{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT03762850","title":"A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA Nephropathy","sponsor":"Travere Therapeutics, Inc.","phases":["PHASE3"],"status":"COMPLETED","indication":null,"disease":"IgA glomerulonephritis"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of EDNRA, AGTR1, SLC5A2 has somatic-mutation evidence in IgA glomerulonephritis","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: EDNRA in IgA glomerulonephritis","url":"https://platform.opentargets.org/evidence/ENSG00000151617/MONDO_0005342","date":"2026-11-12T03:09:40.554Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for EDNRA, AGTR1, SLC5A2 in IgA glomerulonephritis","rule":"Any germline or somatic human genetic evidence for the target in this cancer, counted apart from animal-model and literature evidence","source":{"label":"Open Targets: EDNRA in IgA glomerulonephritis","url":"https://platform.opentargets.org/evidence/ENSG00000151617/MONDO_0005342","date":"2026-11-12T03:09:40.554Z"},"context":[]},{"id":"pediatric_target","label":"Pediatric target","value":"no","detail":"Not on the FDA list","rule":"Target is on FDA's Pediatric Molecular Target List (relevant targets); membership alone neither requires nor waives a study","source":{"label":"FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01))","url":"https://www.fda.gov/media/191157/download?attachment","date":"2026-11-11T07:05:03Z"},"context":[]},{"id":"haematologic","label":"Haematologic cancer","value":"no","detail":"Solid tumour","rule":"From the indication, or the registry conditions when no indication matches","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"},"context":[{"figure":"Likelihood of approval from phase 1: 7.5% haematologic vs 4.6% solid","dataset":"BIO / Informa / QLS 2021","years":"2011–2020","caveat":"Population-level, not a prediction."}]}],"facts":[{"label":"EDNRA (via sparsentan)","text":"Evidence types in IgA glomerulonephritis: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: EDNRA in IgA glomerulonephritis","url":"https://platform.opentargets.org/evidence/ENSG00000151617/MONDO_0005342","date":"2026-11-12T03:09:40.554Z"}},{"label":"AGTR1 (via sparsentan)","text":"Evidence types in IgA glomerulonephritis: literature, animal_model, clinical; literature and animal_model listed separately from human genetics. Datasources: clinical_precedence, impc, europepmc.","source":{"label":"Open Targets: AGTR1 in IgA glomerulonephritis","url":"https://platform.opentargets.org/evidence/ENSG00000144891/MONDO_0005342","date":"2026-11-12T03:09:40.554Z"}},{"label":"SLC5A2 (via Dapagliflozin)","text":"Evidence types in IgA glomerulonephritis: literature, animal_model, clinical; literature and animal_model listed separately from human genetics. Datasources: clinical_precedence, impc, europepmc.","source":{"label":"Open Targets: SLC5A2 in IgA glomerulonephritis","url":"https://platform.opentargets.org/evidence/ENSG00000140675/MONDO_0005342","date":"2026-11-12T03:09:40.554Z"}}],"pending":[{"what":"Normal-tissue expression in vital organs","source":"Human Protein Atlas (ticket 36)"},{"what":"Pan-essential in cell lines","source":"DepMap (ticket 36)"},{"what":"Approved drug on this target","source":"Drugs@FDA (ticket 30)"}]},{"n":2,"question":"Can the result be trusted?","flags":[{"id":"randomised","label":"Randomised","value":"yes","detail":"Randomised allocation","rule":"Registry allocation field","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"406 enrolled; each patient moves a rate by 100 ÷ 406 = 0.2 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Percent Change From Baseline in the Urine Protein/Creatinine (UP/C) at Week 36","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"},"context":[{"figure":"Non-OS primary endpoint: odds ratio 2.79 for a positive phase 3","dataset":"Otsuka 2021, 400 phase 3 oncology trials","years":"to 2020","caveat":"Shown with the next figure: a surrogate is easier to hit, and often a weak guide to survival."},{"figure":"52% of surrogate-to-OS correlations are low","dataset":"Prasad 2015","years":"published to 2014","caveat":"Shown with the figure above."}]},{"id":"results_overdue","label":"Results overdue","value":"no","detail":"Results posted 2024-07-03","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850?tab=results","date":"2026-11-12T03:09:40.554Z"},"context":[]}],"facts":[],"pending":[{"what":"Treated and evaluable counts, assessor, confirmed response, follow-up and data cutoff","source":"Readout extraction (ticket 32)"},{"what":"Primary endpoint changed between registry versions","source":"Registry versions in the store (ticket 32)"}]},{"n":3,"question":"What must it beat?","flags":[],"facts":[],"pending":[{"what":"Control-arm result and approved therapies for the same segment, with label links","source":"Drugs@FDA, FDA labels and readouts (tickets 30, 32)"}]},{"n":4,"question":"Too crowded, or too late?","flags":[],"facts":[{"label":"Stopped on this target","text":"84 terminated, withdrawn or suspended trials of drugs against EDNRA; 10 give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"EDNRA: what already stopped","url":"/target/ENSG00000151617#stopped","date":"2026-11-12T03:09:40.561Z"}}],"pending":[{"what":"Programs on the target by phase and months behind the leader","source":"Programs and Drugs@FDA (ticket 33)"}]},{"n":5,"question":"Can it be approved, and stay approved?","flags":[{"id":"single_country","label":"Single-country","value":"no","detail":"162 sites in 18 countries; 40 in the