[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"RECORDATI GROUP\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":153},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,49,79,104,131],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100340160","phase-2-pharmacokinetic-pk-pharmacodynamic-pd-and-tolerability-of-osilodrostat-in-pediatric-patients-with-cushings-syndrome-100340160",false,"NCT03708900","Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome","A Phase II, Multicenter, Open-label, Non-comparative Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Tolerability of Osilodrostat in Children and Adolescent Patients With Cushing's Syndrome","Inclusion criteria\n\n1. Male and female children and adolescents from 2 to \\\u003C 18 years of age with Cushing's syndrome of endogenous origin: Who have failed surgery (or) who are awaiting surgery (or) for whom surgery is not an immediate option. For patients who are awaiting surgery, the study treatment could be less than 12 weeks.\n2. Patients must weigh \\> 10 kg.\n3. The diagnosis of Cushing's syndrome must be confirmed by each of the following:\n\n3a) The clinical criterion of decreasing growth percentiles with increasing weight (as evidenced by the presence of a contrast in height and BMI SD scores, for example a SDS \\\u003C 0 and BMI SDS \\> 0, or a strong clinical suspicion of Cushing's syndrome, such as photographic evidence of a change in facial appearance); 3b) Abnormal low-dose (0.5 mg Q6h x 48 hours, or overnight 15mcg\u002Fkg \\[max 1 mg\\]) dexamethasone suppression test, defined as plasma cortisol levels \\> 1.8 mcg\u002Fdl, at time point 48 hours (0.5 mg Q6h x 48 hours) or 9 to 12 hours (overnight 15mcg\u002Fkg \\[max 1 mg\\]) after the first dose of dexamethasone; (OR) Midnight serum cortisol levels \\> ULN, assessed while the patient is sleeping and after pre-cannulation (OR) two samples of late-night salivary cortisol greater than ULN for the assay. 3c)Two 24-hour urinary free cortisol values \\> 1.3 x ULN;\n\n4\\. Able to swallow study drug tablets (not crushed or split) or the content of the capsules mixed with water.\n\n5\\. Parents or legal guardians able to provide consent\u002Fassent.\n\nExclusion Criteria\n\n1. Patients with macroadenoma complicated by compressive symptoms (requiring urgent surgical intervention) or at high risk for compressive symptoms due to mass effect of tumor (concern of corticotroph tumor progression).\n2. Insufficient washout period from any other medication used to lower cortisol levels (5 half-lives of any drug).\n3. Use of other investigational drugs at the time of enrollment, or within 30 days, or prior to completion of a wash-out duration that is at least 5 half- lives of the drug, at the time of enrollment, whichever is longer. Local regulations may require a longer wash-out period or specify other limitations for participation in an investigational trial, in which case they will be applicable as well.\n4. History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat.\n5. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.\n6. Patients with moderate to severe renal impairment (estimated GFR \\\u003C 60 mL\u002Fmin by the Creatinine-based \"Bedside Schwartz\" equation).\n7. Patients with serum ALT and\u002For AST \\> 3 x ULN, or total bilirubin \\> 1.5 x ULN.\n8. History of thrombosis.\n9. Patients with risk factors for QTc prolongation or Torsade de Pointes, including: 9a) patients with a baseline QTcF \\> 450 ms 9b) personal or family history of long QT syndrome 9c) concomitant medications known to prolong the QT interval 9d) patients with hypokalemia, hypocalcaemia, or hypomagnesaemia, if not corrected before pre-dose Day 0. In case of uncorrected hypokalemia (\\\u003C3.5 mEq\u002FL), the screening period may be used to correct hypokalemia prior to starting study drug. Use of potassium supplements and\u002For mineralocorticoid antagonists is permitted during the study. 9e) Patients with a history of significant cardiovascular disease (based on the opinion of the investigator) such as: structural cardiovascular abnormalities, arrhythmia,\n10. Hypertensive patients with uncontrolled blood pressure defined as SBP \\> 150 and\u002For DBP \\> 100 or not optimally treated for hypertension as judged by the investigator.\n11. Patients who have undergone any major surgery within 1 month.\n12. Patients who have undergone trans-sphenoidal pituitary surgery within 6 weeks prior to screening are not eligible, unless they have clear evidence of persistent hypercortisolism or persistent biochemical changes consistent with Cushing's syndrome.\n13. Use of or anticipated use of systemic glucocorticoid medications 1 month prior to screening.\n14. Uncontrolled hypothyroidism as evidenced by Free T4 \\\u003C 0.8 ng\u002Fdl.\n15. Uncontrolled hyper thyroidism.\n16. Diabetic patients with poorly controlled diabetes as evidenced by HbA1c \\> 8.5 % or not optimally treated for diabetes mellitus as judged by the investigator.\n17. Positive pregnancy test in females of childbearing potential.\n18. Female patients of childbearing potential who do not agree to use highly effective birth control methods .\n19. Pregnant or nursing (lactating) women.\n20. Any medical condition that would, in the investigator's judgment, prevent the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures. Any severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study treatment administration or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for the study.