[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pleuropulmonary-blastoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pleuropulmonary-blastoma":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,48,74,118],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":16,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":31,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":4,"leadSponsor":44,"locationsCount":47},"100151781","dicer1-related-pleuropulmonary-blastoma-cancer-predisposition-syndrome-a-natural-history-study-100151781",false,"NCT01247597","DICER1-related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study","DICER1-Related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study","* INCLUSION CRITERIA:\n\nAll participants who meet the eligibility criteria outlined below will be eligible for inclusion in this study regardless of their race, gender, ethnicity, or age.\n\n* Affected individual is defined as:\n\n  * an individual with histologically-confirmed PPB and\u002For other DICER1-related tumors\n  * an individual with a known or suspected DICER1 disease-associated variant\n  * an individual from the general population with one or more of the unique tumors of the types associated with DICER1 including (but not exclusively), PPB, cystic nephroma, ovarian Sertoli-Leydig cell and other sex cord-stromal tumors, ocular medulloepithelioma, nasal chondromesenchymal hamartoma, Wilms tumor, embryonal rhabdomyosarcoma, pineoblastoma, pituitary blastoma, ovarian sarcoma, CNS sarcoma and\u002For thyroid cancer - regardless of their family history. Additional DICER1-related neoplasms may be identified in the future, and they will be added to the protocol as needed.\n* Unaffected individual is defined as:\n\n  * a family member (such as parents, siblings, children, or extended family) of an affected participant without a known or suspected DICER1 disease-associated variant or condition and they will be controls.\n\nOther inclusion criteria include:\n\n* All types and amounts of prior therapies are allowed.\n* There is no age restriction.\n* There is no restriction related to organ and marrow function.\n* Ability of the individual or their legal guardian or appropriate surrogate to understand, and their willingness to provide informed consent.\n\nNeonates of affected individuals will be included in the Field Cohort and be eligible for genetic counseling, education, and testing, if indicated and consented by a parent\u002Flegal guardian\u002FLAR.\n\nThis is entirely a function of meeting the inclusion criteria and not being excluded by the exclusion criteria.\n\nIn some instances, patients with histologically-confirmed PPB and\u002For another neoplasm within the DICER1-related tumor risk and their families will be referred to the Clinical Genetics Branch (CGB) by the International Pleuropulmonary Blastoma (PPB) \u002F DICER1 Registry (IPPBR), provided that the family has previously or currently indicated a desire to be notified of such research opportunities. In non IPPBR cases, the diagnosis will be confirmed by reviewing relevant medical records and relevant surgical pathology material.\n\nEXCLUSION CRITERIA:\n\nIndividuals and families referred for evaluation in whom reported diagnoses are not verifiable.",true,"ALL","1 Month","99 Years",{"count":21,"type":22},1500,"ESTIMATED","OBSERVATIONAL","Background:\n\n\\- Pleuropulmonary blastoma (PPB) is a rare fast-growing lung tumor that is associated with other, rare tumor types. Most cases of PPB appear in children younger than 6 years of age. Recently, it has been shown that this condition can be inherited (e.g., mutation of the DICER1 gene). Researchers are studying both clinical and genetic aspects of this newly described condition. They are interested in collecting further medical history and genetic information on individuals and close relatives of individuals who have PPB or other rare associated tumors.