{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT00124644","title":"Combination Chemotherapy and Tipifarnib in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia","sponsor":"Ohio State University Comprehensive Cancer Center","phases":["PHASE1"],"status":"TERMINATED","indication":null,"disease":"leukemia"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of TOP2A, FNTA, FNTB has somatic-mutation evidence in leukemia","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: TOP2A in leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000131747/MONDO_0005059","date":"2026-11-12T01:51:14.159Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for TOP2A, FNTA, FNTB in leukemia","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: TOP2A in leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000131747/MONDO_0005059","date":"2026-11-12T01:51:14.159Z"},"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":"yes","detail":"Haematologic","rule":"From the indication, or the registry conditions when no indication matches","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},"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":"cytarabine, etoposide: standard agents, not counted in these flags."},{"label":"TOP2A (via daunorubicin hydrochloride)","text":"Evidence types in leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: TOP2A in leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000131747/MONDO_0005059","date":"2026-11-12T01:51:14.159Z"}},{"label":"FNTA (via tipifarnib)","text":"Evidence types in leukemia: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: FNTA in leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000168522/MONDO_0005059","date":"2026-11-12T01:51:14.159Z"}},{"label":"FNTB (via tipifarnib)","text":"Evidence types in leukemia: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: FNTB in leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000257365/MONDO_0005059","date":"2026-11-12T01:51:14.159Z"}}],"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":"not published","detail":"The record gives no allocation.","rule":"Registry allocation field","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"30 enrolled; each patient moves a rate by 100 ÷ 30 = 3.3 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"not published","detail":"The record lists no primary outcome.","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},"context":[]},{"id":"results_overdue","label":"Results overdue","value":"yes","detail":"Primary completion 2007-12, 227 months ago; no results posted","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},"context":[{"figure":"40.9% of applicable trials report results within a year","dataset":"Results-reporting studies summarised in spec §11","years":"all therapy areas","caveat":"All areas, not oncology only."}]}],"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":"728 terminated, withdrawn or suspended trials of drugs against TOP2A; 3 of the 200 most recent give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"TOP2A: what already stopped","url":"/target/ENSG00000131747#stopped","date":"2026-11-12T01:51:14.200Z"}}],"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":"The only site is in United States","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644?tab=locations","date":"2026-11-12T01:51:14.159Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-001858-PIP02-16-M04 (daunorubicin;cytarabine, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 25 Oct 2024; EMEA-002871-PIP01-20 (tipifarnib, Treatment of head and neck epithelial malignant neoplasms): W: decision granting a waiver in all age groups for all conditions or indications, 22 Dec 2020","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/3/20/2333 withdrawn 19 Oct 2020: Treatment of peripheral T-cell lymphoma; EU/3/05/269 withdrawn 10 Mar 2005: Treatment of acute myeloid leukaemia; EU/3/11/942 positive 11 Jan 2012: Treatment of acute myeloid leukaemia; EU/3/08/585 positive 3 Dec 2008: Treatment of acute myeloid leukaemia","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":"Vyxeos liposomal (previously Vyxeos): authorised since 23 Aug 2018, orphan medicine","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 EPAR Vyxeos liposomal","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/vyxeos-liposomal","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":"yes","detail":"Daunorubicin Hydrochloride, 19 Dec 1979: In combination with other approved anticancer drugs, for remission induction in acute lymphocytic leukemia of children and adults","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":"2026-11-12T01:51:14.231Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"677 interventional studies of daunorubicin hydrochloride, tipifarnib include patients under 18; 127 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=%22daunorubicin+hydrochloride%22+OR+%22tipifarnib%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-12T01:51:14.239Z"},"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":"Ohio State University Comprehensive Cancer Center (academic or other)","source":{"label":"ClinicalTrials.gov NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"}},{"label":"principal investigator","text":"William G. Blum, MD, Ohio State University Comprehensive Cancer Center","source":{"label":"Person page","url":"/person/William%20G.%20Blum%2C%20MD","date":"2026-11-12T01:51:14.159Z"}}],"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 NCT00124644","url":"https://clinicaltrials.gov/study/NCT00124644","date":"2026-11-12T01:51:14.159Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-12T01:51:14.159Z"},{"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":"2026-11-12T01:51:14.154Z"}