[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hemophagocytic-lymphohistiocytosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hemophagocytic-lymphohistiocytosis":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,47,73,482,504],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100631189","phase-3-comparison-of-two-etoposide-initiation-strategies-for-severe-hemophagocytic-lymphohistiocytosis-100631189",false,"NCT07497438","Comparison of Two Etoposide Initiation Strategies for Severe Hemophagocytic Lymphohistiocytosis","Comparison of Two Etoposide Initiation Strategies for Severe Hemophagocytic Lymphohistiocytosis in Intensive Care: a Randomized Trial","TIC-TAC-SAM","Inclusion Criteria:\n\n* Adult patient\n* Confirmed diagnosis of HLH:\n\n  * Hscore ≥ 169\n  * Diagnosis of HLH established by the multidisciplinary team caring for the patient(A 24\u002F7 hotline will be available in case of diagnostic uncertainty: this will be the direct line to the on-call intensivist at Avicenne Hospital, who can contact the coordinating investigator as needed)\n* First episode of HLH\n* Admission to intensive care unit\n* Presence of one or more organ failures:\n\n  * Circulatory: mBP \\\u003C 65 mmHg with lactate \\> 2 mmol\u002FL, or treatment with catecholamines\n  * Respiratory: oxygen therapy \\> 6 L\u002Fmin or need for non-invasive ventilation, high-flow nasal cannula oxygen therapy, or invasive mechanical ventilation\n  * Renal: stage 2 or higher according to KDIGO criteria, defined by a creatinine increase ≥ 2 times baseline, or urine output \\\u003C 0.5 mL\u002Fkg\u002Fh for ≥ 12 hours, or initiation of renal replacement therapy\n  * Neurological: GCS ≤ 13\n\nExclusion Criteria:\n\n* Moribund patient with refractory distributive shock: multi-organ failure requiring noradrenaline \\> 2.5 µg\u002Fkg\u002Fmin and imminent risk of death.\n* Inability to administer etoposide within 12 hours.\n* Patient treated with etoposide prior to admission to the intensive care unit.\n* Hypersensitivity to etoposide or any of its excipients.\n* Hypersensitivity or contraindication to dexamethasone or any of its excipients, as described in the package insert for the dexamethasone specialty used in the trial and in the protocol.\n* Patient with a history of CAR-T cell therapy.\n* Patient developing HLH within 15 days following chemotherapy for cancer or hematologic malignancy.\n* Patient not covered by social security.\n* Patient under legal guardianship, tutelage, or curatorship.\n* Minor patient.\n* Pregnant women (positive βhCG), women for whom pregnancy has not been excluded (βhCG not performed in women of childbearing age), or lactating women.\n* Recent vaccination with a live attenuated vaccine.\n* Participation in another interventional research study.","ALL","18 Years",{"count":20,"type":21},176,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","Hemophagocytic lymphohistiocytosis (HLH) is an immune-mediated disorder characterized by hyperactivation of the immune system, leading to a cytokine storm responsible for organ failures. Consequently, patients with HLH often require intensive care management, where their short-term prognosis is compromised (1-month mortality: 30 to 40%).\n\nTherapeutic management is urgent and consists in treating associated pathologies and employing immunomodulatory therapy. Currently, there are no clear and consistent recommendations for guiding immunomodulatory treatment in HLH due to the lack of high-level evidence studies. Experts recommend corticosteroid therapy for mild forms, whereas etoposide is proposed for severe cases, especially those with organ failures. However, in clinical practice, its use in patients with multi-organ failure is not systematic due to concerns about potential severe side effects and uncertainty regarding the contribution of severe sepsis to the clinical and biological presentation. Consequently, initiation of etoposide is sometimes delayed.