{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT07616154","title":"Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease","sponsor":"St. Jude Children's Research Hospital","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 CD86, CD80, CD52, RRM1, RRM2B, RRM2 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: CD86 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000114013/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for CD86, CD80, CD52, RRM1, RRM2B, RRM2 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: CD86 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000114013/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"},"context":[]},{"id":"pediatric_target","label":"Pediatric target","value":"yes","detail":"CD52 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 NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},"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":"Cyclophosphamide, Filgrastim: standard agents, not counted in these flags."},{"label":"CD86 (via Abatacept)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: CD86 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000114013/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}},{"label":"CD80 (via Abatacept)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: CD80 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000121594/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}},{"label":"CD52 (via Alemtuzumab)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: CD52 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000169442/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}},{"label":"RRM1 (via Hydroxyurea)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: RRM1 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000167325/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}},{"label":"RRM2B (via Hydroxyurea)","text":"Evidence types in sickle cell disease: animal_model, clinical; animal_model listed separately from human genetics. Datasources: clinical_precedence, impc.","source":{"label":"Open Targets: RRM2B in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000048392/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}},{"label":"RRM2 (via Hydroxyurea)","text":"Evidence types in sickle cell disease: clinical. Datasources: clinical_precedence.","source":{"label":"Open Targets: RRM2 in sickle cell disease","url":"https://platform.opentargets.org/evidence/ENSG00000171848/MONDO_0011382","date":"2026-11-11T17:37:11.694Z"}}],"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 NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"45 planned; each patient moves a rate by 100 ÷ 45 = 2.2 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"GVHD-free and rejection free survival (GRFS)","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},"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 2034-09; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},"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":"39 terminated, withdrawn or suspended trials of drugs against CD86; 3 give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"CD86: what already stopped","url":"/target/ENSG00000114013#stopped","date":"2026-11-11T17:37:11.745Z"}}],"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 NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154?tab=locations","date":"2026-11-11T17:37:11.694Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-001072-PIP01-10-M04 (alemtuzumab, Treatment of multiple sclerosis): PM: decision on the application for modification of an agreed PIP, 15 Jul 2020; EMEA-000118-PIP04-17 (abatacept, condition not given): W: decision granting a waiver in all age groups for all conditions or indications, 22 Jun 2018; EMEA-000118-PIP02-10-M03 (abatacept, Treatment of chronic idiopathic arthritis (including rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis and juvenile idiopathic arthritis)): PM: decision on the application for modification of an agreed PIP, 15 Mar 2018; compliance positive 23 Mar 2018; EMEA-000118-PIP03-15 (abatacept, Treatment of Systemic Lupus Erythematosus (SLE)): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 16 Jan 2017; +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/3/06/424 withdrawn 29 Jan 2007: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation; EU/3/03/154 expired 9 Jul 2003: Treatment of sickle cell syndrome","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":"Lemtrada: authorised since 12 Sept 2013; Thiotepa Riemser: authorised since 26 Mar 2021; Orencia: authorised since 21 May 2007; Tepadina: authorised since 15 Mar 2010; +1 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 Abatacept, Alemtuzumab, Hydroxyurea, Thiotepa","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":"2026-11-11T17:37:11.757Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"517 interventional studies of Abatacept, Alemtuzumab, Hydroxyurea, Thiotepa include patients under 18; 116 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=%22Abatacept%22+OR+%22Alemtuzumab%22+OR+%22Hydroxyurea%22+OR+%22Thiotepa%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T17:37:11.769Z"},"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":"St. Jude Children's Research Hospital (academic or other)","source":{"label":"ClinicalTrials.gov NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"}},{"label":"principal investigator","text":"Akshay Sharma, MD, St. Jude Children's Research Hospital","source":{"label":"Person page","url":"/person/Akshay%20Sharma%2C%20MD","date":"2026-11-11T17:37:11.694Z"}}],"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 NCT07616154","url":"https://clinicaltrials.gov/study/NCT07616154","date":"2026-11-11T17:37:11.694Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T17:37:11.694Z"},{"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":"2026-11-11T17:37:11.680Z"}