score of 0 or 1 (for the Recommended Phase 2 Cohort, a ECOG score of 2 may be allowed on a case-by-case basis)
  Exclusion Criteria:
  -  Major surgery, radiation therapy or anti-cancer therapy within 2 to 4 weeks of starting study treatment, depending on the patient cohort
  -  Prior stem cell transplant except of patients with neuroblastoma, lymphoma or myeloma
  -  Active or unstable cardiac disease or heart attack within 3 months of starting study treatment

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

Clinical trial: I-123-MIBG for Imaging of Neuroblastoma and Pheochromocytoma

Summary: I-123-MIBG is used to image patients with neuroblastoma, pheochromocytoma and other neural crest tumors

Detailed description: Entry criteria--Histologically documented neuroblastoma or pheochromocytoma or strong suspicion of neuroblastoma or pheochromocytoma.
  Exclusion criteria--Pregnancy Randomization--None Procedure--Patients will receive a dose of I-123-MIBG by intravenous injection. Images will obtained at 18-24 hours after administration of the drug and at 42-48 hours as needed. SSKI will be used to block thyroid uptake of free I-123-iodide

Eligibility: Inclusion Criteria:
  -  Histologically proven neuroblastoma or pheochromocytoma or strong suspicion of neuroblastoma or pheochromocytoma
  Exclusion Criteria:
  -  Pregnancy

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

Clinical trial: Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma

Summary: The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCR in children with high-risk neuroblastoma. In the present study, a single arm trial of tandem HDCT/ASCR will be carried out. In the present study, the investigators will investigate whether tandem HDCT/ASCR might improve the survival of patients with high-risk neuroblastoma with acceptable toxicity.

Detailed description: The prognosis of high-risk neuroblastoma after conventional chemoradiotherapy is generally poor. Therefore, a strategy using high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) has been explored to improve the prognosis of patients with high-risk neuroblastoma. This strategy is based on the hypothesis that dose escalation might improve the survival of children with high-risk neuroblastoma. The results of randomized trials comparing HDCT/ASCR with chemotherapy alone showed a better event-free survival (EFS) in the HDCT/ASCR arm than in the continuous chemotherapy arm. However, the overall EFS was unsatisfactory.
  In this context, investigators have examined the efficacy of double or triple tandem HDCT/ASCR to further improve the outcome of high-risk neuroblastoma patients. George et al. carried out a single arm trial of tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 47%) with acceptable toxicity. Kletzel et al. also conducted a single arm trial of triple tandem transplantation and reported improved survival (3-year EFS 57%). They demonstrated that further dose escalation using sequential HDCT/ASCR might result in further improvements in the survival of patients with high-risk neuroblastoma.
  Investigators in the present study also carried out tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 62%) with acceptable toxicity. However, throughout our previous study, multiple modifications were made in the treatment plan, which resulted in significant variability over time between patients. This variability may create doubt as to whether tandem HDCT/ASCR itself resulted in the improved outcome. In addition, toxic death rate was relatively high (15.4%), although final survival rate was very high (best survival rate ever reported). Therefore, prospective study is needed to evaluate the efficacy and toxicity of tandem HDCT/ASCR.

Eligibility: Inclusion Criteria:
  -  Patients with high-risk neuroblastoma
  -  Patients with intermediate-risk neuroblastoma if gross tumor remained after surgery
  Exclusion Criteria:
  -  Patients with progressive disease before high-dose chemotherapy
  -  Patients whose parents want to stop or change the planned treatment
  -  Patients with organ toxicities of NCI grade >2 before high-dose chemotherapy

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

Clinical trial: 131I-MIBG With Myeloablative Chemotherapy and Autologous Stem Cell Transplantation for the Treatment of High-risk Neuroblastoma

Summary: This trial is studying how well iodine I 131 metaiodobenzylguanidine together with combination chemotherapy works in treating patients who are undergoing an autologous peripheral stem cell for high risk or relapsed neuroblastoma.


