{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT01861106","title":"Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations","sponsor":"National Cancer Institute (NCI)","phases":["PHASE2"],"status":"RECRUITING","indication":null,"disease":"GATA2 deficiency with susceptibility to MDS/AML"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of RRM1, RRM2B, RRM2 has somatic-mutation evidence in GATA2 deficiency with susceptibility to MDS/AML","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: RRM1 in GATA2 deficiency with susceptibility to MDS/AML","url":"https://platform.opentargets.org/evidence/ENSG00000167325/MONDO_0042982","date":"2026-11-11T21:03:03.444Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for RRM1, RRM2B, RRM2 in GATA2 deficiency with susceptibility to MDS/AML","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: RRM1 in GATA2 deficiency with susceptibility to MDS/AML","url":"https://platform.opentargets.org/evidence/ENSG00000167325/MONDO_0042982","date":"2026-11-11T21:03:03.444Z"},"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 NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},"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":"RRM1 (via Fludarabine (Fludara)","text":"Open Targets has no association between RRM1 and GATA2 deficiency with susceptibility to MDS/AML.","source":{"label":"Open Targets: RRM1 in GATA2 deficiency with susceptibility to MDS/AML","url":"https://platform.opentargets.org/evidence/ENSG00000167325/MONDO_0042982","date":"2026-11-11T21:03:03.444Z"}},{"label":"RRM2B (via Fludarabine (Fludara)","text":"Open Targets has no association between RRM2B and GATA2 deficiency with susceptibility to MDS/AML.","source":{"label":"Open Targets: RRM2B in GATA2 deficiency with susceptibility to MDS/AML","url":"https://platform.opentargets.org/evidence/ENSG00000048392/MONDO_0042982","date":"2026-11-11T21:03:03.444Z"}},{"label":"RRM2 (via Fludarabine (Fludara)","text":"Open Targets has no association between RRM2 and GATA2 deficiency with susceptibility to MDS/AML.","source":{"label":"Open Targets: RRM2 in GATA2 deficiency with susceptibility to MDS/AML","url":"https://platform.opentargets.org/evidence/ENSG00000171848/MONDO_0042982","date":"2026-11-11T21:03:03.444Z"}}],"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 NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"144 planned; each patient moves a rate by 100 ÷ 144 = 0.7 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"To determine whether allogeneic HSCT approach results in engraftment and restores normal hematopoiesis by one year in…","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},"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 2027-12-31; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},"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":"1131 terminated, withdrawn or suspended trials of drugs against RRM1; 6 of the 200 most recent give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"RRM1: what already stopped","url":"/target/ENSG00000167325#stopped","date":"2026-11-11T21:03:03.462Z"}}],"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 NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106?tab=locations","date":"2026-11-11T21:03:03.444Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"no","detail":"No EMA paediatric investigation plan decision names Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories), Busulfan","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/3/00/011 expired 30 Dec 2000: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation","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/3/00/011","url":"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-00-011","date":"2026-11-11T09:12:26Z"},"context":[]},{"id":"eu_authorised","label":"Authorised in the EU","value":"yes","detail":"Busulfan Fresenius Kabi: authorised since 22 Sept 2014; Busilvex: withdrawn since 9 Jul 2003","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":"Busulfan [Busulfex], 13 Jan 2003: For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia","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":"2026-11-11T21:03:03.476Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"473 interventional studies of Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories), Busulfan include patients under 18; 91 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=%22Busulfan+Test+dose%22+OR+%22Fludarabine+Fludara%22+OR+%22Berlex+Laboratories%22+OR+%22Busulfan%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T21:03:03.486Z"},"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":"National Cancer Institute (NCI) (academic or other)","source":{"label":"ClinicalTrials.gov NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"}},{"label":"principal investigator","text":"Danielle E Pregent-Arnold, M.D., National Cancer Institute (NCI)","source":{"label":"Person page","url":"/person/Danielle%20E%20Pregent-Arnold%2C%20M.D.","date":"2026-11-11T21:03:03.444Z"}}],"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 NCT01861106","url":"https://clinicaltrials.gov/study/NCT01861106","date":"2026-11-11T21:03:03.444Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T21:03:03.444Z"},{"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":"2026-11-11T21:03:03.442Z"}