[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Novartis Pharmaceuticals\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":671},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,231,0,25,[9,55,88,110,139,162,187,212,239,272,299,328,353,379,410,438,464,496,527,552,576,585,609,633,660],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":16,"eligibilityCriteria":17,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":4,"studyType":24,"phases":25,"briefSummary":28,"conditions":29,"keywords":32,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":43,"lastUpdatePostDateStruct":44,"startDateStruct":47,"completionDateStruct":49,"leadSponsor":51,"locationsCount":54},"100054074","phase-1-rollover-study-for-participants-who-have-been-treated-with-and-are-continuing-to-benefit-from-opnurasib-as-a-single-agent-or-in-combination-with-other-study-treatments-100054074",false,"NCT07468071","Rollover Study for Participants Who Have Been Treated With and Are Continuing to Benefit From Opnurasib as a Single Agent or in Combination With Other Study Treatments","KontRASt-R: An Open-label, Multi-center, Rollover Study for Participants Who Have Been Previously Enrolled Into a Novartis-sponsored Opnurasib (JDQ443) Study and Are Continuing to Benefit From Opnurasib as a Single Agent or in Combination With Other Study Treatments","KontRASt-R","Key Inclusion Criteria:\n\n* Participant is currently enrolled in a pre-defined Novartis-sponsored study and is receiving opnurasib as single agent or in combination with other study treatment.\n* Participant has received at least 6 cycles of opnurasib in a parent study.\n* Participant is currently deriving clinical benefit from the study treatment, as determined by the Investigator.\n* Participants currently receiving treatment in one of the KontRASt studies, including those who have not experienced disease progression or who are continuing treatment beyond initial disease progression, may be eligible to enroll in the rollover study if, in the Investigator's judgment, they are expected to continue deriving clinical benefit from treatment with opnurasib (alone or in combination). Clinical benefit, as assessed by the Investigator, may be reflected by delayed disease progression, improvement in disease-related symptoms, improvement in overall quality of life, or a combination of these factors.\n\nKey Exclusion Criteria:\n\n* Participant has been permanently discontinued from opnurasib in the parent protocol for any reason other than enrollment in the CJDQ443B12105B study.\n* Participant is not willing to comply with the contraception requirements outlined in the exclusion criteria of the parent protocol.\n* Participant currently has unresolved toxicities for which opnurasib dosing has been interrupted in the parent study.\n* Participant has known actionable mutations, such as EGFR-sensitizing mutation, ALK rearrangement, or others, identified during the participation in the parent trial for which there are available treatment options.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","ALL","18 Years","100 Years",{"count":22,"type":23},40,"ESTIMATED","INTERVENTIONAL",[26,27],"PHASE1","PHASE2","The purpose of this study is to allow continued access to opnurasib (JDQ443) to participants who are benefitting from treatment with opnurasib as a single agent or in combination with other study treatments in pre-defined Novartis-sponsored opnurasib studies and to continue to assess safety in these participants.",[30,31],"Locally Advanced or Metastatic KRAS G12C-mutated Non-small Cell Lung Cancer","Advanced Solid Tumors Harboring the KRAS G12C Mutation",[33,34,35,36,37,38,39,40,41],"KRAS G12C","non-small cell lung cancer (NSCLC)","colorectal cancer (CRC)","advanced solid tumors","opnurasib (JDQ443)","TNO155","trametinib (TMT212)","cetuximab","tislelizumab (VDT482)","RECRUITING","2026-07-10",{"date":45,"type":46},"2026-07-13","ACTUAL",{"date":48,"type":46},"2026-06-08",{"date":50,"type":23},"2029-09-17",{"name":52,"class":53},"Novartis Pharmaceuticals","INDUSTRY",1,{"id":56,"slug":57,"hasResults":12,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":61,"eligibilityCriteria":62,"healthyVolunteers":12,"sex":18,"minAge":63,"maxAge":64,"enrollmentInfo":65,"targetDuration":4,"studyType":24,"phases":67,"briefSummary":69,"conditions":70,"keywords":72,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":87},"100053454","phase-3-a-study-to-investigate-the-efficacy-safety-and-tolerability-of-votoplam-in-participants-with-huntingtons-disease-100053454","NCT07326709","A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease","A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease","INVEST-HD","Inclusion Criteria:\n\n* Signed informed consents must be obtained prior to participation in the study\n* Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature\n* Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening.\n* Meets all of the following criteria:\n\n  * UHDRS IS score ≥90\n  * UHDRS TFC score = 13\n  * UHDRS TMS score = 7-25, inclusive\n  * CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) \u002F 6.49\n\nExclusion Criteria:\n\n* History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD\n* Serologic evidence for active viral hepatitis as indicated by:\n\n  * positive anti-HBc IgM\n  * positive anti-HBc IgG confirmed by positive HBsAg and\u002For HBV DNA\n  * positive HCV ab test confirmed by positive HCV RNA\n  * Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result\n* History or current diagnosis of ECG or cardiac abnormalities indicating significant risk of safety for participants such as:\n\n  * Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker\n  * History of familial long QT syndrome or known family history of Torsade de Pointes\n* Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral salpingectomy at least six weeks before taking study treatment. In the case of oophorectomy alone, the reproductive status of the woman needs to have been confirmed by follow-up hormone level assessment.\n\n  o WOCBP are excluded unless they are using highly effective methods of contraception (failure rate \\\u003C 1% per year) while taking study treatment and for 8 months after stopping study treatment.\n* Pregnant or nursing (breastfeeding) women\n\nOther protocol defined inclusion\u002Fexclusion criteria may apply","21 Years","70 Years",{"count":66,"type":23},770,[68],"PHASE3","The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.\n\nHTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.",[71],"Huntington Disease",[73,61,74,75,76,77,78,79],"HD","HTT227","VOTOPLAM","HUNTINGTON DISEASE","UHDRS","mHTT","NfL","2026-07-09",{"date":45,"type":46},{"date":83,"type":46},"2026-03-24",{"date":85,"type":23},"2030-04-30",{"name":52,"class":53},41,{"id":89,"slug":90,"hasResults":12,"nctId":91,"briefTitle":92,"officialTitle":93,"acronym":94,"eligibilityCriteria":95,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":96,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":99,"conditions":100,"keywords":102,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":105,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":109,"locationsCount":54},"100053738","real-world-evaluation-of-patient-outcomes-and-experiences-with-ribociclib-early-adopters-100053738","NCT07243002","Real-world Evaluation of Patient Outcomes and Experiences With Ribociclib Early Adopters.","Real-world Evaluation of Patient Outcomes and Experiences With Ribociclib Early Adopters: A Hybrid Study With Prospective Patient-reported Outcomes and Retrospective Clinical Chart Review.","REPOWER","Inclusion Criteria:\n\n* Males or females.\n* Diagnosed with breast cancer, as defined by the International Classification of Diseases (ICD), 9th or 10th Revision, Clinical Modification (ICD-9-CM 174.xx, 175.xx\u002FICD-10-CMC50.xx).\n* Aged ≥18 years (or local legal age of consent) at the date of initial breast cancer diagnosis.\n* Patients with anatomic staging II and III as determined using American Joint Committee on Cancer (AJCC) Criteria.\n* Have initiated adjuvant therapy with ribociclib or abemaciclib, per the approved local label, in combination with ET (within 14 days prior to enrollment).\n* Have HR+ status, as determined by the closest biomarker test on or before the adjuvant therapy initiation date:\n\n  * Tested positive for estrogen receptor (ER+), or\n  * Tested positive for progesterone receptor (PR+), or\n  * Tested positive for both.\n* Tested negative for HER2 (HER2-) using the closest biomarker test on or before the adjuvant therapy initiation date.\n\nExclusion Criteria:\n\n* Patients with local or distant breast cancer recurrence before the ribociclib\u002Fabemaciclib initiation date.\n* Patients enrolled in clinical trials (receiving treatment with clinical study drugs in any setting, i.e., neoadjuvant, adjuvant, local\u002Fregional, metastatic) during the 12-month baseline period.\n* Patients physically\u002Fmentally incapable of understanding the study requirements or fulfilling data collection instruments and require the support of a legally authorized representative.",{"count":97,"type":23},3000,"OBSERVATIONAL","This non-interventional study (NIS) aims to evaluate effectiveness, persistence, treatment patterns, adverse events (AEs), and patient-reported experience (including adherence, treatment satisfaction, health-related quality of life \\[HRQoL\\], work productivity, and etc.), among HR+\u002FHER2- stage II and III eBC patients treated with ribociclib + ET, and to evaluate AEs and patients-reported experience among HR+\u002FHER2- stage II and III eBC patients treated with abemaciclib + ET, as per local label.",[101],"Breast Cancer",[103,101,104],"ribociclib, abemacicblib","Non interventional study",{"date":45,"type":46},{"date":107,"type":46},"2025-12-15",{"date":85,"type":23},{"name":52,"class":53},{"id":111,"slug":112,"hasResults":12,"nctId":113,"briefTitle":114,"officialTitle":115,"acronym":116,"eligibilityCriteria":117,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":119,"enrollmentInfo":120,"targetDuration":4,"studyType":24,"phases":122,"briefSummary":123,"conditions":124,"keywords":126,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":132,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":138},"100053717","phase-3-study-of-efficacy-and-safety-of-iptacopan-in-participants-with-ic-mpgn-100053717","NCT05755386","Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN","A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Idiopathic Immune-complex-mediated Membranoproliferative Glomerulonephritis (IC-MPGN)","APPARENT","Inclusion Criteria:\n\n* Male and female patients including adults (aged at least 18 years to ≤ 60 years) and adolescents (12 -17 years in non-EU countries at screening and 16-17 years in EU countries at screening).\n* Diagnosis of idiopathic IC-MPGN as confirmed by kidney biopsy within 12 months prior to screening in adults and within 3 years of screening in adolescents (a biopsy report, review and confirmation by the Investigator is required). If such a biopsy is not available in an adult participant, this must be obtained at screening (performed and assessed locally for adults only).