Eligibility: Inclusion Criteria:
  -  Histologically confirmed Neuroblastoma and/or demonstration of tumor cells in bone marrow with elevated urinary catecholamine metabolites
  -  High risk according COG (Children Oncology Group)OR Relapse OR Refractory
  -  As at least one I131-MIBG avid target lesion determined by diagnostic MIBG scan
  -  Glomerular filtration rate or creatinine clearance > 60 ml/min
  -  No tumor cell in Bone Marrow by routine morphology aspiration and biopsy before peripheral stem cell collection
  -  No active infection
  Exclusion Criteria:
  -  Impaired renal function
  -  No avid I131-MIBG lesion
  -  Active infection

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

Clinical trial: Collecting and Storing Samples of Blood and Tumor Tissue From Patients With Osteosarcoma

Summary: RATIONALE: Collecting and storing samples of tumor tissue and blood from patients to test in the laboratory may help the study of cancer in the future.
  PURPOSE: The purpose of this study is to collect and store samples of blood and tumor tissue from patients with osteosarcoma.

Detailed description: OBJECTIVES:
  -  To collect biological specimens from patients with osteosarcoma at Children's Oncology Group (COG) institutions.
  -  To provide a repository for long-term storage of osteosarcoma-related biological materials.
  -  To make these specimens available to qualified researchers to understand the biology of osteosarcoma and correlate these results with the patients' clinical data.
  OUTLINE: After the initial submission of blood samples, patients may undergo open or closed biopsy in order to obtain fresh and frozen tissue samples as well as paraffin embedded material. Patients who are enrolled at the time of initial diagnosis but then have a definitive surgery or develop recurrent disease may submit additional samples (paraffin block, frozen and fresh tumor tissue, or slides together with blood samples). Autopsy tumor samples may also be submitted.

Eligibility: DISEASE CHARACTERISTICS:
  -  All osteosarcoma patients seen at COG institutions are eligible, including the following:
  -  Newly diagnosed patients
  -  Patients with recurrent disease who were not enrolled at the time of diagnosis
  -  The following specimens from the initial biopsy (and/or definitive surgery if an up-front resection is performed) are required:
  -  Paraffin embedded block OR 30 unstained slides
  -  Pretreatment blood consisting of 5 mL serum, 10 mL plasma, and 10 mL heparinized whole blood
  -  Fresh and frozen tumor specimens
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  Patients are not required to enter a therapeutic study to enroll on this study

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

Clinical trial: Studying Tumor Samples From Young Patients With Neuroblastoma

Summary: This research study is looking at tumor samples from young patients with neuroblastoma. Studying samples of 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

Detailed description: Study Subtype: Ancillary/Correlative Observational Study Model: Cohort Time Perspective: Retrospective Biospecimen Retention: Samples With DNA Biospecimen Description: Tissue Study Population Description: Patients previously enrolled on clinical trial COG-ANBL00B1 Sampling Method: Non-Probability Sample
  PRIMARY OBJECTIVE:
  I. To comprehensively identify and characterize the spectrum and frequency of mutations in ALK across all neuroblastoma disease subsets using methodologies that will be resource neutral to the Children's Oncology Group Neuroblastoma Nucleic Acids Bank.
  SECONDARY OBJECTIVES:
  I. To formulate genetic screening recommendations for newly diagnosed patients with or without a family history of neuroblastoma.
  II. To identify the functionally relevant ALK mutations that can be pharmacologically inhibited.
  III. To test for the prognostic capability of ALK alterations. IV. To determine the clinical significance of ALK mutations and/or genomic rearrangements by combining ALK mutation, amplification, and translocation data overall and within each neuroblastoma risk group and correlating this information with clinical phenotype (i.e., age, International Neuroblastoma Staging System stage, and International Neuroblastoma Pathology Classification); genetic factors (i.e., ploidy, MYCN status); and patient outcome.
  OUTLINE:
  Tumor DNA samples are examined by mutation analysis for germline and somatic mutations in the ALK tyrosine kinase domain. Samples are analyzed by whole genome amplification using polymerase chain reaction and then sequenced for DNA alterations in the entire ALK coding sequence. Samples are also examined for single nucleotide polymorphisms (SNPs) by polymorphism analysis. Exploratory multivariable analysis is performed to test for the prognostic ability of ALK mutations in the presence of other known prognostic variables (i.e., age, International Neuroblastoma Staging System stage, MYCN status, International Neuroblastoma Pathology Classification, and diploidy).
  A subset of tumor DNA samples from high-risk patients will be resequenced for DNA alterations to determine whether or not additional regions in ALK, outside of the tyrosine kinase domain, are prone to mutations and should be sequenced in a larger panel.

Eligibility: Inclusion Criteria:
  -  Tumor DNA samples available from pediatric patients with newly diagnosed neuroblastoma previously enrolled on clinical trial COG-ANBL00B1

Relevance to patient case: not relevant