\n\nOur hypothesis is that early treatment of severe HLH associated with organ failure using etoposide could reduce organ failures associated with this syndrome. Therefore, we aim to compare two strategies for initiating etoposide in severe HLH in intensive care: an early strategy where etoposide is prescribed at the onset of HLH-related organ failure, and a delayed strategy where etoposide is prescribed only if there is unfavorable progression (or lack of improvement) after treating associated pathologies, associated with corticosteroid therapy.",[27],"Hemophagocytic Lymphohistiocytosis",[29,30,31,32,33],"Hemophagocytic lymphohistiocytosis","Etoposide","Intensive care unit","cytokine storm","organe failures","NOT_YET_RECRUITING","2026-04-20",{"date":37,"type":38},"2026-04-23","ACTUAL",{"date":40,"type":21},"2026-04",{"date":42,"type":21},"2030-02",{"name":44,"class":45},"Assistance Publique - Hôpitaux de Paris","OTHER",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":58,"briefSummary":60,"conditions":61,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":46},"100613764","phase-4-zanubrutinib-combined-with-rituximab-in-the-treatment-of-secondary-hlh-in-b-cell-lymphoma-100613764","NCT07270835","Zanubrutinib Combined With Rituximab in the Treatment of Secondary HLH in B-cell Lymphoma","The Study of Zanubrutinib Combined With Rituximab in the Treatment of Secondary Hemophagocytic Lymphohistiocytosis in B-cell Lymphoma","Inclusion Criteria:\n\n* According to the diagnostic criteria of HLH-04, the patients meet the diagnostic criteria of HLH;\n* HLH patients with definite or highly suspected evidence of B-cell lymphoma; Or HLH patients whose bone marrow flow cytometry show an increased proportion of CD20-positive B cells;\n* Age of 14-80 years old, both sexes;\n* Predicted survival beyond 1 month;\n* Baseline serum creatinine ≤1.5 times ULN; Fibrinogen could be corrected to ≥0.6g\u002FL by infusion;\n* Negative serum HIV antibody; Either negative for HCV antibodies or positive for HCV antibodies but negative for HCV RNA. HBV surface antigen and HBV core antibody were negative. If any of the above was positive, HBV DNA titer in peripheral blood should be tested, and the titer was less than 1×10\\^3 copies \u002Fml;\n* Left ventricular ejection fraction (LVEF) ≥50% measured by echocardiography;\n* The patient have no serious and uncontrollable infections, such as pulmonary infection, intestinal infection, and sepsis;\n* Women of childbearing age must be confirmed to be not pregnant by pregnancy test and willing to take effective measures to prevent pregnancy during the study period and ≥12 months after the last dose; Pregnant and lactating women cannot participate; All male subjects took contraceptive measures during the trial and ≥3 months after the last dose;\n* Patients should be able to sign informed consent.