\n* Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of renin angiotensin system inhibitors (RASi), e.g an ACEi or ARB for at least 90 days (or as according to local guidelines). The doses of other drugs administered to reduce proteinuria and control the disease including mycophenolic acids (MPAs - mycophenolate mofetil or mycophenolate sodium), corticosteroids, SGLT2 inhibitors and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization\n* UPCR ≥ 1.0 g\u002Fg (≥ 113 mg\u002Fmmol) sampled from the first morning void urine sample at Day -75 and Day -15\n* Estimated GFR (using the chronic kidney disease \\[CKD\\]-EPI formula for adult participants and modified Schwartz formula for adolescents aged 12 to 17 years) or measured GFR ≥ 30 ml\u002Fmin\u002F1.73m2 at screening and Day -15.\n* Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, the vaccine should be given according to local regulations at least 2 weeks prior to the first administration of study treatment. If the study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated in accordance with local standard of care.\n* If not previously vaccinated, or if a booster is required, vaccination against Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to the first study treatment administration.\n\nExclusion Criteria:\n\n* Participants who have undergone cell or solid organ transplantation, including kidney transplantation.\n* Participants diagnosed with secondary IC-MPGN including but not limited to any of the following conditions:\n* Deposition of antigen-antibody immune complexes as a result of any chronic infections, including\n\n  * Hepatitis C virus (HCV) including HCV-associated mixed cryoglobulinemia, hepatitis B virus (HBV);\n  * Bacterial-endocarditis, infected ventriculo-atrial shunt, visceral abscesses, leprosy, meningococcal meningitis; chronic bacterial infections\n  * Protozoa\u002Fother infections- malaria, schistosomiasis, mycoplasma, leishmaniasis, filariasis, histroplasmosis\n\nRenal deposition of immune complexes as a result of a systemic autoimmune disease:\n\n* Systemic lupus erythematosus (SLE)\n* Sjögren syndrome\n* Rheumatoid arthritis\n* Mixed connective tissue disease Deposition of monoclonal immunoglobulins because of a monoclonal gammopathy due to plasma cell or B cell disorders. Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.\n\nFibrillary glomerulonephritis\n\n* Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with kidney biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli on the most recent biopsy.\n* Kidney biopsy showing interstitial fibrosis\u002Ftubular atrophy (IF\u002FTA) of more than 50%.\n* Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration or the presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatment administration.\n* A history of recurrent invasive infections caused by encapsulated organisms, e.g., Neisseria meningitidis and Streptococcus pneumoniae.\n* The use of inhibitors of complement factors (e.g., Factor B, Factor D, complement 3 (C3) inhibitors, anti-Complement 5 (C5) antibodies, C5a receptor antagonists) within 3 months or 5 half-lives prior to the Screening visit.\n* The use of immunosuppressants (except MPAs), cyclophosphamide or systemic corticosteroids at a dose \\>7.5 mg\u002Fday (or equivalent for a similar corticosteroid medication) within 90 days of study drug administration.\n* The use of MPAs is not permitted within 90 days prior to randomization in India, as per the local health authority requirement.\n* Acute post-infectious glomerulonephritis at screening, based upon the opinion of the investigator.\n* Body mass index (BMI) \\>38 kg\u002Fm2 at screening and randomization. Body weight \\\u003C35 kg at screening and randomization","12 Years","60 Years",{"count":121,"type":23},106,[68],"This study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis.",[125],"IC-MPGN",[127,125,128,129,130,131],"LNP023","iptacopan","UPCR","eGFR","proteinuria",{"date":45,"type":46},{"date":134,"type":46},"2023-10-02",{"date":136,"type":23},"2029-05-31",{"name":52,"class":53},131,{"id":140,"slug":141,"hasResults":12,"nctId":142,"briefTitle":143,"officialTitle":144,"acronym":145,"eligibilityCriteria":146,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":119,"enrollmentInfo":147,"targetDuration":4,"studyType":24,"phases":149,"briefSummary":150,"conditions":151,"keywords":153,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":155,"startDateStruct":156,"completionDateStruct":158,"leadSponsor":160,"locationsCount":161},"100053840","phase-3-study-of-efficacy-and-safety-of-iptacopan-in-patients-with-c3-glomerulopathy-100053840","NCT04817618","Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.","A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.","APPEAR-C3G","Inclusion Criteria:\n\n* Male and female participants age ≥ 12 and ≤ 60 years at screening.\n* Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.\n* Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acid, corticosteroids and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.\n* Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.\n* UPCR ≥ 1.0 g\u002Fg sampled from the first morning void urine sample at Day -75 and Day -15.\n* Estimated GFR (using the CKD-EPI formula for ages ≥ 18 years and modified Schwartz formula for ages 12 to 17 years) or measured GFR ≥ 30 ml\u002Fmin\u002F1.73m2 at screening and Day -15.\n* Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to the start of study treatment.\n* If not previously vaccinated or if a booster is required, vaccination against Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to the first study treatment administration. If study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated.\n\nExclusion Criteria:\n\n* Participants who have received any cell or organ transplantation, including a kidney transplantation.\n* Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.\n* Renal biopsy showing interstitial fibrosis\u002Ftubular atrophy (IF\u002FTA) of more than 50%\n* Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.\n* Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration\n* The presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatment administration.\n* A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.\n* The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti C5 antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.\n* The use of immunosuppressants (except mycophenolic acids), cyclophosphamide or systemic corticosteroids at a dose \\>7.5 mg\u002Fday (or equivalent for a similar medication) within 90 days of study drug administration.\n* Acute post-infectious glomerulonephritis at screening based upon the opinion of the investigator.",{"count":148,"type":23},98,[68],"The Primary Completion Date and Study Completion Date have been updated to reflect completion of the adolescent cohort, which has been added to the protocol.\n\nThe study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy.",[152],"C3G",[127,128,152,129,130,131,154],"Quality of life",{"date":45,"type":46},{"date":157,"type":46},"2021-07-28",{"date":159,"type":23},"2027-01-29",{"name":52,"class":53},87,{"id":163,"slug":164,"hasResults":12,"nctId":165,"briefTitle":166,"officialTitle":167,"acronym":168,"eligibilityCriteria":169,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":170,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":172,"conditions":173,"keywords":175,"overallStatus":180,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":181,"startDateStruct":182,"completionDateStruct":184,"leadSponsor":186,"locationsCount":4},"100054117","a-study-of-complement-3-glomerulopathy-c3g-patients-treated-with-iptacopan-through-an-early-access-program-in-spain-100054117","NCT07598448","A Study of Complement 3 Glomerulopathy (C3G) Patients Treated With Iptacopan Through an Early Access Program in Spain","Clinical Experience With Iptacopan in Adult Patients With Complement 3 Glomerulopathy (C3G): A Multicenter, Observational, Retrospective Study of Patients Treated Through an Early Access Program in Spain","ROUTE-C3G","Inclusion criteria:\n\n1. Adult patients (≥18 years).\n2. Biopsy-confirmed C3G.\n3. Patients who have received at least one dose of iptacopan through an EAP from October 2020 to June 2025.\n\nExclusion criteria:\n\n1\\. Patients treated with iptacopan through an EAP for indications other than C3G.",{"count":171,"type":23},35,"This study aims to characterize the patient profiles and the clinical experience of iptacopan treatment in adult patients with C3G treated through an early access program (EAP) in Spain.",[174],"Complement 3 Glomerulopathy",[174,152,176,177,178,179],"Iptacopan","Factor B inhibition","Alternative Complement Pathway","Early Access Program","NOT_YET_RECRUITING",{"date":45,"type":46},{"date":183,"type":23},"2026-08-23",{"date":185,"type":23},"2026-12-15",{"name":52,"class":53},{"id":188,"slug":189,"hasResults":12,"nctId":190,"briefTitle":191,"officialTitle":192,"acronym":4,"eligibilityCriteria":193,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":194,"targetDuration":4,"studyType":24,"phases":196,"briefSummary":197,"conditions":198,"keywords":200,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":206,"startDateStruct":207,"completionDateStruct":209,"leadSponsor":211,"locationsCount":54},"100054216","phase-2-a-phase-2-study-to-investigate-the-efficacy-safety-and-tolerability-of-remibrutinib-lou064-in-adult-patients-with-papulopustular-rosacea-ppr-100054216","NCT07681271","A Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR)","A Multicenter, Randomized, Double-blind, Placebo-controlled Phase 2 Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib (LOU064) in Adult Patients With Papulopustular Rosacea (PPR)","Key Inclusion Criteria:\n\n* Signed informed consent must be obtained prior to participation in the study.\n* Adult ≥18 years with a clinical diagnosis of PPR.\n* Moderate-to-severe disease defined by a modified Investigator's Global Assessment (IGA) score of 3 or 4\n* The presence of 15 - 60 inflammatory (papular\u002Fpustular, max. 2 nodular) facial lesions at screening, with at least 15 lesions present at Day 1 (Baseline).\n* Completed requisite washout of systemic antibiotics (30 days) and other prohibited systemic therapies before randomization.\n* Willingness to refrain from initiating treatments or undergoing procedures that target or impact PPR during the double-blind period, and to use only protocol-allowed products.\n\nKey Exclusion Criteria:\n\n* Presence of more than 2 nodular inflammatory lesions\n* Any active facial dermatoses or skin disease or condition that may interfere with assessment of PPR (e.g., seborrheic dermatitis, perioral dermatitis, acne, acneiform eruptions from biologic medications, steroid-induced dermatitis resembling rosacea or acne).\n* History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes\n* Use of biologics within five half-lives prior to screening or until the expected pharmacodynamic (PD) effect has returned to baseline, whichever is longer; or longer if required by local regulations\n* Use of small molecules and\u002For immunosuppressants that are not corticosteroids within 5 half-lives or within 30 days prior to screening, whichever is longer; or longer if required by local regulations\n* Any use of systemic corticosteroids, systemic antibiotics, or topical treatments (including corticosteroids, antibiotics, ivermectin, azelaic acid, or metronidazole) within 30 days prior to randomization, or any planned use of these agents during the study treatment period.