\n21. Use of concomitant prohibited medications","ALL","2 Years","17 Years",{"count":20,"type":21},12,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.",[27],"Cushing Syndrome",[29,30,31,32,33,34,35],"Cushing's syndrome (CS)","LCI699","osilodrostat","Pituitary Gland","Adrenocorticotropic Hormone (ACTH)","Urinary Free Cortisol (UFC)","mean Urinary Free Cortisol (mUFC)","RECRUITING","2026-03-26",{"date":39,"type":40},"2026-03-27","ACTUAL",{"date":42,"type":40},"2021-04-28",{"date":44,"type":21},"2027-07-21",{"name":46,"class":47},"RECORDATI GROUP","INDUSTRY",15,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":22,"phases":60,"briefSummary":62,"conditions":63,"keywords":66,"overallStatus":70,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":4},"100611944","phase-4-osilodrostat-in-patients-with-hypertension-caused-by-hypercortisolaemia-due-to-cushings-syndrome-100611944","NCT07247162","Osilodrostat in Patients With Hypertension Caused by Hypercortisolaemia Due to Cushing's Syndrome","A Double-blind, Randomised, 30-week Placebo-controlled Phase IV Study to Assess the Efficacy and Safety of Osilodrostat in Patients With Hypertension Caused by Hypercortisolaemia Due to Cushing's Syndrome","LINC CARE","Inclusion Criteria:\n\n1. Male or female ≥ 18 years of age\n2. Able to provide and have provided signed written informed consent prior to study participation\n3. Diagnosis of endogenous Cushing's Syndrome\n4. mUFC values from two 24h urinary collections \\> ULN and ≤ 2x ULN\n5. Participants with uncontrolled hypertension on stable doses of BP lowering medications (for at least 4 weeks); qualifying BP measurements by ABPM taken prior to randomisation defined as: Average of 24h ABPM SBP ≥ 135 or DBP ≥ 85 mmHg\n6. Participants under glucocorticoid replacement therapy can be recruited only if this therapy has been already stopped for at least seven days or 5 half-lives prior to screening, whichever was longer\n7. Not taking any drug therapy for CS. The following minimum periods without these medications need to be completed before baseline assessments:\n\n   1. Steroidogenesis inhibitors (e.g. ketoconazole, metyrapone): 1 week\n   2. Mifepristone: 3 weeks\n   3. SC Pasireotide: 1 week\n   4. Pasireotide LAR: 3 months\n   5. Cabergoline: 4 weeks\n8. Able to take oral medication and be willing to comply with the requirements of the study\n\nExclusion Criteria:\n\n1. Previously treated with osilodrostat less than 12 weeks prior to start of screening\n2. Known hypersensitivity to osilodrostat\n3. Presence of any severe and\u002For uncontrolled medical condition or other conditions that could affect participation in the study\n4. Participants who are scheduled for a surgery to treat CS within 32 weeks of randomisation to the study drug\n5. Presence of a known \"long term\" history of both hypertension and diabetes (defined as both hypertension and diabetes diagnosed \\>10 years prior to the initial diagnosis of endogenous CS)\n6. History of cyclic Cushing's Syndrome with fluctuating clinical manifestations\n7. Participants with pseudo-CS\n8. Participants with compression of the optic chiasm due to a macroadenoma or participants at high risk of compression of the optic chiasm (tumour within 2 mm of optic chiasm)\n9. Pituitary radiation therapy within 3 years of screening\n10. Ectopic ACTH syndrome or adrenocortical carcinoma with a life expectancy of \\\u003C3 years or receiving chemotherapy\n11. Having received prior mitotane treatment\n12. Participants who are shift workers or have conditions that can affect the measurement of late night salivary cortisol (LNSC) or the LDDST\n13. Poorly controlled diabetes mellitus with a baseline HbA1c \\> 10.5%\n14. Poorly controlled BP defined as: Average SBP ≥ 170 or average DBP ≥ 110 mmHg as measured by the 24h ABPM\n15. Participants who are hypothyroid and not on adequate replacement therapy\n16. History of major surgery\u002Fsurgical therapy for any cause within 1 month before entering the study.\n17. Presence of bradycardia and\u002For QT-related exclusion criteria\n18. Total bilirubin \\> 1.5 x ULN and ALT or AST \\> 3 x ULN\n19. Participation in any clinical investigation within 4 weeks prior to screening or longer if required by local regulation (Use of an investigational drug within 1 month prior to dosing)\n20. Occurrence of any significant acute illness within the three weeks prior to dosing\u002Frandomisation\n21. Female participants who are pregnant, intending to become pregnant or breastfeed during the study or lactating, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test\n22. Women of childbearing potential (WOCBP) who are unwilling to use highly effective contraception methods\n23. Potentially unreliable or vulnerable participants (e.g. person kept in detention) and those judged by the Investigator to be unsuitable for the study","18 Years",{"count":59,"type":21},63,[61],"PHASE4","Osilodrostat has proven to be a safe and efficacious treatment for patients with CS. Demonstrating normalisation of hypercortisolaemia and in patients with hypertension and\u002For dysglycaemia clinically relevant and statistically significant reductions in blood pressure and glycaemia. This study aims at providing additional evidence on the safety, efficacy and appropriate dosing of osilodrostat in patients