\n\nExclusion Criteria:\n\n* Patients with known severe allergy to rituximab, zanubrutinib, or other BTK inhibitors;\n* Severe active infections (including bacterial, viral or fungal infections) or uncontrolled co-infections;\n* Severe organ dysfunction including NYHA class III-IV heart failure or severe arrhythmia; Liver function: ALT or AST \\>5 times the upper limit, or severe cirrhosis; Renal function: CrCl \\\u003C30 mL\u002Fmin or severe renal insufficiency;\n* Combined with other malignant tumors and the expected survival time was less than 3 months;\n* Have received other experimental drugs and had not completed the drug washout period;\n* Pregnant or lactating women, or unwilling to use effective contraception;\n* any condition that would not be suitable for participation in the study or that might affect adherence, follow-up, or safety assessment, as judged by the investigator.","14 Years","80 Years",{"count":57,"type":21},40,[59],"PHASE4","For patients who met the inclusion criteria, treatment regimens were administered: Rituximab 375 mg\u002Fm², intravenously, once weekly for 4 weeks.\n\nZanubrutinib 160 mg, orally, twice daily for 4 weeks. Combined drugs: prednisone 100 mg\u002Fm²\u002Fd, orally, d1-5; Ruxolitinib 15mg bid orally; With\u002Fwithout emapalumab as appropriate.",[27,62],"B Cell Lymphoma","RECRUITING","2025-11-26",{"date":66,"type":38},"2025-12-08",{"date":68,"type":38},"2025-11-01",{"date":70,"type":21},"2027-08",{"name":72,"class":45},"The First Affiliated Hospital of Soochow University",{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":79,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":81,"targetDuration":83,"studyType":84,"phases":4,"briefSummary":85,"conditions":86,"keywords":429,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":472,"lastUpdatePostDateStruct":473,"startDateStruct":475,"completionDateStruct":477,"leadSponsor":479,"locationsCount":481},"100193378","rare-disease-patient-registry--natural-history-study---coordination-of-rare-diseases-at-sanford-100193378","NCT01793168","Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford","Coordination of Rare Diseases at Sanford","CoRDS","Inclusion Criteria:\n\n* Diagnosis of a rare disease, a disease of unknown prevalence, undiagnosed or an unaffected carrier of a rare\u002Funcommon disease\n\nExclusion Criteria:\n\n* Diagnosis of a disease which is not rare",{"count":82,"type":21},20000,"100 Years","OBSERVATIONAL","CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org\u002FCoRDS to enroll.",[87,88,89,90,91,92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133,134,135,136,137,138,139,140,141,142,143,144,145,146,147,148,149,150,151,152,153,154,155,156,157,158,159,160,161,162,163,164,165,166,167,168,169,170,171,172,173,174,175,176,177,178,179,180,181,182,183,184,185,186,187,188,189,190,191,192,193,194,195,196,197,198,199,200,201,202,203,204,205,206,207,208,209,210,211,212,213,214,215,216,217,218,219,220,221,222,223,224,225,226,227,228,229,230,231,232,233,234,235,236,237,238,239,240,241,242,243,244,245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,276,277,278,279,280,281,282,283,284,285,286,287,288,289,290,291,292,293,294,295,296,297,298,299,300,301,302,303,304,305,306,307,308,309,310,311,312,313,314,315,316,317,318,319,320,321,322,323,324,27,325,326,327,328,329,330,331,332,333,334,335,336,337,338,339,340,341,342,343,344,345,346,347,348,349,350,351,352,353,354,355,356,357,358,359,360,361,362,363,364,365,366,367,368,369,370,371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389,390,391,392,393,394,395,396,397,398,399,400,401,402,403,404,405,406,407,408,409,410,411,412,413,414,415,416,417,418,419,420,421,422,423,424,425,426,427,428],"Rare Disorders","Undiagnosed Disorders","Disorders of Unknown Prevalence","Cornelia De Lange Syndrome","Prenatal Benign Hypophosphatasia","Perinatal Lethal Hypophosphatasia","Odontohypophosphatasia","Adult Hypophosphatasia","Childhood-onset Hypophosphatasia","Infantile Hypophosphatasia","Hypophosphatasia","Kabuki Syndrome","Bohring-Opitz Syndrome","Narcolepsy Without Cataplexy","Narcolepsy-cataplexy","Hypersomnolence