United States (25%)","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850?tab=locations","date":"2026-11-12T03:09:40.554Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMA/PE/0000226166 (sparsentan, Treatment of primary IgA nephropathy): PM: decision on the application for modification of an agreed PIP, 16 May 2025; EMA/PE/0000184400 (dapagliflozin;eplerenone, Treatment of heart failure): W: decision granting a waiver in all age groups for all conditions or indications, 5 Dec 2024; EMEA-003572-PIP01-23 (sitagliptin;dapagliflozin, Treatment of type II diabetes mellitus): W: decision granting a waiver in all age groups for all conditions or indications, 21 Jun 2024; EMEA-003534-PIP01-23 (sitagliptin;dapagliflozin, Treatment of type II diabetes mellitus): W: decision granting a waiver in all age groups for all conditions or indications, 12 Apr 2024; +16 more","rule":"An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name","source":{"label":"EMA data download (dated snapshot)","url":"https://www.ema.europa.eu/en/documents/report/medicines-output-paediatric_investigation_plans-output-json-report_en.json","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_orphan","label":"EU orphan designation","value":"yes","detail":"EU/3/20/2345 positive 19 Oct 2020: Treatment of primary IgA nephropathy","rule":"An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status","source":{"label":"EMA EU/3/20/2345","url":"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2345","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_authorised","label":"Authorised in the EU","value":"yes","detail":"Irbesartan Hydrochlorothiazide Zentiva (previously Irbesartan Hydrochlorothiazide Winthrop): authorised since 18 Jan 2007; Irbesartan Zentiva (previously Irbesartan Winthrop): authorised since 19 Jan 2007; Qtern: authorised since 15 Jul 2016; Edistride: authorised since 9 Nov 2015; +17 more","rule":"An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file","source":{"label":"EMA data download (dated snapshot)","url":"https://www.ema.europa.eu/en/documents/report/medicines-output-medicines_json-report_en.json","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"fda_orphan","label":"FDA orphan designation","value":"not published","detail":"FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link","rule":"Would be: an FDA orphan designation names the drug","source":{"label":"FDA orphan designations search","url":"https://www.accessdata.fda.gov/scripts/opdlisting/oopd/","date":""},"context":[]},{"id":"fda_ped_approval","label":"FDA pediatric oncology approval","value":"no","detail":"FDA's table lists no pediatric approval of sparsentan, irbesartan, Dapagliflozin","rule":"FDA's Pediatric Oncology Drug Approvals table names the drug","source":{"label":"FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03)","url":"https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology-drug-approvals","date":"2026-11-12T03:09:40.572Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"35 interventional studies of sparsentan, irbesartan, Dapagliflozin include patients under 18; 14 are recruiting, not yet recruiting or active","rule":"The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age","source":{"label":"ClinicalTrials.gov search","url":"https://clinicaltrials.gov/search?intr=%22sparsentan%22+OR+%22irbesartan%22+OR+%22Dapagliflozin%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-12T03:09:40.572Z"},"context":[]}],"facts":[{"label":"Caveat","text":"Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where the sites are is a fact about the record, not a judgement of its quality."},{"label":"Rare pediatric disease voucher","text":"FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news.","source":{"label":"FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs","url":"https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs","date":"2026-11-11"}}],"pending":[{"what":"Precedent approvals for target, modality and endpoint; accelerated-approval status","source":"Drugs@FDA and FDA accelerated-approval tables (ticket 30)"}]},{"n":6,"question":"Can the sponsor finish?","flags":[],"facts":[{"label":"Sponsor","text":"Travere Therapeutics, Inc. (industry)","source":{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"}},{"label":"study director","text":"Radko Komers, MD, PhD, Travere Therapeutics, Inc.","source":{"label":"Person page","url":"/person/Radko%20Komers%2C%20MD%2C%20PhD","date":"2026-11-12T03:09:40.554Z"}}],"pending":[{"what":"Filed cash, quarterly spend and the runway arithmetic","source":"SEC XBRL company facts (ticket 37)"},{"what":"Sponsor's completed vs stopped oncology trials and results-reporting record","source":"Registry, by company (ticket 37)"}]}],"limits":"Public records cannot show: sales forecasts; a probability of success for this asset; full deal terms and net prices; FDA designations and action dates before approval (shown only as company-reported); clinical holds unless the company disclosed them; manufacturing; patents on unapproved drugs; whether preclinical findings have been replicated.","sources":[{"label":"ClinicalTrials.gov NCT03762850","url":"https://clinicaltrials.gov/study/NCT03762850","date":"2026-11-12T03:09:40.554Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-12T03:09:40.554Z"},{"label":"FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01))","url":"https://www.fda.gov/media/191157/download?attachment","date":"2026-11-11T07:05:03Z"}]},"source":"ClinicalTrials.gov + Open Targets","fetchedAt":"2026-11-12T03:09:40.552Z"}