\n* History of live attenuated vaccine within 6 weeks prior to randomization or requirement to receive these vaccinations at any time while on study treatment.\n* Use, planned use, or failure to meet the protocol-defined washout periods for prohibited therapies. In particular, patients with pretreatment with remibrutinib or another BTK-inhibitor within 4 months prior to randomization.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":195,"type":23},80,[27],"This Phase 2 study aims to evaluate whether Bruton's tyrosine kinase (BTK) inhibition with remibrutinib can produce a clinically meaningful reduction in inflammatory lesions in adults with moderate-to-severe papulopustular rosacea, while also assessing safety and tolerability of remibrutinib in this indication.",[199],"Papulopustular Rosacea",[199,201,202,203,204,205],"PPR","Ph2","efficacy","safety","tolerability",{"date":45,"type":46},{"date":208,"type":23},"2026-07-29",{"date":210,"type":23},"2027-12-07",{"name":52,"class":53},{"id":213,"slug":214,"hasResults":12,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":4,"eligibilityCriteria":218,"healthyVolunteers":12,"sex":219,"minAge":19,"maxAge":220,"enrollmentInfo":221,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":223,"conditions":224,"keywords":226,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":232,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":238},"100053660","real-world-experience-with-lutetium-vipivotide-tetraxetan-in-metastatic-castration-resistant-prostate-cancer-100053660","NCT06517719","Real-world Experience With Lutetium Vipivotide Tetraxetan in Metastatic Castration Resistant Prostate Cancer","Real-world Experience With Lutetium (177Lu) Vipivotide Tetraxetan in Metastatic Castration Resistant Prostate Cancer, an Observational, Multicenter, Prospective Cohort Study","Inclusion Criteria:\n\nAll patients must meet the following inclusion criteria during the identification period:\n\n* Adult male patients diagnosed with mCRPC and initiating lutetium (177Lu) vipivotide tetraxetan by treating physician as per local label. After treatment decision enrollment is allowed before date of cycle 1 or within 2 weeks after the date of Cycle 1.\n* ≥ 18 years old at the time of enrollment\n* Written informed consent must be obtained prior to any data collection\n* Willing to participate in Quality of Life post treatment date collection for 1 year\n\nExclusion Criteria:\n\nPatients must not meet the following exclusion criterion during the identification period:\n\n\\- Simultaneous participation in any investigational trial or simultaneous participation in another Novartis-sponsored non-interventional study with lutetium (177Lu) vipivotide tetraxetan","MALE","99 Years",{"count":222,"type":23},500,"The purpose of this study is to describe routine clinical practice with lutetium (177Lu) vipivotide tetraxetan on Health related quality of life (HRQoL) at baseline, on treatment, and post progression.",[225],"Metastatic Castration Resistant Prostate Cancer",[227,228,229,230,231],"metastatic castration resistant prostate cancer","mCRPC","Prostate cancer","lutetium vipivotide tetraxetan","177Lu",{"date":45,"type":46},{"date":234,"type":46},"2024-09-04",{"date":236,"type":23},"2028-02-01",{"name":52,"class":53},38,{"id":240,"slug":241,"hasResults":12,"nctId":242,"briefTitle":243,"officialTitle":244,"acronym":245,"eligibilityCriteria":246,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":247,"enrollmentInfo":248,"targetDuration":4,"studyType":24,"phases":250,"briefSummary":251,"conditions":252,"keywords":254,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":265,"startDateStruct":266,"completionDateStruct":268,"leadSponsor":270,"locationsCount":271},"100053289","phase-3-a-study-to-evaluate-the-efficacy-and-safety-of-remibrutinib-in-secondary-progressive-multiple-sclerosis-100053289","NCT07225504","A Study to Evaluate the Efficacy and Safety of Remibrutinib in Secondary Progressive Multiple Sclerosis","A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy and Safety of Remibrutinib in Patients With Secondary Progressive Multiple Sclerosis","REMASTER","Inclusion Criteria:\n\n* Signed informed consent must be obtained prior to any assessment performed.\n* Male or female participants aged 18-65 (inclusive) at Screening.\n* Diagnosis of MS according to the 2017 revised McDonald criteria (Thompson et al 2018), with current SPMS in accordance with the revised clinical course (Lublin et al 2014) at Screening\n* Absence of documented clinical relapses in the 24 months before Screening and randomization.\n* EDSS score of 3.0 to 6.0 (inclusive) at Screening.\n* Documented evidence of disability progression in the 12 months before Screening.\n\nExclusion Criteria:\n\n* Unwilling or unable to undergo MRI scans as per protocol (for example, claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator)).\n* History of clinically significant central nervous system (CNS) disease (e.g. stroke, traumatic brain or spinal injury, history or presence of myelopathy) or neurological disorders which may mimic multiple sclerosis (MS).\n* Ongoing substance abuse (drug or alcohol) or any other factor (e.g. serious psychiatric condition) that may interfere with the participant's ability to cooperate and comply with the study procedures.\n* Participants with history of confirmed Progressive Multifocal Leukoencephalopathy (PML) or neurological symptoms consistent with PML.\n* Women of childbearing potential (WOCBP), unless they are using highly effective methods of contraception (failure rate \\\u003C 1% per year) while taking study treatment and for at least 1 week after stopping study treatment.\n* Significant bleeding risk or coagulation disorders, at Screening.\n* Use of exclusionary medication prior to Screening\u002Frandomization as listed in the protocol.\n\nOther protocol-defined inclusion\u002Fexclusion critria may apply","65 Years",{"count":249,"type":23},1275,[68],"The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)",[253],"Secondary Progressive Multiple Sclerosis (SPMS)",[255,256,257,258,259,260,261,262,263,264],"MS","Multiple sclerosis","secondary progressive multiple sclerosis","SPMS","remibrutinib","LOU064","adult","Expanded Disability Status Scale","EDSS","McDonald diagnostic criteria",{"date":45,"type":46},{"date":267,"type":46},"2025-11-11",{"date":269,"type":23},"2034-01-02",{"name":52,"class":53},236,{"id":273,"slug":274,"hasResults":12,"nctId":275,"briefTitle":276,"officialTitle":277,"acronym":4,"eligibilityCriteria":278,"healthyVolunteers":12,"sex":18,"minAge":279,"maxAge":280,"enrollmentInfo":281,"targetDuration":4,"studyType":24,"phases":283,"briefSummary":284,"conditions":285,"keywords":287,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":292,"startDateStruct":293,"completionDateStruct":295,"leadSponsor":297,"locationsCount":298},"100054316","phase-3-a-study-to-evaluate-the-efficacy-and-safety-of-qmf149-indacaterol-acetatemometasone-furoate-versus-budesonide-in-children-from-6-to-less-than-12-years-of-age-with-asthma-100054316","NCT05562466","A Study to Evaluate the Efficacy and Safety of QMF149 (Indacaterol Acetate\u002FMometasone Furoate) Versus Budesonide in Children From 6 to Less Than 12 Years of Age With Asthma","Double-blind, Randomized, Active-controlled, Two-way Cross-over Study, With 12-week Treatment Duration Per Period, to Evaluate the Efficacy and Safety of QMF149 (Indacaterol Acetate \u002F Mometasone Furoate) Compared to Budesonide in Children From 6 to Less Than 12 Years of Age With Asthma","Inclusion Criteria\n\n1. Male or female children ≥ 6 years and \\\u003C12 years in age at randomization.\n2. Parents\u002Flegal guardian must be willing and able to attend study visits and assist the child with the procedures outlined in the protocol (e.g. compliance with taking study medication and completing the diary) ((≥ 70% during the last 14 days of the Run-in period)).\n3. Confirmed\u002Fdocumented diagnosis of asthma, as defined by national or international asthma guidelines for at least 12 months prior to study enrollment.\n4. Written and signed informed consent by parent(s)\u002Flegal guardian(s) for the pediatric patient and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed.\n5. Patient receiving daily treatment of stable low dose ICS alone (i.e. up to 100ug daily dose of fluticasone propionate DPI or equivalent) without additional controller OR low dose ICS (up to 100ug daily dose of fluticasone propionate DPI or equivalent) with one additional controller prior to starting run-in and eligible after run-in on mono ICS alone (fluticasone 100ug\u002Fday) for at least 3 weeks (run-in period) prior to randomization.\n6. All patients must be symptomatic at randomization (Visit 30), as defined by ACQ-IA≥1.5. Patients previously on low dose ICS may be included for run-in only if ACQ-IA score ≥1.5 at Visit 20 and will be randomized if ACQ-IA score ≥1.5 at Visit 30.\n\n   Patients previously on low dose ICS with one controller may do the wash out of the controller before the start of run-in and be included for run-in only if ACQ-IA score ≥ 1 and \\\u003C1.5 at Visit 20 and will be randomized if ACQ-IA score ≥1.5 at Visit 30.\n7. Pre-Bronchodilator FEV1 ≥50% of predicted normal at start of Run-in (Visit 20) and end of Run-in (Visit 30).\n\n   Withholding period of bronchodilators prior to spirometry at all time:\n\n   SABA for ≥ 6 hours. For loose combinations of ICS\u002FLABA\\* a wash-out of ≥ 48 hours before Visit 20 is required (14 days for once daily combinations, i.e. indacaterol), short acting anticholinergic (SAMA) for ≥ 8 hours and xanthines ≥7 days.\n\n   \\* In case of combination ICS\u002FLABA at screening, ICS alone should be continued. Wash-out period of each drug should be adhered to as above and should not be longer. If wash-out period is considered to be longer, please contact the Novartis Medical Monitor.\n\n   A one-time repeat of percent predicted FEV1 (pre-bronchodilator FEV1) within 5 days of the Visit is allowed at Visit 20 as well as Visit 30. That would provide sufficient time to receive confirmation from the spirometry data central reviewer of the validity of the assessment. At Visit 20, the Run-in medication should be dispensed only once the repeat spirometry was qualified, and if all inclusion criteria at Visit 20 are successfully met.\n\n   If patient fails to meet the pre FEV1 criteria for technical reasons, a rescreen is allowed once and in this circumstance, patients are not required to go back on prior medication (low dose ICS with or without controller) for the full 4 weeks duration and the rescreen can be scheduled at site's convenience. In this case all assessments must be done according to protocol's requirements.