{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT03512756","title":"A Randomized Phase 2/3 Multi-Center Study of SM-88 in Participants With Metastatic Pancreatic Cancer","sponsor":"Tyme, Inc","phases":["PHASE2","PHASE3"],"status":"TERMINATED","indication":{"slug":"pancreatic","name":"Pancreatic cancer"},"disease":"pancreatic adenocarcinoma"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of SCN1A, SCN5A, SCN4A, FKBP1A has somatic-mutation evidence in pancreatic adenocarcinoma","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: SCN1A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000144285/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for SCN1A, SCN5A, SCN4A, FKBP1A in pancreatic adenocarcinoma","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: SCN1A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000144285/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"},"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 NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"},"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":"Background therapy","text":"Capecitabine: standard agents, not counted in these flags."},{"label":"SCN1A (via phenytoin)","text":"Open Targets has no association between SCN1A and pancreatic adenocarcinoma.","source":{"label":"Open Targets: SCN1A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000144285/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"}},{"label":"SCN5A (via phenytoin)","text":"Open Targets has no association between SCN5A and pancreatic adenocarcinoma.","source":{"label":"Open Targets: SCN5A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000183873/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"}},{"label":"SCN4A (via phenytoin)","text":"Open Targets has no association between SCN4A and pancreatic adenocarcinoma.","source":{"label":"Open Targets: SCN4A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000007314/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"}},{"label":"FKBP1A (via sirolimus))","text":"Evidence types in pancreatic adenocarcinoma: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: FKBP1A in pancreatic adenocarcinoma","url":"https://platform.opentargets.org/evidence/ENSG00000088832/MONDO_0006047","date":"2026-11-11T23:10:43.960Z"}}],"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 NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"130 enrolled; each patient moves a rate by 100 ÷ 130 = 0.8 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"yes","detail":"Overall Survival (OS)","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"},"context":[]},{"id":"results_overdue","label":"Results overdue","value":"no","detail":"Results posted 2024-11-06","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756?tab=results","date":"2026-11-11T23:10:43.960Z"},"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":"125 terminated, withdrawn or suspended trials of drugs against SCN1A; 19 give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"SCN1A: what already stopped","url":"/target/ENSG00000144285#stopped","date":"2026-11-11T23:10:43.998Z"}}],"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":"yes","detail":"All 20 sites in United States","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756?tab=locations","date":"2026-11-11T23:10:43.960Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-002982-PIP01-21 (sirolimus, Treatment of ornithine transcarbamylase deficiency): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 10 Jun 2022; EMEA-001416-PIP01-12-M03 (sirolimus, Treatment of chronic non-infectious uveitis): PM: decision on the application for modification of an agreed PIP, 11 Aug 2021; +1 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/3/25/3189 positive 9 Jan 2026: Treatment of osteosarcoma; EU/3/25/3051 positive 16 Apr 2025: Treatment of familial adenomatous polyposis (FAP); EU/3/13/1204 positive 13 Nov 2013: Prevention of arteriovenous access dysfunction in haemodialysis patients; EU/3/17/1910 positive 23 Aug 2017: Treatment of tuberous sclerosis; +10 more","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 data download (dated snapshot)","url":"https://www.ema.europa.eu/en/documents/report/medicines-output-orphan_designations-json-report_en.json","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_authorised","label":"Authorised in the EU","value":"yes","detail":"Hyftor: authorised since 15 May 2023, orphan medicine; Rapamune: authorised since 13 Mar 2001; Opsiria: application withdrawn","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 SM-88 used with MPS (methoxsalen, phenytoin, sirolimus), and 5-FU","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":"2026-11-11T23:10:44.018Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"417 interventional studies of SM-88 used with MPS (methoxsalen, phenytoin, sirolimus), and 5-FU include patients under 18; 102 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=%22SM-88+used+with+MPS+methoxsalen%22+OR+%22phenytoin%22+OR+%22sirolimus%22+OR+%22and+5-FU%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T23:10:44.033Z"},"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":"Tyme, Inc (industry)","source":{"label":"ClinicalTrials.gov NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"}},{"label":"study director","text":"Giuseppe DelPriore, MD, MPH, Tyme, Inc","source":{"label":"Person page","url":"/person/Giuseppe%20DelPriore%2C%20MD%2C%20MPH?ind=pancreatic","date":"2026-11-11T23:10:43.960Z"}}],"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 NCT03512756","url":"https://clinicaltrials.gov/study/NCT03512756","date":"2026-11-11T23:10:43.960Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T23:10:43.960Z"},{"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":"2026-11-11T23:10:43.955Z"}