{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT02416388","title":"Study to Improve OS in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus MMF Prophylaxis of GvHD in Allografted Patients in First CR","sponsor":"University Hospital, Angers","phases":["PHASE2","PHASE3"],"status":"RECRUITING","indication":{"slug":"aml","name":"Acute myeloid leukemia"},"disease":"acute myeloid leukemia"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of POLA1, POLD4, POLD3, PPIA, IMPDH1, IMPDH2, TOP2A, TOP2B has somatic-mutation evidence in acute myeloid leukemia","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: POLA1 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000101868/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for POLA1, POLD4, POLD3, PPIA, IMPDH1, IMPDH2, TOP2A, TOP2B in acute myeloid 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: POLA1 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000101868/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"},"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 NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},"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":"Idarubicin, Daunorubicin, Methotrexate: standard agents, not counted in these flags."},{"label":"POLA1 (via HD Cytarabine)","text":"Evidence types in acute myeloid leukemia: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: POLA1 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000101868/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"POLD4 (via HD Cytarabine)","text":"Evidence types in acute myeloid leukemia: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: POLD4 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000175482/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"POLD3 (via HD Cytarabine)","text":"Evidence types in acute myeloid leukemia: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: POLD3 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000077514/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"PPIA (via Cyclosporine)","text":"Evidence types in acute myeloid leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: PPIA in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000196262/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"IMPDH1 (via Mycophenolic acid)","text":"Evidence types in acute myeloid leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: IMPDH1 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000106348/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"IMPDH2 (via Mycophenolic acid)","text":"Evidence types in acute myeloid leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: IMPDH2 in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000178035/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"TOP2A (via vosaroxin)","text":"Evidence types in acute myeloid leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: TOP2A in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000131747/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}},{"label":"TOP2B (via vosaroxin)","text":"Evidence types in acute myeloid leukemia: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: TOP2B in acute myeloid leukemia","url":"https://platform.opentargets.org/evidence/ENSG00000077097/MONDO_0018874","date":"2026-11-11T16:51:12.493Z"}}],"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 NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"3100 planned; each patient moves a rate by 100 ÷ 3100 = 0.0 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"yes","detail":"Overall survival; Cumulative incidence (CI) of acute Graft versus Host Disease (GvHD) of grade II to IV; Disease free survival","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},"context":[]},{"id":"results_overdue","label":"Results overdue","value":"yes","detail":"Primary completion 2025-07-30, 15 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 NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},"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":"1086 terminated, withdrawn or suspended trials of drugs against POLA1; 6 of the 200 most recent give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"POLA1: what already stopped","url":"/target/ENSG00000101868#stopped","date":"2026-11-11T16:51:12.539Z"}}],"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 56 sites in France","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388?tab=locations","date":"2026-11-11T16:51:12.493Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-001450-PIP01-13-M01 (vosaroxin, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 4 Dec 2015","rule":"An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name","source":{"label":"EMA EMEA-001450-PIP01-13-M01","url":"https://www.ema.europa.eu/en/medicines/human/paediatric-investigation-plans/emea-001450-pip01-13-m01","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_orphan","label":"EU orphan designation","value":"yes","detail":"EU/3/12/990 positive 26 Apr 2012: 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 EU/3/12/990","url":"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-990","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_authorised","label":"Authorised in the EU","value":"no","detail":"Qinprezo: 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 EPAR Qinprezo","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/qinprezo","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 HD Cytarabine, Cyclosporine, Mycophenolic acid, vosaroxin","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":"2026-11-11T16:51:12.567Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"643 interventional studies of HD Cytarabine, Cyclosporine, Mycophenolic acid, vosaroxin include patients under 18; 97 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=%22HD+Cytarabine%22+OR+%22Cyclosporine%22+OR+%22Mycophenolic+acid%22+OR+%22vosaroxin%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T16:51:12.658Z"},"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":"Cited case","text":"In February 2022 FDA asked its Oncologic Drugs Advisory Committee whether sintilimab's application, resting on ORIENT-11, a phase 3 run only in China, needed further trials showing it applies to US patients and US practice. One case, not a rule.","source":{"label":"FDA, ODAC 10 Feb 2022, draft questions","url":"https://www.fda.gov/media/156017/download","date":"2022-02-10"}},{"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":"University Hospital, Angers (academic or other)","source":{"label":"ClinicalTrials.gov NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"}}],"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 NCT02416388","url":"https://clinicaltrials.gov/study/NCT02416388","date":"2026-11-11T16:51:12.493Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T16:51:12.493Z"},{"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":"2026-11-11T16:51:12.487Z"}