with CS, who have hypertension.",[64,65,27],"Hypertension","Hypercortisolemia",[31,67,68,69],"hypertension","hypercolesterolaemia","cushing's syndrome","NOT_YET_RECRUITING","2025-12-03",{"date":73,"type":40},"2025-12-10",{"date":75,"type":21},"2026-08",{"date":77,"type":21},"2028-07",{"name":46,"class":47},{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":4,"eligibilityCriteria":85,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":86,"targetDuration":4,"studyType":88,"phases":4,"briefSummary":89,"conditions":90,"keywords":94,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":96,"lastUpdatePostDateStruct":97,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":5},"100442345","an-observational-study-of-carbaglu-for-the-treatment-of-mma-and-pa-in-adults-and-pediatrics-100442345","NCT05040178","An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics","A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations","Inclusion Criteria:\n\n1. Provision of signed and dated informed consent\u002Fassent form\n2. Prescribed and treated with Carbaglu®\n3. Have an established diagnosis of PA or MMA defined as follows:\n\n   * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR\n   * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).\n\nAND\u002FOR\n\n* Confirmation by molecular genetic testing\n\nExclusion Criteria:\n\n* None",{"count":87,"type":21},20,"OBSERVATIONAL","To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.",[91,92,93],"Hyperammonemia","Methylmalonic Acidemia","Propionic Acidemia",[95],"PA & MMA","2025-04-10",{"date":98,"type":40},"2025-04-15",{"date":100,"type":40},"2022-06-30",{"date":102,"type":21},"2032-06-30",{"name":46,"class":47},{"id":105,"slug":106,"hasResults":11,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":4,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":111,"maxAge":57,"enrollmentInfo":112,"targetDuration":114,"studyType":88,"phases":4,"briefSummary":115,"conditions":116,"keywords":118,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":130},"100381930","a-post-authorisation-safety-study-patient-registry-of-patients-with-neuroblastoma-being-treated-with-dinutuximab-beta-100381930","NCT04253015","A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta","A Post-Authorisation Safety Study Patient Registry of Patients With High-risk Neuroblastoma Being Treated With the Monoclonal Antibody Dinutuximab Beta","Inclusion Criteria:\n\nPatients meeting the following criteria will be considered for inclusion into the registry:\n\n* Patients diagnosed with high-risk neuroblastoma and starting treatment with commercially available dinutuximab beta OR\n* Patients diagnosed with high-risk neuroblastoma and starting treatment with dinutuximab beta in a clinical trial where dinutuximab beta is provided according to the country\u002Fregional marketing authorisation AND\n* Appropriate consent\u002Fassent has been obtained for participation in the registry with a willingness to be followed up for up to 10 years.\n\nExclusion Criteria:\n\nPatient will not be eligible for inclusion if the following criterion applies:\n\n* Patients commencing dinutuximab beta within a clinical trial where the product is being provided outside of the country\u002Fregional marketing authorisation OR\n* Appropriate consent\u002Fassent has not been obtained for participation in the registry or patient\u002Flegal representative is not willing for the patient be followed up for up to 10 years.","1 Year",{"count":113,"type":21},125,"10 Years","This is a non-interventional, multi-national, observational, prospective patient registry to further evaluate the effectiveness and safety of dinutuximab beta - a monoclonal immunoglobulin G 1 (IgG1) antibody, to obtain information on survival, pain severity and incidence of neuro-toxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.",[117],"Neuroblastoma",[119,120,121],"Tumour","Brain tumour","Paediatrics","2024-02-06",{"date":124,"type":40},"2024-02-07",{"date":126,"type":40},"2019-09-30",{"date":128,"type":21},"2032-06-15",{"name":46,"class":47},13,{"id":132,"slug":133,"hasResults":11,"nctId":134,"briefTitle":135,"officialTitle":135,"acronym":136,"eligibilityCriteria":137,"healthyVolunteers":11,"sex":16,"minAge":138,"maxAge":139,"enrollmentInfo":140,"targetDuration":4,"studyType":88,"phases":4,"briefSummary":142,"conditions":143,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"100376054","understanding-the-long-term-management-of-organic-acidemia-patients-with-carbaglu-a-mixed-methods-approach-100376054","NCT04176523","Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach","PROTECT","Inclusion Criteria:\n\n1. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)\n2. Patient initiated treatment with carglumic acid for long-term management of MMA or PA\n3. Patient has been treated with carglumic acid for a minimum of 6 months\n4. Patient (or caregiver) is able to comply with all prospective study procedures\n5. Patient (or caregiver) is able to provide informed consent\n\nExclusion Criteria:\n\n* None","6 Months","99 Years",{"count":141,"type":21},95,"This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \\>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \\>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.",[92,93],"2024-02-02",{"date":146,"type":40},"2024-02-05",{"date":148,"type":40},"2019-01-15",{"date":150,"type":21},"2029-07-30",{"name":46,"class":47},32,""]