{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT03121001","title":"Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease","sponsor":"University of Illinois Chicago","phases":["PHASE2"],"status":"RECRUITING","indication":null,"disease":"sickle cell disease"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of FKBP1A, IMPDH1, IMPDH2 has somatic-mutation evidence in sickle cell disease","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: FKBP1A in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000088832/MONDO_0011382","date":"2026-11-11T16:43:32.349Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for FKBP1A, IMPDH1, IMPDH2 in sickle cell disease","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: FKBP1A in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000088832/MONDO_0011382","date":"2026-11-11T16:43:32.349Z"},"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 NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},"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":"FKBP1A (via Sirolimus)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: FKBP1A in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000088832/MONDO_0011382","date":"2026-11-11T16:43:32.349Z"}},{"label":"IMPDH1 (via mycophenolate mofetil)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: IMPDH1 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000106348/MONDO_0011382","date":"2026-11-11T16:43:32.349Z"}},{"label":"IMPDH2 (via mycophenolate mofetil)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: IMPDH2 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000178035/MONDO_0011382","date":"2026-11-11T16:43:32.349Z"}}],"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":"Single group","rule":"Registry allocation field","source":{"label":"ClinicalTrials.gov NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"50 planned; each patient moves a rate by 100 ÷ 50 = 2.0 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Estimate the number of patients who engraft by Day +60","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},"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 2029-05; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},"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":"578 terminated, withdrawn or suspended trials of drugs against FKBP1A; 8 of the 200 most recent give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"FKBP1A: what already stopped","url":"/target/ENSG00000088832#stopped","date":"2026-11-11T16:43:32.373Z"}}],"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 NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001?tab=locations","date":"2026-11-11T16:43:32.349Z"},"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; CellCept: authorised since 14 Feb 1996; Rapamune: authorised since 13 Mar 2001; Mycophenolate mofetil Teva: authorised since 21 Feb 2008; +3 more","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 ATG, Sirolimus, mycophenolate mofetil","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":"2026-11-11T16:43:32.393Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"965 interventional studies of ATG, Sirolimus, mycophenolate mofetil include patients under 18; 206 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=%22ATG%22+OR+%22Sirolimus%22+OR+%22mycophenolate+mofetil%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T16:43:32.403Z"},"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":"University of Illinois Chicago (academic or other)","source":{"label":"ClinicalTrials.gov NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"}},{"label":"principal investigator","text":"Damiano Rondelli, MD, University of Illinois Chicago","source":{"label":"Person page","url":"/person/Damiano%20Rondelli%2C%20MD","date":"2026-11-11T16:43:32.349Z"}}],"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 NCT03121001","url":"https://clinicaltrials.gov/study/NCT03121001","date":"2026-11-11T16:43:32.349Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T16:43:32.349Z"},{"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":"2026-11-11T16:43:32.346Z"}