{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT04432623","title":"The BENeFiTS Trial in Beta Thalassemia Intermedia","sponsor":"Phoenicia BioScience","phases":["PHASE1","PHASE2"],"status":"ENROLLING_BY_INVITATION","indication":null,"disease":"beta thalassemia"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"not published","detail":"No target found for the interventions.","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T22:10:30.495Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"not published","detail":"No target found for the interventions.","rule":"Any germline or somatic human genetic evidence for the target in this cancer","source":{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T22:10:30.495Z"},"context":[]},{"id":"pediatric_target","label":"Pediatric target","value":"not published","detail":"No target found for the interventions.","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 NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},"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":"Targets","text":"Open Targets lists no mechanism-of-action target for Benserazide Only Product.","source":{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T22:10:30.495Z"}}],"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 NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"36 planned; each patient moves a rate by 100 ÷ 36 = 2.8 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Safety and Tolerability; Maximum plasma concentration (Cmax); Plasma drug concentration over time","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},"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 2026-02-28; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},"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":[],"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":"5 sites in 2 countries; 4 in the United States (80%)","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623?tab=locations","date":"2026-11-11T22:10:30.495Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"no","detail":"No EMA paediatric investigation plan decision names Benserazide Only Product","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":"no","detail":"No EU orphan designation names Benserazide Only Product","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":"no","detail":"EMA lists no centrally authorised medicine of Benserazide Only Product","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 Benserazide Only Product","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":"2026-11-11T22:10:30.511Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"no","detail":"No interventional study of Benserazide Only Product includes patients under 18","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=%22Benserazide+Only+Product%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T22:10:30.519Z"},"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":"Phoenicia BioScience (academic or other)","source":{"label":"ClinicalTrials.gov NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"}},{"label":"study director","text":"Susan Perrine, MD, Phoenicia BioScience","source":{"label":"Person page","url":"/person/Susan%20Perrine%2C%20MD","date":"2026-11-11T22:10:30.495Z"}},{"label":"principal investigator","text":"Kevin Kuo, MD, University Health Network, Toronto General Hospital","source":{"label":"Person page","url":"/person/Kevin%20Kuo%2C%20MD","date":"2026-11-11T22:10:30.495Z"}},{"label":"principal investigator","text":"Sylvia Singer, MD, UCSF Benioff Children's Hospital at Oakland","source":{"label":"Person page","url":"/person/Sylvia%20Singer%2C%20MD","date":"2026-11-11T22:10:30.495Z"}},{"label":"principal investigator","text":"Hanny D Al-Samkari, MD, Massachusetts General Hospital","source":{"label":"Person page","url":"/person/Hanny%20D%20Al-Samkari%2C%20MD","date":"2026-11-11T22:10:30.495Z"}}],"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 NCT04432623","url":"https://clinicaltrials.gov/study/NCT04432623","date":"2026-11-11T22:10:30.495Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T22:10:30.495Z"},{"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":"2026-11-11T22:10:30.492Z"}