Phase 2 N=6 A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
Hemophagocytic Syndrome (HPS)
Primary: Overall Survival at 2 Months — 6 Participants
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N/A N=40 Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis
Hemophagocytic Lymphohistiocytosis
Primary: Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10) — 5040.638; 454.392 picogram/milliliter (pg/mL)
Phase 3 N=45 Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
Primary Haemophagocytic Lymphohistiocytosis
Primary: Overall Response Rate (ORR) Second Line — 63.0 percentage of participants — p=0.0134
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
N/A N=14 Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis
Hemophagocytic Lymphohistiocytoses
Primary: Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10) — 2079.286; 15197.143 picogram/milliliter (pg/mL)
Phase 3 N=35 Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Primary Hemophagocytic Lymphohistiocytosis
Primary: Overall Response at Week 8 or End of Treatment (if Earlier) — 62.9; 50.0; 73.7 Percentage of participants — p=0.0053
Phase 2 N=31 Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis
Hemophagocytic Lymphohistiocytosis
Primary: Overall Survival — 25 Participants
Phase 3 N=58 A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
Hemophagocytic Lymphohistiocytosis
Primary: Number of Participants With Adverse Event (AE) — 37; 12; 7 Participants
Phase 2 N=35 Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Albinism · Inborn Errors of Metabolism · Oculocutaneous Albinism
Primary: Change in Forced Vital Capacity (36 Months) — -23.52; -20.93 % of predicted volume
Phase 2 N=14 A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)
Macrophage Activation Syndrome · Lymphohistiocytosis, Hemophagocytic · Arthritis, Juvenile
Primary: Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) — 13; 6; 2; 10 Participants
Phase 2 N=18 Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With Hemophagocytic Lymphohistiocytosis
Leukemia
Primary: Number of Participants With a Response to Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With…
Phase 3 N=16 Study in Pediatrics With HypEREosinophilic Syndrome (SPHERE)
Hypereosinophilic Syndrome
Primary: Number of Participants Who Experienced HES Flares Over the 52-Week Study Treatment Period — 10; 4; 1; 1 Participants
Phase 2 N=19 Stem Cell Transplant for Immunologic or Histiocytic Disorders
Hemophagocytic Lymphohistiocytosis · X-Linked Lymphoproliferative Disorders · Chediak-Higashi Syndrome
Primary: Number of Subjects With Mixed Chimerism — 3; 8; 4 participants
N/A N=17 Cytokines in Papillon-Lefèvre Syndrome
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml
N/A N=28 Sepsis-Associated Purpura Fulminans International Registry - Europe
Sepsis
Primary: Mortality — 11 Participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 3 N=98 TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Primary Immune Deficiency Disorder · Hematopoietic Stem Cell Transplantation
Primary: Overall Survival — 86; 86; 87 percentage of survival probability — p=0.95
Phase 2 N=23 Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Thrombotic Microangiopathies · Atypical Hemolytic Uremic Syndrome · Multiple Organ Dysfunction Syndrome
Primary: Survival — 15 Participants — p=<0.0001
N/A N=3 Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Lysosomal Storage Disease · Peroxisomal Disorder
Primary: Donor (Allogeneic) Hematopoietic Engraftment — 1 participants
Phase 2 N=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Phase 2 N=2 Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
Sickle Cell Anemia · Severe Aplastic Anemia · Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Number of Patients With Neutrophil Engraftment — 2 participants
N/A N=49 FOB in HSCT and Leukemia Patients With Acute Respiratory Symptoms and Pulmonary Infiltrates
Pulmonary Infiltrate New
Primary: Number of Patients With Positive Culture or Molecular Results After Brochoscopy — 40 participants
Phase 2 N=41 Stem Cell Transplantation for Hurler
Mucopolysaccharidosis I · Mucopolysaccharidosis VI · Mannosidosis
Primary: Mean Percentage of Donor Cells in Study Population (Chimerism). — 85.8; 73.2; 84.6; 81.1 Percentage
N/A N=4 Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Anemia, Aplastic · Amegakaryocytic Thrombocytopenia · Diamond-Blackfan Anemia
Primary: Treatment Failures
Phase 2 N=22 Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
Sickle Cell Disease
Primary: Event -Free Survival Rate — 19 Participants
Phase 4 N=23 Steroid Treatment for Hypereosinophilic Syndrome
Eosinophilia · Hypereosinophilic Syndrome · Leukocyte Disorder
Primary: Mean Percent Change in Eosinophil Count After Glucocorticoid Challenge — -69.6; -13.6 percentage of eosinophil count
Phase 3 N=108 Efficacy and Safety Study of Mepolizumab in Subjects With Severe Hypereosinophilic Syndrome (HES)
Hypereosinophilic Syndrome
Primary: Percentage of Participants Who Experienced an HES Flare or Who Withdrew From the Study During the 32-Week Study Treatment Period — 56; 28 Percentage of participants…
Phase 2 N=73 Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies
Leukemia, Acute Lymphocytic (ALL) · Leukemia, Myeloid, Acute(AML) · Leukemia, Myeloid, Chronic(CML)
Primary: Event-free Survival (EFS) — 54.8 Percentage of participants
Phase 3 N=33 Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
Macrophage Activation Syndrome · Secondary Hemophagocytic Lymphohistiocytosis · Still Disease
Primary: Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab — 11; 4 Participants