[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Travere Therapeutics, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":99},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,48,77],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":33,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100439568","phase-2-study-of-sparsentan-treatment-in-pediatrics-with-proteinuric-glomerular-diseases-100439568",false,"NCT05003986","Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases","A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects With Selected Proteinuric Glomerular Diseases","EPPIK","Inclusion Criteria for All Subjects (All Three Populations):\n\nA subject must meet all of the following criteria to be eligible for participation in this study:\n\n* The subject or parent\u002Flegal guardian (as appropriate) is willing and able to provide signed informed consent\u002Fassent, and where required, the subject is willing to provide assent before any screening procedures per local requirements.\n* The subject has an estimated glomerular filtration rate (eGFR) ≥30 mL\u002Fmin\u002F1.73 m2 at screening.\n* The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height.\n\nInclusion Criteria for Population 1:\n\n* The subject is male or female ≥1 year at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has a UP\u002FC ≥1.5 g\u002Fg (170 mg\u002Fmmol) at screening AND one of the following:\n* Kidney biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and\u002For other immunosuppressive disease-modifying agents.\n* Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy.\n* Kidney biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion.\n\nNote: The kidney biopsy may have been performed at any time in the past but must include light microscopy and electron microscopy characteristics and\u002For immunofluorescence findings consistent with FSGS or MCD.\n\nInclusion Criteria for Population 2:\n\n* The subject is male or female ≥2 years at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has UP\u002FC ≥0.6 g\u002Fg (68 mg\u002Fmmol) at screening AND one of the following diagnoses:\n* Kidney biopsy-confirmed IgAN, IgAV, or AS\n* Diagnosis of AS by genetic testing (pathogenic X-linked Collagen, Type IV, Alpha-5 (COL4A5) mutation OR autosomal-recessive mutations in both alleles of Collagen, Type IV, Alpha-3 (COL4A3) and\u002For Collagen, Type IV, Alpha-4 (COL4A4) OR autosomal-dominant COL4A3 and\u002For COL4A4 and digenic mutations \\[ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes\\])\n\nInclusion Criteria for Population 3:\n\n* The subject is male or female ≥8 years at screening and \\\u003C18 years of age at Day 1 (Baseline).\n* The subject has UP\u002FC ≥1.0 g\u002Fg (113 mg\u002Fmmol) at screening AND has kidney biopsy-confirmed IgAN\n* Subject weighs ≥40 kg\n* The subject has been on ACEI and\u002For ARB therapy for at least 12 weeks prior to screening\n\nExclusion Criteria for All Subjects (All Three Populations):\n\nA subject who meets any of the following will be excluded from this study:\n\n* The subject weighs \\\u003C7.3 kg at screening.\n* The subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies.\n* The subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition; eg, systemic lupus erythematosus and liver cirrhosis).\n* The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment (including, but not limited to, systemic corticosteroids, calcineurin inhibitors and mycophenolate mofetil, abatacept, cyclophosphamide, rituximab, ofatumumab, and ocrelizumab) within 6 months before screening.\n* Subjects taking chronic immunosuppressive medications (including systemic steroids) not on a stable dose for ≥1 month before screening.\n* The subject requires any of the prohibited concomitant medications as defined in the study protocol.\n* The subject has undergone any organ transplantation, with the exception of corneal transplants.\n* The subject has a documented history of congenital or acquired heart failure (modified Ross heart failure classification for children Class II to Class IV) and\u002For previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and\u002For peripheral edema.\n* The subject has hemodynamically significant cardiac valvular disease.\n* The subject has clinically significant congenital vascular disease.\n* The subject has jaundice, hepatitis, or known hepatobiliary disease, or alanine aminotransferase and\u002For aspartate aminotransferase \\>2 times the upper limit of the normal range at screening.\n* The subject has a history of malignancy within the past 2 years.\n* The subject has a screening hematocrit \\\u003C27% (0.27 L\u002FL) or a hemoglobin value \\\u003C9 g\u002FdL (90 g\u002FL).\n* The subject has a screening potassium value \\>5.5 milliequivalent (mEq)\u002FL (5.5 mmol\u002FL).\n* The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant.\n* The subject has a history of allergic response to any angiotensin II antagonist or endothelin receptor antagonist, including sparsentan, or has a hypersensitivity to any of the excipients in the study medication.\n* The female subject is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.