Phase 2 N=35 Single-agent Cobimetinib for Adults With Histiocytic Disorders
Histiocytic Disorders
Primary: Best Overall Response — 21; 5; 3; 6 Participants
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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
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=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)
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=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 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=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=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 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…
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 2 N=31 Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis
Hemophagocytic Lymphohistiocytosis
Primary: Overall Survival — 25 Participants
Phase 4 N=1 A Research Study to Bank Samples for Future Evaluation to Identify Biomarkers That Predispose Crohn's Disease and Ulcerative Colitis Patients to Develop Hepatosplenic T-Cell Lymphoma (HSTCL)
Hepatosplenic T-Cell Lymphoma
Primary: Number of Samples Collected for the Identification of Biomarkers for Early Evaluation of Risk of Developing Hepatosplenic T-cell Lymphoma (HSTCL)
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=94 Assessment of Safety of Air Travel in Patients With Pulmonary Langerhans Cell Histiocytosis
Pulmonary Langerhans Cell Histiocytosis
Primary: Number of Participants With PLCH Who Experienced Spontaneous Pneumothorax During or Within 24 Hours Following Air Travel — 2 Participants
Phase 2 N=15 LMB-2 to Treat Hairy Cell Leukemia
Hairy Cell Leukemia
Primary: Number of Patients Who Obtain Partial Response/Complete Remission — 1; 5 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=40 Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
Congenital Pediatric Disorders
Primary: Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients. — 0.947 probability of overall survival
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
N/A N=340 Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau Disease
Primary: Number of Patients With Pancreatic Lesions Defined by Simple Cysts, Microcystic Adenomas, Neuroendocrine Tumors & Other Solid Lesions of the Pancreas Who Had Significant…
N/A N=36 Macrophage Markers in Periodontal and Peri-implant Health and Disease
Peri-Implantitis · Periodontitis
Primary: CD80 — 7340.34; 10854.71; 6448.43; 9874.8 arbitrary density units
Phase 2 N=5 Stem Cell Transplant Therapy With Campath-1H for Treating Advanced Mycosis Fungoides and Sezary Syndrome
Mycosis Fungoides · Sezary Syndrome
Primary: Efficacy of Nonmyeloablative Preparative Regimen — 4 Participants
N/A N=177 Diabetes and Lipid Accumulationand Heart Transplant
Diabetes Mellitus · Heart Failure
Primary: Myocyte Lipid Accumulation as Oil Red-O Positive Biopsie — 54; 21; 0 Endomyocardial Biopsies
Phase 2 N=44 Campath-1H Plus Rituximab for CD52- and CD20- Positive Refractory or Relapsed Chronic Lymphoid Disorders
Chronic Lymphocytic Leukemia
Primary: Overall Response — 8; 14; 19 Participants
N/A N=125 Therapy of HES, PV, Atypical Chronic Myelocytic Leukemia (CML) or Chronic Myelomonocytic Leukemia (CMML), and Mastocytosis With Imatinib Mesylate
Chronic Myelomonocytic Leukemia · Chronic Myeloid Leukemia · Polycythemia Vera
Primary: Number of Participants With a Complete Response (CR) — 0; 0; 2; 0 participants
Phase 2 N=118 Randomized Trial of Liposomal Amphotericin B for Histoplasmosis in AIDS Patients
Histoplasmosis · AIDS
Primary: Clinical Response — 32; 25; 28 Participants
Phase 2 N=30 Sirolimus for Autoimmune Disease of Blood Cells
Autoimmune Pancytopenia · Autoimmune Lymphoproliferative Syndrome (ALPS) · Evans Syndrome
Primary: Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus — 1 participants
Phase 2 N=6 A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
Hemophagocytic Syndrome (HPS)
Primary: Overall Survival at 2 Months — 6 Participants
N/A N=18 Role of Infected Blood Dendritic Cells in Heart Disease Risk
Chronic Periodontitis
Primary: Frequency of Blood Dendritic Cells From Baseline — 12; 14 % MFI
Early Phase 1 N=48 Donor Stem Cell Transplantation for Congenital Immunodeficiencies
Inherited Immune Deficiencies
Primary: Stem Cell Transplant Engraftment — 7; 31; 3; 0 Participants