\n\nObjectives:\n\n\\- To study individuals with a personal or a family history of pleuropulmonary blastoma (PPB) or other rare tumors that can be associated with PPB (e.g., cystic nephroma, nasal chondromesenchymal hamartoma, ovarian Sertoli-Leydig cell tumors, ocular medulloepithelioma).\n\nEligibility:\n\n* Individuals who have been diagnosed with PPB and\u002For PPB-related tumors.\n* Close blood relatives (e.g., parents, siblings, grandparents) of individuals who have been diagnosed with PPB and\u002For PPB-related tumors.\n\nDesign:\n\n* Interested participants can enroll or inquire about this study by calling 1-800-518-8474.\n* Participants will be asked to complete family history and medical history questionnaires. They will complete the questionnaire if they are at least 18 years of age, or another person will complete the questionnaire if the key family member is too young to do so on his or her own.\n* Participants will be asked to sign a medical record release form to allow researchers to examine detailed medical history information.\n* Participants may be asked to have a physical examination and imaging studies, provide blood and saliva samples, or provide tumor tissue from prior biopsies or cancer surgeries.\n* Annually, participants will update the family history and individual information questionnaires to document important changes in medical history, and will also update the medical record release form. Participants may be asked to provide additional cheek lining cells and\u002For blood samples, as well as tumor tissue from any new or planned biopsies or tumor surgeries.\n* Treatment will not be provided as part of this protocol.",[26,27,28,29,30],"Pleuropulmonary Blastoma","Cystic Nephroma","Ovarian Sertoli-Leydig Cell Tumors","Ocular Medulloepithelioma","Nasal Chondromesenchymal Hamartoma",[32,33,34,35,26,36],"Thyroid Cancer","Germline DICER1 Mutation","MicroRNA Biogenesis","Natural History","PPB","RECRUITING","2026-06-26",{"date":40,"type":41},"2026-06-29","ACTUAL",{"date":43,"type":41},"2011-02-13",{"name":45,"class":46},"National Cancer Institute (NCI)","NIH",2,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":59,"briefSummary":61,"conditions":62,"keywords":4,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":73},"100565882","phase-3-testing-a-standardized-approach-to-surgery-and-chemotherapy-for-type-i-pleuropulmonary-blastoma-or-the-addition-of-an-anti-cancer-drug-topotecan-to-the-usual-treatment-for-types-ii-and-iii-pleuropulmonary-blastoma-100565882","NCT06647953","Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma","Prospective Treatment of Types I, II and III Pleuropulmonary Blastoma (PPB)","Inclusion Criteria:\n\n* 21 years of age or younger\n* Newly diagnosed PPB. Note that patients with known germline DICER1 variant or mosaicism with a large, solid unresectable thoracic mass with imaging features characteristic for Type II or III PPB are eligible without histologic confirmation of the diagnosis if a biopsy of the mass is not considered safe or feasible\n\n  * Individuals are eligible based on institutional diagnosis of Type I, Ir, II or III PPB diagnosed within 60 days prior to enrollment. Children with Type II or III PPB at risk for clinical decompensation may receive protocol therapy while awaiting rapid central pathology review. Children with Type I or Ir PPB will be assigned to chemotherapy vs. observation based on imaging and central pathology review diagnosis. Type I and Ir patients should not begin chemotherapy prior to return of central pathology results\n* For patients with Type II or III PPB (within 7 days prior to enrollment): A serum creatinine based on age\u002Fsex as follows:\n\n  * Age: 1 month to \\\u003C 6 months - Maximum Serum Creatinine (mg\u002FdL): 0.4 (Male), 0.4 (Female)\n  * Age: 6 months to \\\u003C 1 year - Maximum Serum Creatinine (mg\u002FdL): 0.5 (Male), 0.5 (Female)\n  * Age: 1 to \\\u003C 2 years - Maximum Serum Creatinine (mg\u002FdL): 0.6 (Male), 0.6 (Female)\n  * Age: 2 to \\\u003C 6 years - Maximum Serum Creatinine (mg\u002FdL): 0.8 (Male), 0.8 (Female)\n  * Age: 6 to \\\u003C 10 years - Maximum Serum Creatinine (mg\u002FdL): 1 (Male), 1 (Female)\n  * Age: 10 to \\\u003C 13 years - Maximum Serum Creatinine (mg\u002FdL): 1.2 (Male), 1.2 (Female)\n  * Age: 13 to \\\u003C 16 years - Maximum Serum Creatinine (mg\u002FdL): 1.5 (Male), 1.4 (Female)\n  * Age: ≥ 16 years - Maximum Serum Creatinine (mg\u002FdL): 1.7 (Male), 1.4 (Female) OR - A 24 hour urine creatinine clearance ≥ 60 mL\u002Fmin\u002F1.73 m\\^2 OR - A glomerular filtration rate (GFR) ≥ 60 mL\u002Fmin\u002F1.73 m\\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)\n  * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility\n* For patients with Type II or III PPB (within 7 days prior to enrollment): Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age\n* For patients with Type II or III PPB (within 7 days prior to enrollment): Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \\[ALT\\]) ≤ 135 U\u002FL\n\n  * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U\u002FL\n* Shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of ≥ 50% by radionuclide angiogram (within 21 days prior to start of protocol therapy)\n* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible as long as they are NOT receiving anti-retroviral agents that are strong inhibitors or inducers of CYP3A4\n\nExclusion Criteria:\n\n* Administration of prior PPB-directed chemotherapy is an exclusion criterion. Prior treatment for another malignancy is not an exclusion criterion\n* Patients with known Charcot-Marie-Tooth disease\n* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential\n* Lactating females who plan to breastfeed their infants\n* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation\n* All patients and\u002For their parents or legal guardians must sign a written informed consent\n* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met","21 Years",{"count":57,"type":22},110,"INTERVENTIONAL",[60],"PHASE3","This phase III trial tests how well surgery plus chemotherapy compared to surgery alone works in treating patients with type I pleuropulmonary blastoma (PPB), and tests how well surgery plus standard chemotherapy with the addition of topotecan works compared to surgery plus standard chemotherapy alone in treating patients with type II and III PPB.\n\nHistorically, most children with type I PPB had surgery and approximately 40% of children with type I PPB received chemotherapy following their surgery, usually for 22-42 weeks. There has not been a consistent standard for which children with type I PPB receive chemotherapy after surgery. For patients whose tumor has been removed completely with surgery, observation without chemotherapy may work as well as giving chemotherapy after surgery in preventing a return of the PPB tumor.\n\nThe standard chemotherapy for patients with types II or III PPB in the United States is four cycles of IVADo (ifosfamide, vincristine, dactinomycin, and doxorubicin) followed by 8 cycles of IVA (ifosfamide, vincristine and dactinomycin). Ifosfamide is in a class of medications called alkylating agents. It works by slowing or stopping the growth of tumor cells in the body. Vincristine is in a class of medications called vinca alkaloids. It works by stopping tumor cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy (antineoplastic antibiotic). It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill tumor cells. Doxorubicin is in a class of medications called anthracyclines. Doxorubicin damages the cell's DNA and may kill tumor cells. It also blocks a certain enzyme needed for cell division and DNA repair. Topotecan is in a class of medications called topoisomerase I inhibitors. It works by interfering with tumor cell DNA which kills them. Giving topotecan in addition to standard IVADo and IVA chemotherapy regimens may shrink the cancer as well as or better than the standard therapy or could decrease the chance the tumor spreads while causing fewer side effects.",[26],"2026-05-01",{"date":65,"type":41},"2026-05-05",{"date":67,"type":41},"2025-03-21",{"date":69,"type":22},"2029-03-31",{"name":71,"class":72},"Children's Oncology