\n8. FEV1 bronchodilator responsiveness testing using up to 4 puffs of SABA (up to 400μg salbutamol or 360μg albuterol) at Run-in Visit (Visit 20): increase \\> and\u002For = 12% (performed according to ATS\u002FERS 2019 guidelines). All patients must perform a bronchodilator responsiveness test at start of Run-in. If responsiveness is not demonstrated at Run-in, it may be repeated once on the same day. If responsiveness is still not demonstrated after repeat, documentation of historical reversibility is accepted. If not available patients must be screen failed. Spacers may be used for bronchodilator responsiveness testing.\n9. Demonstrate acceptable inhaler use technique with Breezhaler® at randomization, as well as acceptable use of other study devices and be able to complete spirometry procedures.\n10. A parent\u002Flegal guardian is to complete all e-Diary entries and attend all clinic visits with the patient. It is recommended, if possible, to have the same parent\u002Flegal guardian to complete the e-diary entries and attend clinic visits with the patient.\n11. Have a documented negative COVID-19 test (validated PCR or antigenic test)) within 3 days prior to randomization visit.\n12. For optional Pharmacokinetics (PK) analysis: Participants willing to participate in the optional PK analysis will need to weigh at least 25 kg at screening.\n\nExclusion Criteria Participants meeting any of the following criteria are not eligible for inclusion in this study.\n\n1. Prior intubation for asthma.\n2. Patients who have had a severe asthma exacerbation requiring in the previous month either systemic steroids or hospitalization due to asthma (\\>24h) or emergency room visit (≤24 hours).\n3. Subjects receiving any medications in the classes specified in Table 6 6 unless they undergo the required washout period prior to Treatment Visit (Day 1) and follow the adjustment through the treatment period.\n4. Use of other investigational drugs within 5 half-lives of enrollment, or within 30 days, whichever is longer.\n5. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years prior to screening, regardless of whether there is evidence of local recurrence or metastases.\n6. History or presence of impaired renal function as indicated by clinically significant abnormal creatinine or blood urea nitrogen (BUN) and\u002For urea values, or abnormal urinary constituents (e.g. albuminuria) according to investigator's judgement.\n\n   * Evidence of urinary obstruction, or difficulty in voiding\n   * Evidence of congenital renal abnormalities with an established effect on renal function\n   * Calculated eGFR \\\u003C60 mL\u002Fmin\u002F1.73m2 using the Bedside Schwartz formula.\n7. Patients who have had a respiratory tract infection as determined by the investigator within 4 weeks prior to Visit 1, or between Visit 1 and Visit 30.\n\n   Patients may be re-screened once, 4 weeks after recovery from their respiratory tract infection.\n8. Any chronic condition of the respiratory tract which in the opinion of the investigator may interfere with study evaluation or optimal participation in the study.\n9. Patient with evidence upon visual inspection (laboratory culture not required) of clinically significant (upon the opinion of the investigator) oropharyngeal candidiasis at Visit 30 or earlier, with or without treatment, Patients may be rescreened once their candidiasis has been treated and has resolved.\n10. History of chronic lung disease other than asthma such as and not limited to, sarcoidosis interstitial lung disease, cystic fibrosis, mycobacterial or other infection (including active tuberculosis or atypical mycobacterial disease), chronic obstructive pulmonary disease (COPD) and asthma\u002FCOPD overlap syndrome (ACOS).\n11. Patients with a history of long QT syndrome or whose corrected QT interval (QTc) measured at start of Run-in or Baseline (Fridericia method) is prolonged (≥ 450 msec for boys and girls) and confirmed by a central assessor (these patients should not be rescreened).\n12. Subjects who have a clinically significant ECG abnormality reported before Visit 30 (End of Run-in).\n13. Subjects who have a clinically significant abnormal laboratory values as per investigator judgement or abnormal liver chemistry results (i.e. ALT, AST, total bilirubin, alkaline phosphatase, GGT and albumin above the upper limit of normal) reported before Visit 30 (End of Run-in).\n14. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the subject in case of participation in the study.\n15. Subjects who, in the opinion of the investigator, are not able to be compliant with study treatment or who have any medical or mental disorder, situation, or diagnosis which could interfere with the proper completion of the protocol requirements or risk the subject's safety while participating in the study.\n16. Subject is an immediate family member of the participating investigator, sub-investigator, study coordinator, or employee of the participating investigator.\n17. Patients who have been treated with long-acting theophylline preparations within four weeks prior to Screening and\u002For during the screening period or who have been treated with short-acting theophylline preparations within two weeks prior to Screening.\n18. Patients who have been treated with non-approved and according to international guidelines not recommended experimental drugs for routine asthma therapy within four weeks prior to Visit 1 and\u002For during the screening period.\n19. Use of Long-Acting Muscarinic Antagonist (LAMA) as maintenance treatment within 3 months prior to Screening.\n20. Evidence of unstable disease within 4 weeks prior to Screening (Visit 1) that in the opinion of the investigator would put the safety of the subject at risk through study participation or would confound the interpretation of the results if the condition\u002Fdisease exacerbated during the study.\n21. History of hypersensitivity to any ingredients of the study drugs including fluticasone propionate, indacaterol acetate, mometasone furoate, budesonide and salmeterol\u002Falbuterol or drug of similar chemical classes. This includes any known hypersensitivity or intolerance to the excipients, including lactose.\n22. Patients with Type I diabetes or uncontrolled Type II diabetes either by HBA1c\\>8 or as per judgement of investigator prior to End of Run-In (Visit 30)\n23. Patients receiving any asthma-related or non asthma-related prohibited medications as specified in the protocol.\n24. Immunotherapy or desensitization for allergies started within 3 months prior to Visit 20, or where the maintenance dose is expected to change during the study.\n25. Female patients of childbearing potential defined as all females physiologically capable of becoming pregnant (including female pediatric patients who are menarchal or who become menarchal during the study)) who do not agree to abstinence or, if sexually active, do not agree to the use of contraception as defined in the exclusion criteria.\n\nEffective contraception methods include:\n\n* Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception\n* Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical\u002Fvault caps). For UK: with spermicidal foam\u002Fgel\u002Ffilm\u002Fcream\u002F vaginal suppository\n* Use of oral, (estrogen and progesterone), injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate \\\u003C1%), for example hormone vaginal ring or transdermal hormone contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) If using oral contraception females should have been stable on the same pill for a minimum of 3 months before taking investigational drug. The decision on the contraceptive method should be reviewed at least every 3 months to evaluate the individual need and compatibility of the method chosen.","6 Years","11 Years",{"count":282,"type":23},200,[68],"The purpose of this study is to evaluate the superiority in terms of efficacy and evaluate the safety of QMF149 (indacaterol (acetate) \u002F mometasone (furoate)) compared to budesonide in children from 6 to less than 12 years of age with asthma.\n\n* The study duration will be up to 37 weeks including an investigational treatment duration of 12 weeks and a comparator treatment duration of 12 weeks.\n* The visit frequency will be 3 weeks for screening, run-in and wash-out period, 6 weeks interval for visits during each treatment period, 30 days for safety follow-up.",[286],"Asthma",[286,288,289,290,291],"Pediatric","Breezhaler","QMF149","Budesonide",{"date":45,"type":46},{"date":294,"type":46},"2023-05-11",{"date":296,"type":23},"2028-05-30",{"name":52,"class":53},63,{"id":300,"slug":301,"hasResults":12,"nctId":302,"briefTitle":303,"officialTitle":304,"acronym":4,"eligibilityCriteria":305,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":306,"targetDuration":4,"studyType":24,"phases":308,"briefSummary":309,"conditions":310,"keywords":314,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":321,"startDateStruct":322,"completionDateStruct":324,"leadSponsor":326,"locationsCount":327},"100054172","phase-1-study-of-eci830-single-agent-or-in-combination-in-patients-with-advanced-hrher2--breast-cancer-and-in-patients-with-other-advanced-solid-tumors-100054172","NCT06726148","Study of ECI830 Single Agent or in Combination in Patients With Advanced HR+\u002FHER2- Breast Cancer and in Patients With Other Advanced Solid Tumors","An Open-label, Multi-center, Phase I\u002FII Study of ECI830 as a Single Agent and in Combination With Ribociclib and Endocrine Therapy in Patients With Advanced Hormone Receptor Positive, HER2-negative Breast Cancer and Advanced Solid Tumors","Inclusion Criteria:\n\nAge ≥ 18 years old.\n\nPatients with one of the following indications:\n\nPhase I:\n\nHR+\u002FHER2- aBC with disease progression on or following at least one line of hormone-based therapy in combination with a CDK4\u002F6i and at least one additional line of systemic therapy for metastatic disease.\n\nHistologically and\u002For cytologically confirmed diagnosis of locally advanced or metastatic cancer with a CCNE1 amplification. For dose expansion only: no more than 3 prior lines of therapy for advanced or metastatic disease.\n\nPatients with ES-SCLC and disease progression on or after standard of care (SoC). For dose expansion only: no more than 2 prior lines of therapy for advanced or metastatic disease are allowed.\n\nPhase II:\n\nHR+\u002FHER2- aBC with disease progression on an aromatase inhibitor or tamoxifen in combination with a CDK4\u002F6 inhibitor for unresectable\u002Fmetastatic disease with no more than 2 lines of endocrine therapy.\n\nMeasurable disease as determined by RECIST v1.1.\n\nBC only: If no measurable disease is present, then at least one predominantly lytic bone lesion must be present that can be accurately assessed at baseline and is suitable for repeated assessment.\n\nExclusion Criteria:\n\nPrevious treatment with a CDK2 inhibitor at any time.\n\nPatients with inadequate bone marrow and\u002For organ functions with out-of-range laboratory values.\n\nClinically significant, uncontrolled heart disease and\u002For cardiac repolarization abnormality including MI, CABG, long QT syndrome, or risk factors for TdP.\n\nPresence of symptomatic CNS metastases or CNS metastases that require local therapy or increasing doses of corticosteroids within 2 weeks prior to study entry.\n\nFor the combination treatment:\n\nPatients with symptomatic visceral disease or any disease burden that makes the patient ineligible for endocrine-based therapy.