{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT04855253","title":"Ph I/II Study of E7777 Prior to CAR-T for R/R LBCL","sponsor":"Masonic Cancer Center, University of Minnesota","phases":["PHASE1","PHASE2"],"status":"SUSPENDED","indication":null,"disease":"diffuse large B-cell lymphoma"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of EEF2, IL2RA, IL2RG, CD19 has somatic-mutation evidence in diffuse large B-cell lymphoma","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: EEF2 in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000167658/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for EEF2, IL2RA, IL2RG, CD19 in diffuse large B-cell lymphoma","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: EEF2 in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000167658/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"},"context":[]},{"id":"pediatric_target","label":"Pediatric target","value":"yes","detail":"CD19 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 NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},"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":"Fludarabine, Cyclophosphamide: standard agents, not counted in these flags."},{"label":"EEF2 (via E7777)","text":"Evidence types in diffuse large B-cell lymphoma: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: EEF2 in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000167658/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"}},{"label":"IL2RA (via E7777)","text":"Evidence types in diffuse large B-cell lymphoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: IL2RA in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000134460/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"}},{"label":"IL2RG (via E7777)","text":"Evidence types in diffuse large B-cell lymphoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: IL2RG in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000147168/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"}},{"label":"CD19 (via Tisagenlecleucel)","text":"Evidence types in diffuse large B-cell lymphoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: CD19 in diffuse large B-cell lymphoma","url":"https://platform.opentargets.org/evidence/ENSG00000177455/MONDO_0018905","date":"2026-11-11T17:37:35.702Z"}}],"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":"no","detail":"Non-randomised","rule":"Registry allocation field","source":{"label":"ClinicalTrials.gov NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"30 planned; 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 NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Establish maximum tolerated dose (MTD)","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},"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":"Primary completion expected 2027-09; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},"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":"32 terminated, withdrawn or suspended trials of drugs against EEF2; 6 give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"EEF2: what already stopped","url":"/target/ENSG00000167658#stopped","date":"2026-11-11T17:37:35.742Z"}}],"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 NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253?tab=locations","date":"2026-11-11T17:37:35.702Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-001654-PIP02-17-M01 (tisagenlecleucel, Treatment of mature B-cell neoplasms): PM: decision on the application for modification of an agreed PIP, 11 Sept 2019; compliance positive 24 Jun 2022; EMEA-002010-PIP01-16-M03 (axicabtagene ciloleucel, Treatment of mature B-cell neoplasms): PM: decision on the application for modification of an agreed PIP, 13 Apr 2022; EMEA-001654-PIP01-14-M04 (tisagenlecleucel, Treatment of B cell acute lymphoblastic leukaemia/lymphoblastic lymphoma): PM: decision on the application for modification of an agreed PIP, 10 Jun 2022; EMEA-002335-PIP01-18 (autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel), Treatment of mantle cell lymphoma): W: decision granting a waiver in all age groups for all conditions or indications, 15 Aug 2018","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/3/14/1393 positive 16 Dec 2014: Treatment of diffuse large B-cell lymphoma; EU/3/15/1579 positive 11 Nov 2015: Treatment of follicular lymphoma; EU/3/21/2464 positive 19 Jul 2021: Treatment of follicular lymphoma; EU/3/15/1552 withdrawn 9 Oct 2015: Treatment of mantle cell lymphoma; +6 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":"Kymriah: authorised since 23 Aug 2018, orphan medicine; Yescarta: 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 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":"yes","detail":"Tisagenlecleucel [Kymriah], 30 Aug 2017: Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse","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":"2026-11-11T17:37:35.761Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"15 interventional studies of E7777, Tisagenlecleucel, Axicabtagene Ciloleucel, Lisocabtagene Maraleucel Intravenous Suspension include patients under 18; 6 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=%22E7777%22+OR+%22Tisagenlecleucel%22+OR+%22Axicabtagene+Ciloleucel%22+OR+%22Lisocabtagene+Maraleucel+Intravenous+Suspension%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T17:37:35.766Z"},"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":"Masonic Cancer Center, University of Minnesota (academic or other)","source":{"label":"ClinicalTrials.gov NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"}},{"label":"principal investigator","text":"Veronika Bachanova, MD, Masonic Cancer Center, University of Minnesota","source":{"label":"Person page","url":"/person/Veronika%20Bachanova%2C%20MD","date":"2026-11-11T17:37:35.702Z"}}],"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 NCT04855253","url":"https://clinicaltrials.gov/study/NCT04855253","date":"2026-11-11T17:37:35.702Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T17:37:35.702Z"},{"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":"2026-11-11T17:37:35.696Z"}