\n* Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable (ie, can achieve a failure rate of \\\u003C1% per year) method of contraception from 7 days before the first dose of the study medication until 28 days after the last dose of study medication. Examples of highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with the inhibition of ovulation or an intrauterine device. One additional barrier method must also be used during vaginal sexual activity, such as a diaphragm, diaphragm with spermicide (preferred), or male partner's use of male condom or male condom with spermicide (preferred), from Day 1\u002FRandomization until 28 days after the last dose of study medication. Female subjects of childbearing potential are defined as those who are fertile after menarche, unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. All female subjects of childbearing potential must have a negative serum pregnancy test result at screening (Visit 1) and a negative urine pregnancy test result, with positive results confirmed by serum, at every study visit from Day 1 (Visit 3) and after.\n\nNote: Before menarche, pregnancy testing and contraceptive use are not required. However, subjects and their parents\u002Flegal guardians must be advised that, immediately upon menarche, subjects will be required to begin pregnancy testing and initiate contraceptive use. This requirement cannot be waived.\n\n* The subject has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study.\n* The subject has had prior exposure to sparsentan.\n* The subject or parent\u002Flegal guardian (as appropriate), in the opinion of the Investigator, are unable to adhere to the requirements of the study including but not limited to, a history of noncompliance and\u002For any other reason that causes the Investigator to believe the subject would not be a good candidate for the study.\n* For Population 3 - the subject is unable to swallow the study medication tablets whole.","ALL","1 Year","17 Years",{"count":21,"type":22},67,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.",[28,29,30,31,32],"Focal Segmental Glomerulosclerosis","Minimal Change Disease","Immunoglobulin A Nephropathy","IgA Vasculitis","Alport Syndrome",[34],"Alport, AS, FSGS, IgAN, IgAV, MCD, pediatrics, peds","RECRUITING","2026-05-08",{"date":38,"type":39},"2026-05-12","ACTUAL",{"date":41,"type":39},"2021-08-12",{"date":43,"type":22},"2027-04-12",{"name":45,"class":46},"Travere Therapeutics, Inc.","INDUSTRY",47,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":54,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":17,"minAge":56,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":23,"phases":60,"briefSummary":62,"conditions":63,"keywords":65,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":76},"100535073","phase-3-a-study-to-investigate-efficacy-and-safety-of-pegtibatinase-compared-with-placebo-in-participants-12-to-65-years-of-age-with-classical-homocystinuria-hcu-due-to-cystathionine-beta-synthase-deficiency-receiving-standard-of-care-treatment-100535073","NCT06247085","A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment","A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)","HARMONY","Inclusion Criteria:\n\n* Must be ≥12 to ≤65 years of age, at the time of signing the informed consent\n* Must have a diagnosis of classical HCU based on clinical, biochemical, and\u002For molecular genetic testing\n* Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \\\u003C80 µM\n* Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose.\n* Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical\u002Fsafety reasons)\n* Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical\u002Fsafety reasons)\n\nExclusion Criteria:\n\n* Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism\n* Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU).\n* History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months.\n* Body weight ≥160 kg.\n* Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines)\n* Any previous exposure to pegtibatinase and\u002For previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase\n* Prior severe immune reaction to a PEG-containing product","12 Years","65 Years",{"count":59,"type":22},70,[61],"PHASE3","The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care. Study details include:\n\n* Total Study duration: up to 38 weeks\n* Screening:\n\n  * Initial Screening duration: up to 4 weeks\n  * Pre-treatment Diet Standardization Period duration: up to 6 weeks\n* Blinded Treatment Duration: 24 weeks\n\n  * 2-week blinded dose titration period\n  * 22-week blinded assessment period\n* Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)",[64],"Homocystinuria",[66,67,68],"HCU","cystathionine beta synthase deficiency","Classical Homocystinuria","2026-05-05",{"date":36,"type":39},{"date":72,"type":39},"2023-12-28",{"date":74,"type":22},"2027-09",{"name":45,"class":46},52,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":4,"eligibilityCriteria":83,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":57,"enrollmentInfo":84,"targetDuration":4,"studyType":86,"phases":4,"briefSummary":87,"conditions":88,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":98},"100285674","natural-history-study-of-homocystinuria-caused-by-cystathionine-beta-synthase-deficiency-acappella-100285674","NCT02998710","Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)","A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)","Inclusion Criteria:\n\n* Patients who are clinically diagnosed with homocystinuria\n* Male\u002Ffemale patients aged 1 to 65 years\n* Patients who consented and\u002For assented\n* Patients who are willing and able to comply with all study-related procedures.\n\nExclusion Criteria:\n\n* Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria\n* Received any experimental therapy for homocystinuria during the 6 months prior to enrollment or expected to receive any such therapy during duration of the study",{"count":85,"type":22},150,"OBSERVATIONAL","The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices",[89],"Homocystinuria Due to CBS Deficiency","2024-11-19",{"date":92,"type":39},"2024-11-22",{"date":94,"type":39},"2017-01",{"date":96,"type":22},"2026-08",{"name":45,"class":46},11,""]