Disorder","Idiopathic Hypersomnia Without Long Sleep Time","Idiopathic Hypersomnia With Long Sleep Time","Idiopathic Hypersomnia","Kleine-Levin Syndrome","Kawasaki Disease","Leiomyosarcoma","Leiomyosarcoma of the Corpus Uteri","Leiomyosarcoma of the Cervix Uteri","Leiomyosarcoma of Small Intestine","Acquired Myasthenia Gravis","Addison Disease","Hyperacusis (Hyperacousis)","Juvenile Myasthenia Gravis","Transient Neonatal Myasthenia Gravis","Williams Syndrome","Lyme Disease","Myasthenia Gravis","Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome)","Isolated Klippel-Feil Syndrome","Frasier Syndrome","Denys-Drash Syndrome","Beckwith-Wiedemann Syndrome","Emanuel Syndrome","Isolated Aniridia","Axenfeld-Rieger Syndrome","Aniridia-intellectual Disability Syndrome","Aniridia - Renal Agenesis - Psychomotor Retardation","Aniridia - Ptosis - Intellectual Disability - Familial Obesity","Aniridia - Cerebellar Ataxia - Intellectual Disability","Aniridia - Absent Patella","Aniridia","Peters Anomaly - Cataract","Peters Anomaly","Potocki-Shaffer Syndrome","Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11","Silver-Russell Syndrome Due to Imprinting Defect of 11p15","Silver-Russell Syndrome Due to 11p15 Microduplication","Syndromic Aniridia","WAGR Syndrome","Wolf-Hirschhorn Syndrome","4p16.3 Microduplication Syndrome","4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome","Autosomal Recessive Stickler Syndrome","Stickler Syndrome Type 2","Stickler Syndrome Type 1","Stickler Syndrome","Mucolipidosis Type 4","X-linked Spinocerebellar Ataxia Type 4","X-linked Spinocerebellar Ataxia Type 3","X-linked Intellectual Disability - Ataxia - Apraxia","X-linked Progressive Cerebellar Ataxia","X-linked Non Progressive Cerebellar Ataxia","X-linked Cerebellar Ataxia","Vitamin B12 Deficiency Ataxia","Toxic Exposure Ataxia","Unclassified Autosomal Dominant Spinocerebellar Ataxia","Thyroid Antibody Ataxia","Sporadic Adult-onset Ataxia of Unknown Etiology","Spinocerebellar Ataxia With Oculomotor Anomaly","Spinocerebellar Ataxia With Epilepsy","Spinocerebellar Ataxia With Axonal Neuropathy Type 2","Spinocerebellar Ataxia Type 8","Spinocerebellar Ataxia Type 7","Spinocerebellar Ataxia Type 6","Spinocerebellar Ataxia Type 5","Spinocerebellar Ataxia Type 4","Spinocerebellar Ataxia Type 37","Spinocerebellar Ataxia Type 36","Spinocerebellar Ataxia Type 35","Spinocerebellar Ataxia Type 34","Spinocerebellar Ataxia Type 32","Spinocerebellar Ataxia Type 31","Spinocerebellar Ataxia Type 30","Spinocerebellar Ataxia Type 3","Spinocerebellar Ataxia Type 29","Spinocerebellar Ataxia Type 28","Spinocerebellar Ataxia Type 27","Spinocerebellar Ataxia Type 26","Spinocerebellar Ataxia Type 25","Spinocerebellar Ataxia Type 23","Spinocerebellar Ataxia Type 22","Spinocerebellar Ataxia Type 21","Spinocerebellar Ataxia Type 20","Spinocerebellar Ataxia Type 2","Spinocerebellar Ataxia Type 19\u002F22","Spinocerebellar Ataxia Type 18","Spinocerebellar Ataxia Type 17","Spinocerebellar Ataxia Type 16","Spinocerebellar Ataxia Type 15\u002F16","Spinocerebellar Ataxia Type 14","Spinocerebellar Ataxia Type 13","Spinocerebellar Ataxia Type 12","Spinocerebellar Ataxia Type 11","Spinocerebellar Ataxia Type 10","Spinocerebellar Ataxia Type 1 With Axonal Neuropathy","Spinocerebellar Ataxia Type 1","Spinocerebellar Ataxia - Unknown","Spinocerebellar Ataxia - Dysmorphism","Non Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Spasticity-ataxia-gait Anomalies Syndrome","Spastic Ataxia With Congenital Miosis","Spastic Ataxia - Corneal Dystrophy","Spastic Ataxia","Rare Hereditary Ataxia","Rare Ataxia","Recessive Mitochondrial Ataxia Syndrome","Progressive