[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100497853":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":19,"centralContacts":24,"locations":34,"responsibleParty":53,"collaborators":55,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":64,"eligibilityCriteria":65,"healthyVolunteers":60,"sex":66,"minAge":11,"maxAge":67,"enrollmentInfo":68,"targetDuration":11,"studyType":71,"phases":72,"briefSummary":74,"conditions":75,"keywords":77,"overallStatus":37,"whyStopped":11,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":89},{"fullName":5,"class":6},"Assistance Publique - Hôpitaux de Paris","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Ruxolitinib","EXPERIMENTAL",null,[13],"Drug: Ruxolitinib",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":11},"DRUG","Form: tablets, 50 mg\u002Fm2\u002Fday in two administrations. Maximum dose is 100 mg\u002Fday. Administration in association with Methylprednisolone IV (or Prednisolone PO) starting at 2 mg\u002Fkg\u002Fday in two administrations.\n\nDuration of treatment: until D-1 of conditioning for allogeneic HSCT OR 9weeks for patients who are not eligible for HSCT.",[9],[20],{"name":21,"affiliation":22,"role":23},"Despina MOSHOUS, MD, PhD","Hôpital Necker-Enfants Malades","STUDY_CHAIR",[25,30],{"name":21,"role":26,"phone":27,"phoneExt":28,"email":29},"CONTACT","01 44 49 48 23","+33","despina.moshous@aphp.fr",{"name":31,"role":26,"phone":32,"phoneExt":28,"email":33},"Laure CHOUPEAUX","01 44 38 17 11","laure.choupeaux@aphp.fr",[35],{"facility":36,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":49},"Hopital Necker Enfants malades","RECRUITING","Paris","Île-de-France Region","75015","France","FR",{"type":44,"coordinates":45},"Point",[46,47],2.3488,48.85341,{"lat":47,"lon":46},[50],{"name":51,"role":26,"phone":27,"phoneExt":28,"email":52},"Despina MOSHOUS, MD, PHD","despina.mouhous@aphp.fr",{"type":54,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[56],{"name":57,"class":6},"URC-CIC Paris Descartes Necker Cochin","100497853","phase-2-ruxolitinib-as-first-line-treatment-in-primary-haemophagocytic-lymphohistiocytosis-r-hlh-100497853",false,"NCT05762640","Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)","Efficacy of Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (HLH) in Children: a Phase 2, Multicentre, Non-comparative Study","R-HLH","Inclusion Criteria\n\n* Patient aged 0 to 22 years\n* Patient with HLH syndrome confirmed by at least one of the two criteria:\n\n  1. Confirmed genetic diagnosis of a condition predisposing to primary HLH (see table 1 and table 2) or abnormal expression of perforin, MUNC13-4, SAP or XIAP in FACS and\u002For positive family history OR\n  2. Presence of at least 5 of the 8 following HLH diagnostic criteria:\n* Fever\n* Splenomegaly\n* Cytopenia (affecting at least two cell lineages)\n* Haemoglobin \\\u003C 9 g\u002Fdl (\\\u003C10 g\u002FdL in neonates)\n* Platelets \\\u003C 100,000\u002FµL\n* Absolute neutrophil count (ANC) \\\u003C 1,000\u002FµL\n* Hypertriglyceridemia and\u002For hypofibrinogenemia\n* Fasting triglycerides ≥ 3 mmol\u002Fl\n* Fibrinogen \\\u003C1.5 g\u002FL\n* Haemophagocytosis found in a histological sample (without evidence of a malignant process or an underlying rheumatic disorder)\n* Decreased or absent NK function\n* Ferritin ≥ 500 µg\u002Fl\n* Presence of activated T cells in the immune phenotyping as evidenced by expression of the activation marker DR (superior to the normal value of the laboratory) OR CD25 soluble (sIL-2 receptor) ≥ 2,400 U\u002FmL.\n* Patient with no previous specific treatment for HLH syndrome\n* For patients of childbearing age : using an effective method of contraception during the trial, and through to 90 days after EOS for male participants and 30 days after EOS for female participants\n* Freely given, informed and written consent of legal representative of the participant or consent of the adult participant\n* Affiliation to Social Security.\n\nExclusion Criteria\n\n* Previous treatment with ATG, Alemtuzumab, Etoposide, JAK-inhibitors, rifampicin and\u002For anti-Interferon gamma antibodies. St. John's Wort, or any other strong CYP3A4 inducers.\n* Previous treatment with corticosteroids and\u002For cyclosporine A for more than 14 days\n* Isolated CNS disease.\n* Contraindication to receive Ruxolitinib:\n* History of hypersensitivity to the active substance or to any of the excipients\n* Pregnant or lactating female patient\n* Contraindication to receive methylprednisolone or prednisolone\n* History of hypersensitivity to the active substance or to any of the excipients\n* Any infectious condition with the exception of infections, which are the trigger for lymphohistiocytic activation.\n* Patient with acute very severe renal impairment (Creatinine Clearance \\\u003C15 mL\u002Fmin\u002F1.73m²) who are NOT receiving dialysis.\n* Patient with Grade 4 hepatic failure according to the CTCAE v5.0 of 27 November 2017 (Life-threatening consequences; moderate to severe encephalopathy; coma)\n* Past or know active tuberculosis\n* Known rheumatologic disorder.\n* Known active malignancy.\n* Patient who is taking another investigational agent or is enrolled in another treatment protocol.\n* Patient who cannot tolerate administration of drugs PO or through NG","ALL","22 Years",{"count":69,"type":70},20,"ESTIMATED","INTERVENTIONAL",[73],"PHASE2","The purpose of this project is to study the survival of patients until Haematopoietic Stem Cell Transplantation following the use of Ruxolitinib as first-line treatment associated to corticosteroids in primary HLH.",[76],"Haemophagocytic Lymphohistiocytosis",[76,78,9,79],"Lymphohistiocytic activation syndrome","Jakavi","2026-04-20",{"date":82,"type":83},"2026-04-23","ACTUAL",{"date":85,"type":83},"2024-11-10",{"date":87,"type":70},"2028-03",{"name":5,"class":6},1]