\n\nPatients who could not tolerate the prescribed dose of ribociclib during a previous course of treatment, requiring dose reduction or permanent discontinuation due to adverse events.\n\nFor patients with BC: Patient is concurrently using hormone replacement therapy.\n\nWOCBP who are unwilling to use highly effective contraception methods, pregnant or nursing women.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":307,"type":23},280,[26,27],"Phase I: Characterize safety and tolerability of ECI830 as a single agent and in combination with ribociclib and fulvestrant. Identify dose range for optimization\u002Frecommended dose for future studies.\n\nPhase II: Assess the anti-tumor activity of ECI830 in combination with ribociclib and fulvestrant in patients with hormone receptor-positive\u002Fhuman epidermal growth factor receptor 2-negative (HR+\u002FHER2-) advanced breast cancer.",[311,312,313],"Advanced HR+\u002FHER2- Breast Cancer","Advanced CCNE1-amplified Solid Tumors","Extensive-stage Small Cell Lung Cancer",[315,316,317,318,319,320],"ECI830","Ribociclib","Fulvestrant","Breast cancer","CCNE1 amplification","Small cell lung cancer",{"date":45,"type":46},{"date":323,"type":46},"2025-04-03",{"date":325,"type":23},"2028-09-25",{"name":52,"class":53},33,{"id":329,"slug":330,"hasResults":12,"nctId":331,"briefTitle":332,"officialTitle":333,"acronym":4,"eligibilityCriteria":334,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":335,"targetDuration":4,"studyType":24,"phases":337,"briefSummary":338,"conditions":339,"keywords":341,"overallStatus":180,"whyStopped":4,"lastUpdateSubmitDate":346,"lastUpdatePostDateStruct":347,"startDateStruct":348,"completionDateStruct":350,"leadSponsor":352,"locationsCount":4},"100054294","phase-3-a-study-to-evaluate-efficacy-safety-and-tolerability-of-remibrutinib-in-adult-participants-with-severe-chronic-pruritus-of-unknown-origin-cpuo-100054294","NCT07700056","A Study to Evaluate Efficacy, Safety and Tolerability of Remibrutinib in Adult Participants With Severe Chronic Pruritus of Unknown Origin (CPUO)","A 52-week Multi-center, Randomized, Double-blind, Placebo Controlled Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Remibrutinib in Adult Participants With Severe Chronic Pruritus of Unknown Origin (CPUO)","Key Inclusion Criteria:\n\n* Participant must be ≥ 18 years of age, at the time of signing the informed consent.\n* Participants with chronic pruritus for at least 4 months prior to screening.\n* Chronic pruritus considered of unknown origin as assessed by the investigator at baseline (e.g., excluding chronic pruritus related to primary dermatological or systemic conditions, neuropathic or psychogenic origin or secondary to drugs or other allergen exposures).\n* Chronic pruritus must affect at least 2 of the following body areas: trunk, arms, or legs (cannot be unilateral and\u002For dermatomal in distribution).\n* Participants with ongoing, severe chronic pruritus despite the use of emollients and who are candidates for systemic therapy.\n* Participants must have severe itch defined by a WI-NRS ≥7 at screening; score scale ranges from 0 to 10; higher score indicates worse itch.\n* Participants must have an average WI-NRS ≥7 over the 7 days prior to randomization\u002Fbaseline visit.\n* The average WI-NRS score over the preceding 7 days prior to the randomization\u002Fbaseline visit will be calculated based on the daily WI-NRS scores (0-10).\n* Participants must have PGIS of pruritus scored as 3 \"severe\" at screening and baseline visits.\n\nKey Exclusion Criteria:\n\n* Any active skin conditions (e.g., atopic dermatitis, psoriasis, etc.) that may interfere with the assessment of CPUO.\n* Known systemic condition(s) or medication(s) that are considered by the investigator to be the primary cause of current pruritus.\n* Known or suspected infectious disease that is active, chronic or recurrent which precludes the participant from participating in the clinical trial as per Investigator´s assessment. These infectious diseases include but are not limited to opportunistic infections (e.g., tuberculosis, atypical mycobacterioses, listeriosis or aspergillosis) and\u002For known or suspected HIV infection.\n* History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.\n* Significant bleeding risk or coagulation disorders. History of gastrointestinal bleeding, e.g., in association with use of nonsteroidal anti-inflammatory drugs (NSAIDs), that was clinically relevant (e.g., where intervention was indicated or requiring hospitalization or blood transfusion). Requirement for anti-platelet medication, except for acetylsalicylic acid up to 100 mg\u002Fd or clopidogrel up to 75 mg\u002Fd. The use of dual anti-platelet therapy (e.g., acetylsalicylic acid + clopidogrel) is prohibited.\n* History or current hepatic disease, including but not limited to, acute or chronic hepatitis, cirrhosis or hepatic failure or aspartate aminotransferase (AST)\u002F alanine aminotransferase (ALT) levels of more than 1.5 x upper limit of normal (ULN) or International Normalized Ratio (INR) of more than 1.5 at screening.",{"count":336,"type":23},180,[68],"The purpose of this phase 3 study is to establish the efficacy, safety and tolerability of remibrutinib in adult participants with severe chronic pruritus of unknown origin (CPUO).",[340],"Chronic Pruritus of Unknown Origin",[342,343,344,345,259],"BTK inhibitor","chronic pruritus","chronic pruritus of unknown origin","CPUO","2026-07-07",{"date":45,"type":46},{"date":349,"type":23},"2026-08-14",{"date":351,"type":23},"2030-04-15",{"name":52,"class":53},{"id":354,"slug":355,"hasResults":12,"nctId":356,"briefTitle":357,"officialTitle":358,"acronym":359,"eligibilityCriteria":360,"healthyVolunteers":12,"sex":219,"minAge":19,"maxAge":4,"enrollmentInfo":361,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":363,"conditions":364,"keywords":367,"overallStatus":180,"whyStopped":4,"lastUpdateSubmitDate":372,"lastUpdatePostDateStruct":373,"startDateStruct":374,"completionDateStruct":376,"leadSponsor":378,"locationsCount":4},"100053259","pluvicto-real-world-investigation-in-survival-in-metastatic-crpc-100053259","NCT07697989","Pluvicto Real-world Investigation in Survival in Metastatic CRPC","Real World Clinical Effectiveness of [177Lu]Lu-PSMA-617 in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients: A Non-Interventional Study","PRISM","Inclusion criteria:\n\n1. Patients with at least one inpatient OR two outpatient primary prostate cancer (PC) diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification \\[ICD-10-CM\\]: C61) during the identification period. For outpatient diagnoses, the second confirmatory PC diagnosis must be at least 30 to 365 days after the first primary diagnosis date.\n2. Patients with a metastatic diagnosis (ICD-10-CM: C77-C79) on or after the primary PC diagnosis date. The earliest metastatic diagnosis will be the patient's metastatic diagnosis date.\n3. Patients with an mCRPC diagnosis on or after the metastatic diagnosis or satisfying any of the proxy criteria.\n4. Patients with evidence of treatment with \\[177Lu\\]Lu-PSMA-617 after the mCRPC diagnosis date. The date of \\[177Lu\\]Lu-PSMA-617 administration will be considered as the index date.\n5. Patients ≥18 years of age on metastatic diagnosis date.\n6. Patients who are male.\n7. Patients with at least 12 months pre-index and at least six months post-index (unless patient died) of medical history or continuous medical and pharmacy enrollment or activity (three-month allowable gap).\n\nExclusion criteria:\n\n1. Patients with other non-prostate primary cancer (≥ two ICD codes for one specific type of cancer in the baseline period at least 30 days apart) within three years prior to the first PC diagnosis.\n2. Patients enrolled in a current clinical trial\u002Finvestigational study within the 30-day period immediately prior to and including the index date or within five half-lives of the investigational product (whichever is longer) or during post-index period (ICD-10-CM: Z00.6).\n3. Patients with missing age and gender information.",{"count":362,"type":23},1085,"This study aims to evaluate the various aspects of treatment effectiveness of \\[177Lu\\]Lu-PSMA-617 (Pluvicto) in mCRPC patients in both pre- and post-taxane settings. The study will be conducted using real-world data sources from the United States (US) and Germany.",[365,366],"Prostatic Neoplasms, Castration-Resistant","Neoplasm Metastasis",[368,369,225,370,371],"Pluvicto","177Lu-PSMA-617","Effectiveness","Survival","2026-07-06",{"date":45,"type":46},{"date":375,"type":23},"2026-06-30",{"date":377,"type":23},"2026-10-31",{"name":52,"class":53},{"id":380,"slug":381,"hasResults":12,"nctId":382,"briefTitle":383,"officialTitle":384,"acronym":385,"eligibilityCriteria":386,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":387,"targetDuration":4,"studyType":24,"phases":389,"briefSummary":390,"conditions":391,"keywords":394,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":402,"startDateStruct":404,"completionDateStruct":406,"leadSponsor":408,"locationsCount":409},"100625325","phase-2-a-study-to-assess-the-tolerability-of-ianalumab-vay736-with-investigators-choice-thrombopoietin-receptor-agonist-ic-tpo-ra-in-patients-with-primary-immune-thrombocytopenia-itp-100625325","NCT07421167","A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)","A Phase 2 Open-label Study to Evaluate the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least One Treatment (VAY2EXPLORE)","VAY2EXPLORE","Key Inclusion Criteria:\n\n* Signed informed consent must be obtained prior to participation in the study.\n* Patients aged 18 years and older on the day of signing the informed consent.\n* ITP cohort only: Confirmed diagnosis of primary ITP that has previously responded to corticosteroid treatment or IVIG treatment but the response was not sustained (response is defined as a platelet count ≥ 50 G\u002FL).\n* ITP cohort only: Received at least one prior treatment for ITP.\n* ITP cohort only: Patients with a platelet count \\\u003C 100 G\u002FL who are receiving a TPO-RA. Patients may already be receiving a TPO-RA or may start a TPO-RA at the time of screening. All patients should be on a stable dose of TPO-RA for at least 14 days prior to first dose of ianalumab. Note: during the screening period, a documented assessment of platelets \\\u003C 100 G\u002FL is mandatory for enrollment. For patients who received rescue medication before screening, platelet count results obtained prior to the start of the rescue therapy should be used to assess eligibility if collected within 14 days prior to screening.\n* ES cohort only: Patients with clinical diagnosis of primary ES with active thrombocytopenia (\\\u003C 100 G\u002FL) with warm autoimmune hemolytic anemia (wAIHA) for whom a TPO-RA is appropriate per Investigator.\n* ES cohort only: Inadequate response to or relapse after treatment with corticosteroid therapy.\n* ES cohort only: Diagnosis confirmed by current or past positive direct antiglobulin test (DAT) (IgG+, with or without C3+) and evidence of hemolysis.\n* ES cohort only: any supportive care treatment administered for wAIHA must be stable for at least 4 weeks prior to enrollment.