{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT02559778","title":"Pediatric Precision Laboratory Advanced Neuroblastoma Therapy","sponsor":"Giselle Sholler","phases":["PHASE2"],"status":"RECRUITING","indication":{"slug":"neuroblastoma","name":"Neuroblastoma"},"disease":"neuroblastoma"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"yes","detail":"ALK in neuroblastoma","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: ALK in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000171094/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"},"context":[{"figure":"2.3× relative success for targets with somatic (IntOGen) support in oncology","dataset":"Minikel et al., Nature 2024","years":"drug programs to 2023","caveat":"A ratio between groups, not a probability for this program; mostly phase 2 to 3."}]},{"id":"human_genetic","label":"Human genetic evidence","value":"yes","detail":"ALK: curated genetic literature, germline, somatic","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: ALK in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000171094/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"},"context":[{"figure":"2.6× relative probability of success, phase 1 to launch, with human genetic support","dataset":"Minikel et al., Nature 2024","years":"drug programs to 2023","caveat":"All therapy areas, mostly germline evidence; not a probability for this program."}]},{"id":"pediatric_target","label":"Pediatric target","value":"yes","detail":"ALK, ABL1, PDGFRB, KIT 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 NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},"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":"ALK (via Ceritinib)","text":"Evidence types in neuroblastoma: clinical, genetic_literature, affected_pathway, literature, genetic_association, somatic_mutation; literature listed separately from human genetics. Datasources: uniprot_variants, intogen, genomics_england, uniprot_literature, clingen, eva_somatic, clinical_precedence, cancer_gene_census, crispr, europepmc.","source":{"label":"Open Targets: ALK in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000171094/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"EML4 (via Ceritinib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: EML4 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000143924/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"NPM1 (via Ceritinib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: NPM1 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000181163/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"ABL1 (via dasatinib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: ABL1 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000097007/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"BCR (via dasatinib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: BCR in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000186716/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"RAF1 (via sorafenib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: RAF1 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000132155/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"PDGFRB (via sorafenib)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: PDGFRB in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000113721/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"KIT (via sorafenib)","text":"Evidence types in neuroblastoma: literature, animal_model, clinical; literature and animal_model listed separately from human genetics. Datasources: clinical_precedence, europepmc, impc.","source":{"label":"Open Targets: KIT in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000157404/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"HDAC1 (via vorinostat)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: HDAC1 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000116478/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"HDAC3 (via vorinostat)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: HDAC3 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000171720/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}},{"label":"HDAC6 (via vorinostat)","text":"Evidence types in neuroblastoma: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc.","source":{"label":"Open Targets: HDAC6 in neuroblastoma","url":"https://platform.opentargets.org/evidence/ENSG00000094631/MONDO_0005072","date":"2026-11-12T08:51:08.551Z"}}],"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":"yes","detail":"Randomised allocation","rule":"Registry allocation field","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"no","detail":"500 planned; each patient moves a rate by 100 ÷ 500 = 0.2 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Number of days from start of therapy to date of first relapse; Number of subjects that have a targeted agent chosen for treatment.; Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},"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 2030-09; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},"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":"48 terminated, withdrawn or suspended trials of drugs against ALK; 6 give no reason. Reasons are quoted as the registry gives them on the target page.","source":{"label":"ALK: what already stopped","url":"/target/ENSG00000171094#stopped","date":"2026-11-12T08:51:08.574Z"}}],"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":"28 sites in 2 countries; 24 in the United States (86%)","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778?tab=locations","date":"2026-11-12T08:51:08.551Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMEA-000567-PIP01-09-M05 (dasatinib, Treatment of Philadelphia-chromosome (BCR-ABL translocation)-positive acute lymphoblastic leukaemia;Treatment of Philadelphia-chromosome (BCR-ABL translocation)-positive chronic myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 16 Feb 2018; compliance positive 23 Feb 2018; EMEA-000781-PIP01-09 (sorafenib tosilate, Differentiated thyroid cancer): W: decision granting a waiver in all age groups for all conditions or indications, 4 May 2010; EMEA-000805-PIP01-09 (vorinostat, Malignant pleural mesothelioma;Treatment of Cutaneous T-Cell Lymphoma): W: decision granting a waiver in all age groups for all conditions or indications, 30 Apr 2010","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/3/26/3246 positive 22 Jun 2026: Treatment of Diamond-Blackfan anaemia; EU/3/13/1200 expired 13 Nov 2013: Treatment of papillary thyroid cancer; EU/3/13/1199 expired 13 Nov 2013: Treatment of follicular thyroid cancer; - negative 23 Sept 2019: Treatment of hepatocellular carcinoma; +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":"Sprycel: authorised since 20 Nov 2006; Sorafenib Accord: authorised since 9 Nov 2022; Dasatinib Accord Healthcare: authorised since 26 Jul 2024; Zykadia: authorised since 6 May 2015; +4 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":"yes","detail":"Dasatinib [Sprycel], 21 Dec 2018: Treatment of pediatric patients 1 year of age and older with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy; Dasatinib [Sprycel], 9 Nov 2017: Treatment of pediatric patients 1 year of age and older with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase","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":"2026-11-12T08:51:08.582Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"164 interventional studies of Ceritinib, dasatinib, sorafenib, vorinostat include patients under 18; 46 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=%22Ceritinib%22+OR+%22dasatinib%22+OR+%22sorafenib%22+OR+%22vorinostat%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-12T08:51:08.587Z"},"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":"Giselle Sholler (academic or other)","source":{"label":"ClinicalTrials.gov NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"}},{"label":"study chair","text":"Giselle Sholler, MD, Beat Childhood Cancer","source":{"label":"Person page","url":"/person/Giselle%20Sholler%2C%20MD?ind=neuroblastoma","date":"2026-11-12T08:51:08.551Z"}}],"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 NCT02559778","url":"https://clinicaltrials.gov/study/NCT02559778","date":"2026-11-12T08:51:08.551Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-12T08:51:08.551Z"},{"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":"2026-11-12T08:51:08.548Z"}