{"ok":true,"data":{"subject":{"kind":"trial","nctId":"NCT06550141","title":"Emapalumab Prevention of CAR-T Cell Associated Toxicities","sponsor":"Marcela V. Maus, M.D.,Ph.D.","phases":["PHASE2"],"status":"RECRUITING","indication":null,"disease":"Lymphomatoid Papulosis"},"rows":[{"n":1,"question":"Is the biology right?","flags":[{"id":"somatic_driver","label":"Somatic driver evidence","value":"no","detail":"None of IFNG, RRM1, RRM2B, RRM2, CD19 has somatic-mutation evidence in Lymphomatoid Papulosis","rule":"Open Targets has somatic-mutation evidence for the target in this cancer","source":{"label":"Open Targets: IFNG in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000111537/EFO_1000341","date":"2026-11-11T17:19:08.967Z"},"context":[]},{"id":"human_genetic","label":"Human genetic evidence","value":"no","detail":"No germline or somatic evidence for IFNG, RRM1, RRM2B, RRM2, CD19 in Lymphomatoid Papulosis","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: IFNG in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000111537/EFO_1000341","date":"2026-11-11T17:19:08.967Z"},"context":[]},{"id":"pediatric_target","label":"Pediatric target","value":"yes","detail":"IFNG, CD19 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":"yes","detail":"Haematologic","rule":"From the indication, or the registry conditions when no indication matches","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},"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: standard agents, not counted in these flags."},{"label":"IFNG (via Emapalumab)","text":"Open Targets has no association between IFNG and Lymphomatoid Papulosis.","source":{"label":"Open Targets: IFNG in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000111537/EFO_1000341","date":"2026-11-11T17:19:08.967Z"}},{"label":"RRM1 (via Fludarabine Phosphate)","text":"Open Targets has no association between RRM1 and Lymphomatoid Papulosis.","source":{"label":"Open Targets: RRM1 in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000167325/EFO_1000341","date":"2026-11-11T17:19:08.967Z"}},{"label":"RRM2B (via Fludarabine Phosphate)","text":"Open Targets has no association between RRM2B and Lymphomatoid Papulosis.","source":{"label":"Open Targets: RRM2B in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000048392/EFO_1000341","date":"2026-11-11T17:19:08.967Z"}},{"label":"RRM2 (via Fludarabine Phosphate)","text":"Open Targets has no association between RRM2 and Lymphomatoid Papulosis.","source":{"label":"Open Targets: RRM2 in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000171848/EFO_1000341","date":"2026-11-11T17:19:08.967Z"}},{"label":"CD19 (via Axicabtagene Ciloleucel)","text":"Open Targets has no association between CD19 and Lymphomatoid Papulosis.","source":{"label":"Open Targets: CD19 in Lymphomatoid Papulosis","url":"https://platform.opentargets.org/evidence/ENSG00000177455/EFO_1000341","date":"2026-11-11T17:19:08.967Z"}}],"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 NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},"context":[]},{"id":"small_denominator","label":"Small denominator","value":"yes","detail":"28 planned; each patient moves a rate by 100 ÷ 28 = 3.6 points","rule":"Enrolment under 30; the arithmetic is shown, not a benchmark","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},"context":[]},{"id":"primary_os","label":"Primary endpoint is overall survival","value":"no","detail":"Incidence of grade 2+ CRS per ASTCT","rule":"A registered primary outcome measure names overall survival","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},"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 2027-08-01; not yet due","rule":"Primary completion (actual) more than 12 months ago and no results posted to the registry","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},"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":"5 terminated, withdrawn or suspended trials of drugs against IFNG. Reasons are quoted as the registry gives them on the target page.","source":{"label":"IFNG: what already stopped","url":"/target/ENSG00000111537#stopped","date":"2026-11-11T17:19:08.977Z"}}],"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":"All 2 sites in United States","rule":"All listed sites are in one country; the share of US sites is shown","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141?tab=locations","date":"2026-11-11T17:19:08.967Z"},"context":[]},{"id":"eu_pip","label":"EU paediatric investigation plan","value":"yes","detail":"EMA/PE/0000225490 (emapalumab, Treatment of haemophagocytic lymphohistiocytosis): PM: decision on the application for modification of an agreed PIP, 11 Jul 2025; EMEA-001995-PIP01-16-M04 (lisocabtagene maraleucel, Treatment of B-lymphoblastic leukaemia/lymphoma;Treatment of mature B-cell neoplasms): PM: decision on the application for modification of an agreed PIP, 27 Oct 2023; EMEA-002010-PIP01-16-M03 (axicabtagene ciloleucel, Treatment of mature B-cell neoplasms): PM: decision on the application for modification of an agreed PIP, 13 Apr 2022; EMEA-002335-PIP01-18 (autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel), Treatment of mantle cell lymphoma): W: decision granting a waiver in all age groups for all conditions or indications, 15 Aug 2018","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/3/14/1393 positive 16 Dec 2014: Treatment of diffuse large B-cell lymphoma; EU/3/15/1579 positive 11 Nov 2015: Treatment of follicular lymphoma; EU/3/18/2099 withdrawn 19 Nov 2018: Treatment of primary mediastinal large B-cell lymphoma; EU/3/18/2018 withdrawn 25 May 2018: Treatment of follicular lymphoma; +7 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":"Breyanzi: authorised since 4 Apr 2022; Yescarta: authorised since 23 Aug 2018, orphan medicine; Gamifant: refused, orphan medicine","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":"Emapalumab-lzsg [Gamifant], 20 Nov 2018: Treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy","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":"2026-11-11T17:19:08.983Z"},"context":[]},{"id":"studies_under_18","label":"Registered studies open to children","value":"yes","detail":"915 interventional studies of Emapalumab, Fludarabine Phosphate, Axicabtagene Ciloleucel, Lisocabtagene maraleucel include patients under 18; 265 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=%22Emapalumab%22+OR+%22Fludarabine+Phosphate%22+OR+%22Axicabtagene+Ciloleucel%22+OR+%22Lisocabtagene+maraleucel%22&aggFilters=ages%3Achild%2CstudyType%3Aint","date":"2026-11-11T17:19:08.992Z"},"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":"Marcela V. Maus, M.D.,Ph.D. (academic or other)","source":{"label":"ClinicalTrials.gov NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"}},{"label":"principal investigator","text":"Matthew Frigault, MD, Massachusetts General Hospital","source":{"label":"Person page","url":"/person/Matthew%20Frigault%2C%20MD","date":"2026-11-11T17:19:08.967Z"}}],"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 NCT06550141","url":"https://clinicaltrials.gov/study/NCT06550141","date":"2026-11-11T17:19:08.967Z"},{"label":"Open Targets Platform","url":"https://platform.opentargets.org","date":"2026-11-11T17:19:08.967Z"},{"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":"2026-11-11T17:19:08.965Z"}