Group","NETWORK",82,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":17,"minAge":82,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":86,"conditions":87,"keywords":101,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":117},"100598488","an-international-study-on-pediatric-patients-with-rare-tumors-100598488","NCT07072143","An International Study on Pediatric Patients With Rare Tumors.","The PARTNER Study - An International Prospective Observational Study on Pediatric Patients With Very Rare Tumors.","PARTNER","Inclusion Criteria:\n\n* Children and adolescents (age 0-18 years) with a primary or relapsed Very Rare Tumor diagnosed and\u002For treated in a participating country\u002Fcenter.\n* Written informed consent from the patient and\u002For the parent\u002Flegal guardian\n\nExclusion Criteria:\n\n* Absence of Written informed consent from the patient and\u002For the parent\u002Flegal guardian","0 Years","18 Years",{"count":85,"type":22},6250,"The PARTNER study is an international, prospective, observational study of paediatric patients with very rare tumours.",[88,89,90,91,92,93,94,95,96,26,97,98,99,100],"Paraganglioma\u002F Phaeochromocytoma","Melanoma and Other Malignant Neoplasms of Skin","Gastrointestinal Stromal Tumor (GIST)","Adrenocortical Tumor","Pancreatic Tumors","Esthesioneuroblastoma, Olfactory","Mesothelioma","Nasopharyngeal Carcinoma (NPC)","NUT Carcinoma","Salivary Gland Tumors","Thymic Tumors","Thyroid Carcinoma","Appendiceal Cancers",[102,103,104,105,106],"children","rare tumors","very rare tumors","adolescents","PARTNER Study","2025-07-16",{"date":109,"type":41},"2025-07-18",{"date":111,"type":41},"2025-01-25",{"date":113,"type":22},"2049-06-06",{"name":115,"class":116},"Azienda Ospedaliera di Padova","OTHER",1,{"id":119,"slug":120,"hasResults":11,"nctId":121,"briefTitle":122,"officialTitle":123,"acronym":4,"eligibilityCriteria":124,"healthyVolunteers":16,"sex":17,"minAge":125,"maxAge":126,"enrollmentInfo":127,"targetDuration":129,"studyType":23,"phases":4,"briefSummary":130,"conditions":131,"keywords":147,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":166,"lastUpdatePostDateStruct":167,"startDateStruct":169,"completionDateStruct":171,"leadSponsor":173,"locationsCount":117},"100315102","international-ppbdicer1-registry-100315102","NCT03382158","International PPB\u002FDICER1 Registry","International Pleuropulmonary Blastoma\u002FDICER1 Registry (for PPB, DICER1 and Associated Conditions)","Inclusion Criteria:\n\n1. Known or suspected PPB or related thoracic tumor\n2. Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)\n3. Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others\n4. Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition\n5. Informed consent by patient\u002F or parent\u002Fguardian (also, where appropriate: assent and HIPAA consent)\n\nExclusion criteria:\n\nAbsence of appropriate consent for Registry participation","0 Minutes","100 Years",{"count":128,"type":22},3400,"10 Years","Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.",[26,132,133,27,134,135,136,137,30,138,139,140,141,142,143,99,144,145,146],"Sertoli-Leydig Cell Tumor","DICER1 Syndrome","Wilms Tumor","Pineoblastoma","Renal Sarcoma","Nodular Hyperplasia of Thyroid","Ciliary Body Medulloepithelioma","Neuroblastoma","Pituitary Cancer","Embryonal Rhabdomyosarcoma","Ovarian Sarcoma","Gynandroblastoma","Embryonal Rhabdomyosarcoma of Vagina (Diagnosis)","Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)","Embryonal Rhabdomyosarcoma of Cervix",[148,36,149,150,132,27,151,152,153,134,135,136,154,137,155,99,30,156,138,157,139,140,141,158,142,143,159,160,161,162,163,164,165],"pleuropulmonary blastoma","DICER1","SLCT","CN","DICER1 mutation","DICER1 syndrome","ASK","Thyroid Nodules","NCMH","CBME","ERMS","Peritoneal PPB","pPPB","multinodular goiter","PPB Type I","PPB Type II","PPB Type III","PPB Type Ir","2025-01-28",{"date":168,"type":41},"2025-01-30",{"date":170,"type":41},"2016-12-06",{"date":172,"type":22},"2035-12-06",{"name":174,"class":116},"Children's Hospitals and Clinics of Minnesota"]