Epilepsy and\u002For Ataxia With Myoclonus as a Major Feature","Posterior Column Ataxia - Retinitis Pigmentosa","Post-Stroke Ataxia","Post-Head Injury Ataxia","Post Vaccination Ataxia","Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract","Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus","Non-hereditary Degenerative Ataxia","Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity","Olivopontocerebellar Atrophy - Deafness","NARP Syndrome","Myoclonus - Cerebellar Ataxia - Deafness","Multiple System Atrophy, Parkinsonian Type","Multiple System Atrophy, Cerebellar Type","Multiple System Atrophy","Maternally-inherited Leigh Syndrome","Machado-Joseph Disease Type 3","Machado-Joseph Disease Type 2","Machado-Joseph Disease Type 1","Leigh Syndrome","Late-onset Ataxia With Dementia","Infection or Post Infection Ataxia","GAD Ataxia","Hereditary Episodic Ataxia","Gliadin\u002FGluten Ataxia","Friedreich Ataxia","Fragile X-associated Tremor\u002FAtaxia Syndrome","Familial Paroxysmal Ataxia","Exposure to Medications Ataxia","Episodic Ataxia With Slurred Speech","Episodic Ataxia Unknown Type","Episodic Ataxia Type 7","Episodic Ataxia Type 6","Episodic Ataxia Type 5","Episodic Ataxia Type 4","Episodic Ataxia Type 3","Episodic Ataxia Type 1","Epilepsy and\u002For Ataxia With Myoclonus as Major Feature","Early-onset Spastic Ataxia-neuropathy Syndrome","Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity","Early-onset Cerebellar Ataxia With Retained Tendon Reflexes","Early-onset Ataxia With Dementia","Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia","Dilated Cardiomyopathy With Ataxia","Cataract - Ataxia - Deafness","Cerebellar Ataxia, Cayman Type","Cerebellar Ataxia With Peripheral Neuropathy","Cerebellar Ataxia - Hypogonadism","Cerebellar Ataxia - Ectodermal Dysplasia","Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss","Brain Tumor Ataxia","Brachydactyly - Nystagmus - Cerebellar Ataxia","Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia","Autosomal Recessive Syndromic Cerebellar Ataxia","Autosomal Recessive Spastic Ataxia With Leukoencephalopathy","Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay","Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria","Autosomal Recessive Spastic Ataxia","Autosomal Recessive Metabolic Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine","Autosomal Recessive Ataxia, Beauce Type","Autosomal Recessive Ataxia Due to Ubiquinone Deficiency","Autosomal Recessive Ataxia Due to PEX10 Deficiency","Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia","Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency","Autosomal Recessive Congenital Cerebellar Ataxia","Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency","Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome","Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity","Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency","Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect","Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion","Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation","Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness","Autosomal Recessive Cerebellar Ataxia","Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly","Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation","Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy","Autosomal Dominant Spastic Ataxia Type 1","Autosomal Dominant Spastic Ataxia","Autosomal Dominant Optic Atrophy","Ataxia-telangiectasia Variant","Ataxia-telangiectasia","Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy","Autosomal Dominant Cerebellar Ataxia Type 4","Autosomal Dominant Cerebellar