\n\nKey Exclusion Criteria:\n\n* Patients being treated with TPO-RA for \\> 6 months.\n* Current life-threatening bleeding (related to thrombocytopenia).\n* Prior splenectomy within 6 months of first administration of ianalumab.\n* Patients with the following laboratory abnormalities:\n\n  * Neutrophils: \\\u003C 1000\u002Fmm3\n  * Serum creatinine \\> 1.5 × upper limit of normal (ULN)\n  * Aspartate aminotransferase (AST) \\> 3.0 × ULN\n  * Alanine aminotransferase (ALT) \\> 3.0 × ULN\n  * Immunoglobulin G (IgG) \\\u003C 5 g\u002FL\n  * ITP cohort only: hemoglobin \\\u003C 10 g\u002FL, total bilirubin \\> 1.5 × ULN\n* Patients with significantly compromised liver disease (Child-Pugh 7 to 9) and decompensated liver disease (Child-Pugh 10 to 15).\n* Treatment with a B-cell depleting therapy (e.g. rituximab or anti-B cell Activating Factor (e.g. belimumab) within 12 weeks prior to the first administration of ianalumab. Patients who are refractory to rituximab will be excluded from this trial, where refractory is defined as:\n\n  \\~ Patients who have not achieved a response (defined as platelet count ≥ 30 G\u002FL and at least doubling from baseline within 12 weeks in the absence of rescue therapy) following completion of a standard course of rituximab\n* History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes.\n* Known history of primary or secondary immunodeficiency, or a positive human immunodeficiency virus (HIV) enzyme-linked immunosorbent assay (ELISA) and Western blot) test result.\n* Patients exposed to more than 4 prior treatments for ITP.\n* ITP cohort only: Diagnosis of secondary thrombocytopenia.\n* ITP cohort: Use of immunosuppressant drugs other than corticosteroids or rituximab.\n* ES cohort only: Diagnosis of secondary ES.\n* ES cohort only: Life-threatening hemolysis.\n* ES cohort only: patients with autoimmune hemolytic anemia other than wAIHA",{"count":388,"type":23},164,[27],"The purpose of this study is to investigate the tolerability of ianalumab (9 mg\u002Fkg) with investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in participants diagnosed with primary immune thrombocytopenia (ITP) who have been treated with at least one but no more than four prior treatments, and with no change in IC TPO-RA dose in at least the last 14 days prior to the start of ianalumab.",[392,393],"Primary Immune Thrombocytopenia (ITP)","Primary Evans Syndrome (ES)",[395,396,397,398,399,400],"Immune thrombocytopenia (ITP)","Evans syndrome (ES)","Ianalumab","VAY736","B-cell depletion","B-cell Activating Factor Receptor (BAFF-R)","2026-07-01",{"date":403,"type":46},"2026-07-02",{"date":405,"type":23},"2026-06-29",{"date":407,"type":23},"2030-08-08",{"name":52,"class":53},4,{"id":411,"slug":412,"hasResults":12,"nctId":413,"briefTitle":414,"officialTitle":415,"acronym":416,"eligibilityCriteria":417,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":418,"targetDuration":4,"studyType":24,"phases":420,"briefSummary":421,"conditions":422,"keywords":426,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":431,"startDateStruct":432,"completionDateStruct":434,"leadSponsor":436,"locationsCount":437},"100620446","phase-3-a-phase-3-study-of-pelabresib-dak539-and-ruxolitinib-in-myelofibrosis-mf-100620446","NCT07357727","A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)","A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive","MANIFEST-3","Key Inclusion Criteria:\n\n* Participants have diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocythemia myelofibrosis (post-ET MF) according to the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemias 2022\n* DIPSS risk category of intermediate-1, intermediate-2 or high-risk\n* Spleen volume ≥ 450 cm3 by CT or MRI scan (local read sufficient if no central read available)\n* Have an average TSS of ≥15 within 7 days prior to randomization, using MFSAF v. 4.0 (at least 4 out of 7 TSS assessments required for average calculation)\n* Participants with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2\n* Blasts \\\u003C5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening\n* Platelet count ≥ 100 x 10\\^9\u002FL in the absence of growth factors or transfusions for the previous 4 weeks\n\nKey Exclusion Criteria:\n\n* Prior splenectomy at any time or splenic irradiation in the previous 6 months\n* Prior hematopoietic cell transplant or participant anticipated to receive a hematopoietic cell transplant within 24 weeks from the date of randomization\n* Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase (AP) or leukemic transformation\n* History of a malignancy (other than MF, PPV-MF or PET-MF) in the past 3 years in need of systemic treatment\n* Received any approved or investigational agent other than hydroxyurea or anagrelide for the treatment of MF within 14 days of first dose of study treatment or within 5 half-lives of the approved or investigational agent, whichever is longer\n* Prior treatment with any JAK inhibitor or Bromodomain and extraterminal domain (BET) inhibitor\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":419,"type":23},460,[68],"The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxolitinib alone in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not previously received Janus kinase (JAK) inhibitor therapy.",[423,424,425],"Primary Myelofibrosis (PMF)","Post-polycythemia Vera Myelofibrosis (PPV-MF)","Post-essential Thrombocythemia Myelofibrosis (PET-MF)",[427,428,429,430],"Pelabresib (DAK539)","Ruxolitinib","Adult participants","Myelofibrosis (MF)",{"date":403,"type":46},{"date":433,"type":46},"2026-05-27",{"date":435,"type":23},"2030-12-27",{"name":52,"class":53},20,{"id":439,"slug":440,"hasResults":12,"nctId":441,"briefTitle":442,"officialTitle":443,"acronym":4,"eligibilityCriteria":444,"healthyVolunteers":12,"sex":219,"minAge":19,"maxAge":4,"enrollmentInfo":445,"targetDuration":4,"studyType":24,"phases":447,"briefSummary":448,"conditions":449,"keywords":451,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":457,"startDateStruct":458,"completionDateStruct":460,"leadSponsor":462,"locationsCount":463},"100610392","phase-1-a-phase-ibii-open-label-study-of-amo959-with-lutetium-177lu-vipivotide-tetraxetan-aaa617-in-combination-with-arpi-in-adult-participants-with-psma-positive-mcrpc-100610392","NCT07226986","A Phase Ib\u002FII Open-label Study of AMO959 With Lutetium (177Lu) Vipivotide Tetraxetan (AAA617) in Combination With ARPI in Adult Participants With PSMA-positive mCRPC","A Phase Ib\u002FII Open-label, Multi-center Study of AMO959 With Lutetium (177Lu) Vipivotide Tetraxetan (AAA617) in Combination With an Androgen Receptor Pathway Inhibitor (ARPI) in Adult Participants With PSMA-positive Metastatic Castration Resistant Prostate Cancer (mCRPC)","Key Inclusion Criteria:\n\n* Signed informed consent must be obtained prior to participation in the study.\n* Participants must be adults ≥ 18 years of age.\n* Participants must have an ECOG performance status of 0 to 2.\n* Participants must have histologically confirmed adenocarcinoma of the prostate. Participants with other histology (e.g. neuroendocrine, intraductal subtype) are not eligible.\n* Phase Ib: Prior exposure of up to 1 line of taxane-based chemotherapy is permissible. Phase II: Participants must not have received taxane-based chemotherapy in mCRPC setting (allowed in mHSPC setting).\n* Participants must have PSMA-PET positive disease assessed by using a PSMA imaging agent that is approved as per protocol and are eligible as determined by the sponsor's central reading rules.\n* Castration level of testosterone (\\\u003C 50 ng\u002FdL), and\u002For use of concomitant ADT\n* Participant must have been diagnosed with mCRPC with documented progressive disease while on treatment with ARPI in mHSPC or earlier setting as their last treatment (and did not progress on more than one ARPI), based on at least 1 of the following criteria:\n\n  * Serum\u002Fplasma PSA progression is defined as 2 increases in PSA measured at least 1 week apart. The minimal start value is 2.0 ng\u002FmL; 1.0 ng\u002FmL is the minimal starting value if confirmed rise in PSA is the only indication of progression as per PCWG3 guidelines.\n  * Soft-tissue progression defined PCWG3-modified RECIST v1.1 (Eisenhauer et al 2009, Scher et al 2016).\n  * Progression of bone disease: 2 new lesions; only positivity on the bone scan defines metastatic disease to bone (PCWG3 criteria Scher et al 2016).\n\nKey Exclusion Criteria:\n\n* Concurrent local (radiation therapy to the prostate with curative intent or other prostate antineoplastic ablative procedures) or systemic (hormonal ablation, chemotherapy, immunotherapy, , RLTs) antineoplastic treatments, or within 28 days of enrollment (Phase Ib) or randomization (Phase II)\n* Prior treatment with any RLT or PSMA-targeted agents (approved or investigational)\n* Any other investigational agents within 28 days prior to first dose of any study treatment\n* Concurrent serious medical conditions that may interfere with study procedures or followup\n* Participants with a history of CNS metastases must have received therapy (surgery, whole brain radiation therapy, stereotactic radiosurgery) and be neurologically stable, asymptomatic, and not taking corticosteroids for the purpose of maintaining neurologic integrity.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":446,"type":23},123,[26,27],"The purpose of this phase Ib\u002FII study is to (a) in Phase Ib evaluate the safety, tolerability, and pharmacokinetics (PK) of AMO959 when given in combination with lutetium (177Lu) vipivotide tetraxetan (also known as \\[177Lu\\]Lu-PSMA-617 or 177Lu-PSMA-617 and hereafter referred to as AAA617) with an androgen receptor pathway inhibitor (ARPI) in participants with metastatic castration resistant prostate cancer (mCRPC) who have failed one prior ARPI and with or without prior taxane exposure, and (b) in Phase II evaluate the preliminary efficacy of AMO959 in combination with AAA617 and ARPI in participants with mCRPC who have failed one prior ARPI, but who have not yet been exposed to taxane treatment.",[450],"PSMA-positive Metastatic Castration Resistant Prostate Cancer (mCRPC) With Prior Exposure to One Prior ARPI Who Are Candidates for Taxane-based Chemotherapy",[452,453,454,455,456],"PSMA-positive metastatic castration resistant prostate cancer","AMO959","Lutetium (177Lu) vipivotide tetraxetan","Androgen Receptor Pathway Inhibitor (ARPI)","dose escalation",{"date":403,"type":46},{"date":459,"type":46},"2025-12-05",{"date":461,"type":23},"2029-09-13",{"name":52,"class":53},21,{"id":465,"slug":466,"hasResults":12,"nctId":467,"briefTitle":468,"officialTitle":469,"acronym":470,"eligibilityCriteria":471,"healthyVolunteers":12,"sex":18,"minAge":472,"maxAge":20,"enrollmentInfo":473,"targetDuration":4,"studyType":24,"phases":475,"briefSummary":476,"conditions":477,"keywords":479,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":489,"startDateStruct":490,"completionDateStruct":492,"leadSponsor":494,"locationsCount":495},"100512168","phase-2-alpelisib-in-pediatric-and-adult-patients-with-lymphatic-malformations-associated-with-a-pik3ca-mutation-100512168","NCT05948943","Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.","A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.","EPIK-L1","Key inclusion criteria:\n\n1. Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.\n2. Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.\n3. Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).\n4. Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.\n5. Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.\n6. Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.\n7. Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension \\[Groups 1 to 4\\] or granules or as an oral suspension \\[Group 5\\]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed.\n\nKey exclusion criteria:\n\n1. Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.\n2. Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.\n3. Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.\n4. Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.\n5. Participant had previous treatment with alpelisib and\u002For any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent.\n\nOther inclusion\u002Fexclusion criteria may apply","0 Years",{"count":474,"type":23},232,[27,68],"The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.",[478],"Lymphatic Malformations",[480,481,482,483,484,485,486,487,488],"Lymphatic malformations (LyM)","Cystic lymphangioma","Cystic hygroma","Lymphangioma circumscriptum","Cavernous lymphangioma","Lymphangioma","Macrocystic lymphatic malformation","Microcystic lymphatic malformationn","Alpelisib",{"date":403,"type":46},{"date":491,"type":46},"2023-11-24",{"date":493,"type":23},"2033-05-02",{"name":52,"class":53},59,{"id":497,"slug":498,"hasResults":12,"nctId":499,"briefTitle":500,"officialTitle":501,"acronym":502,"eligibilityCriteria":503,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":504,"targetDuration":4,"studyType":24,"phases":506,"briefSummary":507,"conditions":508,"keywords":510,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":401,"lastUpdatePostDateStruct":520,"startDateStruct":521,"completionDateStruct":523,"leadSponsor":525,"locationsCount":526},"100502809","phase-3-phase-iiib-study-of-ribociclib--et-in-early-breast-cancer-100502809","NCT05827081","Phase IIIb Study of Ribociclib + ET in Early Breast Cancer","A Phase IIIb Study to Characterize the Efficacy and Safety of Adjuvant Ribociclib Plus Endocrine Therapy in a Close-to-clinical Practice Patient Population With HR+ HER2- Early Breast Cancer (Adjuvant WIDER)","Adjuvant WIDER","Key Inclusion criteria:\n\n* Participant is an adult, male or female ≥ 18 years of age at the time of informed consent form signature (IC).\n* Participant has a histologically and\u002For cytologically confirmed diagnosis of estrogen-receptor positive and\u002For progesterone receptor positive breast cancer (BC) based on the most recently analyzed tissue sample tested by a local laboratory prior to enrollment.\n* Participant has HER2- BC defined as a negative in situ hybridization test or an immunohistochemistry (IHC) status of 0, 1+ or 2+. If IHC is 2+, a negative in situ hybridization (FISH, CISH, or SISH) test is required by local laboratory testing based on the most recently analyzed tissue sample.\n* Participants may have already received any standard neoadjuvant and\u002For adjuvant ET, including tamoxifen or toremifene at the time of informed consent signature, but enrollment should occur within 36 months of prior ET start date and participants should have at least 3 years remaining of endocrine adjuvant therapy.\n* For participants with prior ET treatment \\> 12 months, restaging is highly recommended (unless contradictory to local regulations) to rule out disease recurrence prior to enrollment.\n* The number of participants with prior ET between 12 and 36 months will be capped at 30%. The cap will not apply to Black or African American participants.\n* Participant has no contraindication to receive adjuvant ET in the study.\n* Participant after surgical resection where tumor was removed completely, with the final surgical specimen microscopic margins free from tumor, and belongs to one of the following categories:\n\n  * Anatomic Stage Group III, or\n  * Anatomic Stage Group IIB, or\n  * A subset of Anatomic Stage Group IIA.\n* Participant has an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1 or 2.\n* Participant has adequate bone marrow and organ function.\n* ECG values assessed by KardiaMobile-6L device, or standard 12-lead ECG per local investigator where KardiaMobile-6L cannot be used, as:\n\n  * QTcF interval at Screening \\\u003C 450 msec (QT interval using Fridericia's correction).\n  * Mean resting heart rate 50-99 beats per minute (determined from the ECG).\n\nKey Exclusion criteria:\n\n* Participant with distant metastases of BC beyond regional lymph nodes (Stage IV according to AJCC 8th edition) and\u002For evidence of recurrence after curative surgery.\n* Participant is concurrently using other antineoplastic therapy with the exception of adjuvant ET.\n* Participant has any other concurrent severe and\u002For uncontrolled medical condition that would, in the Investigator's judgment, cause unacceptable safety risks, contraindicate participant participation in the clinical study or compromise compliance with the protocol, or limit life expectancy to ≤5 years.\n* Clinically significant, uncontrolled heart disease and\u002For cardiac repolarization abnormality.\n* Pregnant or breast-feeding (lactating) women or women who plan to become pregnant or breast-feed during the trial.\n* Women of child-bearing potential (CBP), defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 21 days after stopping the treatment.\n\nOther inclusion\u002Fexclusion criteria may apply",{"count":505,"type":23},1400,[68],"The purpose of this open-label, multicenter, phase IIIb, single-arm study is to characterize the efficacy and safety of the combination of ribociclib and standard adjuvant endocrine therapy (ET) on invasive breast cancer-free survival (iBCFS), in a close to clinical practice patient population with HR-positive (HR+), HER2-negative (HER2-), Anatomic Stage Group III, IIB, and a subset of Stage IIA Early Breast Cancer (EBC).",[509],"Early Breast Cancer",[511,512,513,514,515,516,517,518,519],"Hormone receptor positive (HR+)","Human epidermal growth factor receptor-2 negative (HER2-)","Early breast cancer (EBC)","premenopausal","postmenopausal","male breast cancer","ribociclib","LEE011","Endocrine therapy (ET)",{"date":403,"type":46},{"date":522,"type":46},"2024-02-28",{"date":524,"type":23},"2030-09-20",{"name":52,"class":53},223,{"id":528,"slug":529,"hasResults":12,"nctId":530,"briefTitle":531,"officialTitle":532,"acronym":4,"eligibilityCriteria":533,"healthyVolunteers":12,"sex":18,"minAge":472,"maxAge":4,"enrollmentInfo":534,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":536,"conditions":537,"keywords":539,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":375,"lastUpdatePostDateStruct":545,"startDateStruct":546,"completionDateStruct":548,"leadSponsor":550,"locationsCount":551},"100637948","a-study-to-assess-the-effectiveness-and-safety-of-canakinumab-in-clinical-use-in-patients-with-schnitzlers-syndrome-100637948","NCT07598422","A Study to Assess the Effectiveness and Safety of Canakinumab in Clinical Use in Patients With Schnitzler's Syndrome","Specified Use-Results Study of ILARIS Solution for S.C. Injection (Schnitzler's Syndrome)","Inclusion criteria\n\n1\\. Patients diagnosed with Schnitzler's syndrome using canakinumab for the first time for the indication of Schnitzler's syndrome as described in the package insert.\n\nPatients starting treatment with canakinumab before conclusion of the contract for this study will also be included in the study so that all patients diagnosed with Schnitzler's syndrome who received canakinumab will be registered in this study.\n\nExclusion criteria\n\n1. Patients previously treated with canakinumab in Study IACT21071.\n2. All patients treated with canakinumab for the following indications:\n\n   Indications: Cryopyrin-associated periodic syndromes (familial cold autoinflammatory syndrome, Muckle-Wells syndrome, neonatal onset multisystem inflammatory disease), hyper immunoglobulin D (IgD) syndrome (mevalonate kinase deficiency), tumor necrosis factor (TNF) receptor-associated periodic syndrome, familial mediterranean fever, systemic juvenile idiopathic arthritis, adult-onset Still's disease.\n3. Patients receiving canakinumab for off-label use under the Clinical Trials Act or GCP (e.g., patient-proposed healthcare services, investigator-initiated clinical trial).",{"count":535,"type":23},10,"The aim of this study is to assess the effectiveness and safety of Ilaris (canakinumab) in clinical use in patients with Schnitzler's syndrome.",[538],"Schnitzler Syndrome",[540,541,542,543,544],"Schnitzler's syndrome","Canakinumab","ILARIS","Post-marketing surveillance","All-case surveillance",{"date":401,"type":46},{"date":547,"type":46},"2026-05-13",{"date":549,"type":23},"2032-06-30",{"name":52,"class":53},2,{"id":553,"slug":554,"hasResults":12,"nctId":555,"briefTitle":556,"officialTitle":557,"acronym":4,"eligibilityCriteria":558,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":559,"targetDuration":4,"studyType":24,"phases":561,"briefSummary":562,"conditions":563,"keywords":564,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":569,"startDateStruct":570,"completionDateStruct":572,"leadSponsor":574,"locationsCount":575},"100639848","phase-1-study-of-iev407-as-single-agent-or-in-combination-in-patients-with-advanced-hrher2--breast-cancer-100639848","NCT07604571","Study of IEV407 as Single Agent or in Combination in Patients With Advanced HR+\u002FHER2- Breast Cancer","An Open-label, Multi-center, Phase I\u002FIb Study of IEV407 as a Single Agent and in Combination With Endocrine Therapy in Patients With Advanced Hormone Receptor Positive, HER2- Negative Breast Cancer","Inclusion Criteria:\n\n* Age ≥ 18 years old\n* Patients with one of the following indications:\n\n  * Dose escalation (IEV407 single agent and in combination with fulvestrant or letrozole):\n\nHR+\u002FHER2- aBC with disease progression on or following, or have been intolerant to, at least one line of endocrine-based therapy in combination with a CDK4\u002F6 inhibitor and at least one additional line of systemic therapy in the unresectable\u002Fmetastatic setting and not be a candidate for any available standard therapy, in the investigator's judgement.\n\n\\- Dose expansion of IEV407 in combination with fulvestrant: HR+\u002FHER2- aBC with disease progression on or following, or have been intolerant to, endocrine-based therapy in combination with a CDK4\u002F6 inhibitor. They must not have received more than two prior lines of endocrine-based therapy in the unresectable\u002Fmetastatic setting. Prior cytotoxic chemotherapy and\u002For antibody-drug conjugate therapies in the unresectable\u002Fmetastatic setting are not allowed.\n\nExclusion Criteria:\n\n* Patients with inadequate bone marrow and\u002For organ functions with out-of-range laboratory values.\n* Impaired cardiac function or clinically significant cardiac disease.\n* Concurrent use of hormone replacement therapy.\n* Women of childbearing potential who are unwilling to use highly effective contraception methods, pregnant or nursing women.