Ataxia Type 3","Autosomal Dominant Cerebellar Ataxia Type 2","Autosomal Dominant Cerebellar Ataxia Type 1","Autosomal Dominant Cerebellar Ataxia","Ataxia-telangiectasia-like Disorder","Ataxia With Vitamin E Deficiency","Ataxia With Dementia","Ataxia - Oculomotor Apraxia Type 1","Ataxia - Other","Ataxia - Genetic Diagnosis - Unknown","Acquired Ataxia","Adult-onset Autosomal Recessive Cerebellar Ataxia","Alcohol Related Ataxia","Multiple Endocrine Neoplasia","Multiple Endocrine Neoplasia Type II","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Multiple Endocrine Neoplasia, Type IV","Multiple Endocrine Neoplasia, Type 3","Multiple Endocrine Neoplasia (MEN) Syndrome","Multiple Endocrine Neoplasia Type 2B","Multiple Endocrine Neoplasia Type 2A","Atypical Hemolytic Uremic Syndrome","Atypical HUS","Wiedemann-Steiner Syndrome","Breast Implant-Associated Anaplastic Large Cell Lymphoma","Autoimmune\u002FInflammatory Syndrome Induced by Adjuvants (ASIA)","Behcet&#39;s Disease","Alagille Syndrome","Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD)","Lowe Syndrome","Pitt Hopkins Syndrome","1p36 Deletion Syndrome","Jansen Type Metaphyseal Chondrodysplasia","Cockayne Syndrome","Chronic Recurrent Multifocal Osteomyelitis","CRMO","Malan Syndrome","Hereditary Sensory and Autonomic Neuropathy Type Ie","VCP Disease","Hypnic Jerking","Sleep Myoclonus","Mollaret Meningitis","Recurrent Viral Meningitis","CRB1","Leber Congenital Amaurosis","Retinitis Pigmentosa","Rare Retinal Disorder","KCNMA1-Channelopathy","Primary Biliary Cirrhosis","ZMYND11","Transient Global Amnesia","Glycogen Storage Disease","Alstrom Syndrome","White Sutton Syndrome","DNM1","EIEE31","Myhre Syndrome","Recurrent Respiratory Papillomatosis","Laryngeal Papillomatosis","Tracheal Papillomatosis","Refsum Disease","Nicolaides Baraitser Syndrome","Leukodystrophy","Tango2","Cauda Equina Syndrome","Rare Gastrointestinal Disorders","Achalasia-Addisonian Syndrome","Achalasia Cardia","Achalasia Icrocephaly Syndrome","Anal Fistula","Congenital Sucrase-Isomaltase Deficiency","Eosinophilic Gastroenteritis","Idiopathic Gastroparesis","Hirschsprung Disease","Rare Inflammatory Bowel Disease","Intestinal Pseudo-Obstruction","Scleroderma","Short Bowel Syndrome","Sacral Agenesis","Sacral Agenesis Syndrome","Caudal Regression","Scheuermann Disease","SMC1A Truncated Mutations (Causing Loss of Gene Function)","Cystinosis","Juvenile Nephropathic Cystinosis","Nephropathic Cystinosis","Kennedy Disease","Spinal Bulbar Muscular Atrophy","Warburg Micro Syndrome","Mucolipidoses","Mitochondrial Diseases","Mitochondrial Aminoacyl-tRNA Synthetases","Mt-aaRS Disorders","Hypertrophic Olivary Degeneration","Non-Ketotic Hyperglycinemia","Fish Odor Syndrome","Halitosis","Isolated Congenital Asplenia","Lambert Eaton (LEMS)","Biliary Atresia","STAG1 Gene Mutation","Coffin Lowry Syndrome","Borjeson-Forssman-Lehman Syndrome","Blau Syndrome","Arginase 1 Deficiency","HSPB8 Myopathy","Beta-Mannosidosis","TBX4 Syndrome","DHDDS Gene Mutations","MAND-MBD5-Associated Neurodevelopmental Disorder","Constitutional Mismatch Repair Deficiency (CMMRD)","SPATA5 Disorder","SPATA5L1 Related Disorder","Acrodysostosis","Multi-systematic Smooth Muscle Dysfunction Syndrome","CRELD1 (Cysteine Rich With EGF Like Domains 1)","GNB1 Syndrome","Pyruvate Dehydrogenase Complex Deficiency Disease","Beta Mannosidosis","Kbg Syndrome","Labrune Syndrome","Metachromatic Leukodystrophy (MLD)","Moyamoya Disease","OPHN1 Syndrome","Oculopharyngeal Muscular Dystrophy (OPMD)","TUBB3 Mutation","WOREE (WWOX-related Epileptic Encephalopathy","SCAR12","Skraban-Deardorff Syndrome","Hereditary Myopathy With Early