\n* For the combination treatment of IEV407 with fulvestrant or letrozole: Patients with symptomatic visceral disease or any disease burden that makes the patient ineligible for endocrine-based therapy.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":560,"type":23},194,[26],"The purpose of this study is to evaluate the safety, tolerability and preliminary activity of IEV407 as a single agent and in combination with endocrine therapy (fulvestrant or letrozole) in patients with advanced hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-negative) breast cancer.",[311],[565,566,567,568],"IEV407","HR+\u002FHER2- advanced breast cancer","letrozole","fulvestrant",{"date":375,"type":46},{"date":571,"type":46},"2026-05-28",{"date":573,"type":23},"2032-06-08",{"name":52,"class":53},3,{"id":577,"slug":4,"hasResults":12,"nctId":165,"briefTitle":166,"officialTitle":167,"acronym":168,"eligibilityCriteria":169,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":578,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":172,"conditions":579,"keywords":580,"overallStatus":180,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":581,"startDateStruct":582,"completionDateStruct":583,"leadSponsor":584,"locationsCount":4},"100638991",{"count":171,"type":23},[174],[174,152,176,177,178,179],{"date":375,"type":46},{"date":183,"type":23},{"date":185,"type":23},{"name":52,"class":53},{"id":586,"slug":587,"hasResults":12,"nctId":588,"briefTitle":589,"officialTitle":590,"acronym":4,"eligibilityCriteria":591,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":592,"targetDuration":4,"studyType":24,"phases":594,"briefSummary":595,"conditions":596,"keywords":598,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":602,"startDateStruct":603,"completionDateStruct":605,"leadSponsor":607,"locationsCount":608},"100634702","phase-2-an-open-label-extension-safety-and-efficacy-study-of-qcz484-in-hypertensive-patients-100634702","NCT07543120","An Open-label Extension Safety and Efficacy Study of QCZ484 in Hypertensive Patients.","A Multicenter Open-label Extension Study to Evaluate Long-term Safety and Efficacy of QCZ484 in Hypertensive Patients.","Inclusion Criteria:\n\n1. Signed informed consent must be obtained prior to participation in the study.\n2. Successful completion of treatment period on QCZ484 study medication in parent study.\n3. No significant safety issue that may place participant at safety risk as determined by the Investigator.\n\nExclusion Criteria:\n\nParticipants meeting any of the following criteria, may it be pre-existing or developed during the parent study, are not eligible for inclusion in this study:\n\n1. Evidence of hepatic disease that would put participant at risk by taking QCZ484 for long term as determined by the Investigator.\n2. Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), high-grade atrioventricular (AV) block (e.g., Mobitz type II and third-degree AV block in absence of a pacemaker) developed during the parent study.\n3. Permanent or persistent atrial fibrillation developed during the parent study.\n4. Clinically significant valvular heart disease developed during the parent study.\n5. Acute myocardial infarction (AMI) or unstable angina, or any percutaneous coronary intervention (PCI) or coronary artery bypass graft (CABG) developed during the parent study. Any history of ischemic or hemorrhagic stroke or transient ischemic attack any time prior to enrollment.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply",{"count":593,"type":23},323,[27],"The purpose of this extension study is to collect additional safety and efficacy on QCZ484 for hypertensive patients who successfully completed the parent study, CQCZ484A12201 or additional QCZ484 studies as specified in the respective parent study protocols.",[597],"Hypertension (HTN)",[599,600,601],"Hypertension","open-label extension study","QCZ484",{"date":401,"type":46},{"date":604,"type":46},"2026-05-07",{"date":606,"type":23},"2031-11-30",{"name":52,"class":53},13,{"id":610,"slug":611,"hasResults":12,"nctId":612,"briefTitle":613,"officialTitle":614,"acronym":4,"eligibilityCriteria":615,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":616,"targetDuration":4,"studyType":24,"phases":618,"briefSummary":619,"conditions":620,"keywords":622,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":626,"startDateStruct":627,"completionDateStruct":629,"leadSponsor":631,"locationsCount":632},"100615111","phase-1-study-of-gvv858-as-a-single-agent-or-in-combination-with-endocrine-therapy-in-patients-with-hrher2--breast-cancer-and-other-advanced-solid-tumors-100615111","NCT07288359","Study of GVV858 as a Single Agent or in Combination With Endocrine Therapy in Patients With HR+\u002FHER2- Breast Cancer and Other Advanced Solid Tumors","An Open-label, Multi-center, Phase I\u002FII Study of GVV858 as a Single Agent and in Combination With Endocrine Therapy in Patients With Advanced Hormone Receptor Positive, HER2- Negative Breast Cancer and Other Advanced Solid Tumors","Inclusion Criteria:\n\n* Age ≥ 18 years old.\n* Patients with one of the following histologically or cytologically confirmed advanced cancers:\n\nPhase I (patients with one of the following cancers, from whom no standard therapy is available or appropriate in the judgment of the investigator):\n\n* HR+\u002FHER2- advanced breast cancer (aBC) with disease progression on or following at least one line of hormone-based therapy in combination with a CDK4\u002F6i and at least one additional line of systemic therapy for metastatic disease.\n* Locally advanced or metastatic cancer with a CCNE1 amplification. For dose expansion only: no more than 3 prior lines of therapy for advanced or metastatic disease.\n* Metastatic castration-resistant prostate adenocarcinoma, with no documented neuroendocrine component, castrate level of testosterone, and no more than 3 prior lines of systemic therapy for metastatic disease.\n\nPhase II:\n\n* HR+\u002FHER2- aBC with disease progression on or after an endocrine therapy in combination, with a CDK4\u002F6 inhibitor for advanced disease with no more than 2 lines of endocrine therapy and no prior cytotoxic chemotherapy or antibody-drug-conjugate for advanced disease.\n\n  \\- Measurable disease as determined by RECIST v1.1.\n* BC only: If no measurable disease is present, then at least one predominantly lytic bone lesion must be present that can be accurately assessed at baseline and is suitable for repeated assessment.\n* metastatic Castration-Resistant Prostate Cancer (mCRPC) only: If no measurable disease is present per PCWG3 modified RECIST, then at least 1 metastatic lesion must be present on bone scan imaging.\n\nExclusion Criteria:\n\n* Patients with inadequate bone marrow and\u002For organ functions with out-of-range laboratory values.\n* Clinically significant, uncontrolled heart disease and\u002For cardiac repolarization abnormality including myocardial infarction (MI), coronary artery bypass graft (CABG), long QT syndrome, or risk factors for Torsades de Pointes (TdP).\n* Presence of symptomatic central nervous system (CNS) metastases or CNS metastases that require local therapy or increasing doses of corticosteroids within 2 weeks prior to study entry.\n* Patients with symptomatic visceral disease, including visceral crisis.\n* For patients with BC: Patient is concurrently using hormone replacement therapy.\n* Women of childbearing potential who are unwilling to use highly effective contraception methods, pregnant or nursing women.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":617,"type":23},205,[26,27],"Phase I: Characterize safety and tolerability of GVV858 as a single agent and in combination with fulvestrant or letrozole. Identify dose range for optimization\u002Frecommended dose for further clinical evaluation.\n\nPhase II: Further characterize the safety and tolerability of GVV858 in combination with fulvestrant in patients with hormone receptor-positive\u002Fhuman epidermal growth factor receptor 2-negative (HR+\u002FHER2-) advanced breast cancer.",[311,312,621],"Metastatic Castration-resistant Prostate Cancer",[623,317,624,101,319,625],"GVV858","Letrozole","Prostate Cancer",{"date":375,"type":46},{"date":628,"type":46},"2025-12-29",{"date":630,"type":23},"2031-05-09",{"name":52,"class":53},12,{"id":634,"slug":635,"hasResults":12,"nctId":636,"briefTitle":637,"officialTitle":638,"acronym":4,"eligibilityCriteria":639,"healthyVolunteers":640,"sex":18,"minAge":118,"maxAge":641,"enrollmentInfo":642,"targetDuration":4,"studyType":24,"phases":644,"briefSummary":645,"conditions":646,"keywords":648,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":405,"lastUpdatePostDateStruct":653,"startDateStruct":654,"completionDateStruct":656,"leadSponsor":658,"locationsCount":659},"100558096","phase-1-a-phase-iii-study-of-itu512-in-healthy-participants-and-patients-with-sickle-cell-disease-100558096","NCT06546670","A Phase I\u002FII Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease","A Phase I\u002FII Clinical Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ITU512 in Healthy Participants and Patients With Sickle Cell Disease","Key Inclusion Criteria:\n\nPart 1 (Healthy participants)\n\n* Healthy male participants and female participants of non-childbearing potential between 18-55 years of age\n* In good health as determined by the investigator's assessment of medical history, physical examination, vital signs, ECG, and laboratory tests\n* Participants must weigh at least 50 kg at screening and first baseline (admission) and must have a body mass index (BMI) within the range of 18.0-32.0 kg\u002Fm2 inclusive.\n\nPart 2 (Sickle Cell Disease)\n\n\\- Male and female participants with a diagnosis of sickle cell disease\n\nKey Exclusion Criteria:\n\nPart 1 (Healthy participants)\n\n* QTcF ≥ 450 msec (as a mean value of triplicates)\n* History of arrhythmias\n* History of significant illness which has not resolved within two (2) weeks prior to initial dosing\n* Women of child-bearing potential (WOCBP)\n\nPart 2 (Sickle Cell Disease)\n\n* Current use of hydroxyurea\u002Fhydroxycarbamide (HU\u002FHC)\n* QTcF ≥ 450 msec (as a mean value of triplicates)\n* History of arrhythmias\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",true,"55 Years",{"count":643,"type":23},161,[26,27],"The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).",[647],"Sickle Cell Disease",[649,650,651,652],"ITU512","Low molecular weight","Small molecule","Sickle cell disease",{"date":375,"type":46},{"date":655,"type":46},"2024-08-15",{"date":657,"type":23},"2030-02-19",{"name":52,"class":53},6,{"id":661,"slug":4,"hasResults":12,"nctId":190,"briefTitle":191,"officialTitle":192,"acronym":4,"eligibilityCriteria":193,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":4,"enrollmentInfo":662,"targetDuration":4,"studyType":24,"phases":663,"briefSummary":197,"conditions":664,"keywords":665,"overallStatus":180,"whyStopped":4,"lastUpdateSubmitDate":666,"lastUpdatePostDateStruct":667,"startDateStruct":668,"completionDateStruct":669,"leadSponsor":670,"locationsCount":4},"100645354",{"count":195,"type":23},[27],[199],[199,201,202,203,204,205],"2026-06-26",{"date":403,"type":46},{"date":208,"type":23},{"date":210,"type":23},{"name":52,"class":53},""]