Respiratory Failure",[430,431,432,433,434,141,435,436,148,437,107,438,439,440,311,320,441,442,98,443,444,106,445,108,446,97,447,448,323,449,450,325,326,451,328,329,452,453,332,454,455,456,382,457,458,459,460,461,462,463,387,464,465,466,392,393,467,468,469,470,471],"Rare Diseases","Neglected Diseases","Orphan Diseases","Rare Disease Research","Registries","Ataxia","Cornelia de Lange Syndrome","Ataxia Telangiectasia","Batten Disease","Mucolipidosis IV","Klippel-Feil Syndrome","Undiagnosed","Uncommon Disease","Hypersomnia","Hyperacusis","Marinesco-Sjogren Syndrome","4p-\u002FWolf-Hirschhorn Syndrome","Narcolepsy","Wiedermann-Steiner Syndrome","Autoimmune\u002Finflammatory Syndrome Induced by Adjuvants (ASIA)","Hemophagocytic Lymphohistiocytosis (HLH)","Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia (IBMPFD)","1p36 deletion syndrome","Jansen metaphyseal chondrodysplasia","Chronic recurrent multifocal osteomyelitis (CRMO)","Malan syndrome","Hereditary Sensory and Autonomic Neuropathy","Juvenile nephropathic cystinosis","Nephropathic infantile cystinosis","Ocular cystinosis","Kennedy disease","Spinal Bulbar Muscular Atrophy (SBMA)","SMC1A Truncated Mutations (causing loss of gene function)","Leigh syndrome","Mucolipidosis","Mitochondrial aminoacyl-tRNA synthetases (Mt-aaRS Disorders)","Shine Syndrome","Intestinal Bromhidrosis Syndrome","Fish odor syndrome","Autosomal recessive extra oral halitosis","CACNA1H mutation","Dimethylglycine dehydrogenase deficiency","2025-05-22",{"date":474,"type":38},"2025-05-29",{"date":476,"type":38},"2010-07",{"date":478,"type":21},"2100-12",{"name":480,"class":45},"Sanford Health",2,{"id":483,"slug":484,"hasResults":11,"nctId":485,"briefTitle":486,"officialTitle":487,"acronym":4,"eligibilityCriteria":488,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":489,"targetDuration":4,"studyType":84,"phases":4,"briefSummary":491,"conditions":492,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":495,"lastUpdatePostDateStruct":496,"startDateStruct":498,"completionDateStruct":500,"leadSponsor":502,"locationsCount":46},"100526188","segmental-resection-combined-with-dep-regimen-for-ebv-hlh-patients-with-intestinal-involvement-100526188","NCT06131489","Segmental Resection Combined With DEP Regimen for EBV-HLH Patients With Intestinal Involvement","Efficacy and Safety of Segmental Resection Combined With the DEP Regimen for Epstein-Barr Virus Associated Hemophagocytic Lymphohistiocytosis With Intestinal Involvement","Inclusion Criteria:\n\n* Met HLH-2004 diagnostic criteria;\n* EBV-DNA in the peripheral blood \\> 1000 copies\u002Fml or EBV-encoded small RNA (EBER) detected in tissues;\n* Imaging revealed severe intestinal lesions and biopsy confirmed EBV-encoded small RNA (EBER) in the tissues;\n* Age \\>18 years old, gender is not limited;\n* Estimated survival time \\> 1 month;\n* Informed consent obtained.\n\nExclusion Criteria:\n\n* Active infections (viral, bacterial, fungal or parasitic);\n* Diagosed with malignant tumos within 5 years；\n* Uncontrolled symptoms or other diseases including, but not limited to, unstable angina pectoris, arrhythmia, respiratory failure, severe hepatic or renal insufficiency, severe coagulation dysfunction, mental illness, or other conditions deemed by the attending physician to be contraindications to surgery;\n* Heart function above grade II (NYHA);\n* Severe myocardial injury：TNT、TNI、CK-MB \\> 3 ULN;\n* Accumulated dose of doxorubicin above 400mg\u002Fm2 、epirubicin above 750mg\u002Fm2、pirarubicin above 800mg\u002Fm2 or the patients treated with anthracycline induced cardiovascular disease;\n* Pregnancy or lactating Women;\n* Allergic to pegylated liposomal doxorubicin and etoposide;\n* HIV antibody positivity;\n* Acute or chronic active hepatitis B (HBsAg positivity, HBV DNA negative acceptable), acute or chronic active hepatitis C (HCV antibody negatively acceptable; HCV antibody positivity, HCV RNA negative acceptable);\n* Participate in other clinical research at the same time;\n* The researchers considered that patients are not suitable for the study.",{"count":490,"type":21},20,"EBV-HLH is a rare disease with high mortality, especially for those with intestinal involvement. In order to reduce disease burden and improve survival of these patients, we conduct a prospective observational study to explore the efficacy and safety of segmental resection combined with the DEP regimen.",[27,493,494],"Epstein-Barr Virus","Bowel Resection","2023-11-09",{"date":497,"type":38},"2023-11-14",{"date":499,"type":21},"2023-12-01",{"date":501,"type":21},"2027-01-01",{"name":503,"class":45},"Beijing Friendship Hospital",{"id":505,"slug":506,"hasResults":11,"nctId":507,"briefTitle":508,"officialTitle":509,"acronym":4,"eligibilityCriteria":510,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":511,"enrollmentInfo":512,"targetDuration":4,"studyType":22,"phases":514,"briefSummary":515,"conditions":516,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":519,"lastUpdatePostDateStruct":520,"startDateStruct":522,"completionDateStruct":524,"leadSponsor":526,"locationsCount":46},"100498857","phase-3-l-dep-regimen-combined-with-pd-1-antibody-as-induction-therapy-for-epstein-barr-virus-positive-la-hlh-100498857","NCT05775705","L-DEP Regimen Combined With PD-1 Antibody as Induction Therapy for Epstein-Barr Virus-positive LA-HLH","The Efficacy and Safety of L-DEP Regimen Combined With PD-1 Antibody an Induction Therapy for Epstein-Barr Virus (EBV)-Positive Lymphoma-associated Hemophagocytic Lymphohistiocytosis","Inclusion Criteria:\n\n* Diagnosed as lymphoma-Hemophagocytic Lymphohistiocytosis.\n* EBV-DNA in peripheral blood \\> 1000 copies\u002Fml or EBER detected in tissue specimens.\n* Age 18\\~65，gender is not limited.\n* Estimated survival time ≥ 1 month.\n* Cardiac ultrasound LVEF≥50%; No Active bleeding of the internal organs(digestive tract, lung, brain, etc.); If the patient has dyspnea, oxygenation index \\>250.\n* Signed informed consent.\n\nExclusion Criteria:\n\n* Heart function above grade II (NYHA).\n* Severe myocardial injury：TNT、TNI、CK-MB \\> 3 ULN.\n* Accumulated dose of doxorubicin above 400mg\u002Fm2 、epirubicin above 750mg\u002Fm2、pirarubicin above 800mg\u002Fm2 or the patients treated with anthracycline induced cardiovascular disease.\n* Pregnancy or lactating Women.\n* Allergic to pegylated liposomal doxorubicin，etoposide，or PD-1 antibody.\n* Thyroid dysfunction.\n* HIV antibody positivity.\n* Acute or chronic active hepatitis B (HBsAg positivity, HBV DNA negative acceptable), acute or chronic active hepatitis C (HCV antibody negatively acceptable; HCV antibody positivity, HCV RNA negative acceptable).\n* Participate in other clinical research at the same time.\n* The researchers considered that patients are not suitable for the study.","65 Years",{"count":513,"type":21},25,[24],"The efficacy and safety of L-DEP (PEG-aspargase, liposomal doxorubicin, etoposide, and methylprednisolone) regimen combined with PD-1 Antibody an induction therapy for Epstein-Barr virus (EBV)-positive lymphoma-associated hemophagocytic lymphohistiocytosis.",[517,27,493,518],"PD-1 Antibody","Lymphoma","2023-03-08",{"date":521,"type":38},"2023-03-20",{"date":523,"type":21},"2023-08-01",{"date":525,"type":21},"2026-10-01",{"name":503,"class":45}]