30 closest matches · ranked by relevance
CD55-deficient Protein-losing Enteropathy · CHAPLE
Primary: Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24 — 100…
Recurrent Miscarriage · Spontaneous Abortion · Mannose-Binding Lectin Deficiency
Primary: Plasma MBL Level (ug/ml) — 717; 1717 ug/l — p=<0.001
Paroxysmal Nocturnal Haemoglobinuria (PNH)
Primary: Measurement of Ratio of LDH to the Upper Limit of Normal (ULN) — 5.6; 1.9 Ratio of LDH:ULN (250 U/L)
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Measurement of Serum Lactate Dehydrogenase (LDH) — 2913; 3056; 1453; 564 U/L
Congenital Protein C Deficiency
Primary: PK Part: Protein C Activity Level of TAK-662 — 0.000; 1.746; 1.616; 1.458 international unit per milliliter(IU/ml)
Hemophilia · Factor XIII Deficiency
Primary: Response to Treatment of Bleeding Events Requiring Additional Factor XIII Infusions — 30; 1; 0; 3 Number of bleeds
Antiphospholipid Antibody Syndrome · End Stage Renal Disease
Primary: Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant — 1 Participants
Diarrhea · Gastrointestinal Complications · Unspecified Childhood Solid Tumor, Protocol Specific
Primary: Number of Participants With Treatment-free Remission at 1 Year After Study Completion — 2 Participants
Common Variable Immunodeficiency (CVID), APDS / PASLI
Primary: Part I: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 2; 0; 2; 0 Participants
Hypoplasminogenemia · Congenital Plasminogen Deficiency
Primary: Overall Clinical Success in Number and Size of Lesions as Measured by Photographic or Other Imaging Modality Depending on the Organ System Affected or Change in Affected…
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change From Baseline in Hgb at Week 12 — 29.40; 4.96; 28.08; 4.62 g/L — p=0.0007
Focal Segmental Glomerulosclerosis
Primary: Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide. — 0; 0; 0.5; 0.25 proportion of participants with…
Hemophilia A
Primary: To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the…
Severe Sepsis
Primary: Mean Change in Protein C Levels From Day 1 to Day 7 — 55; 52; 79; 83 Percent Protein C Activity — p=0.011
Exocrine Pancreatic Insufficiency · Cystic Fibrosis
Primary: Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline — -11.852 percent change from baseline
Dense Deposit Disease · Membranoproliferative Glomerulonephritis
Primary: Number of Patients With Change in Proteinuria or Serum Creatinine Over Treatment Period — 5 Participants
Factor XIII Deficiency
Primary: Adverse Events — 42; 2; 2 participants
ALPS · Hypersplenism · Lymphadenopathy
Primary: Number of Participants With Response — 0 participants
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage of Participants With Reduction in Serum Lactate Dehydrogenase (LDH) Levels Within the First 4 Weeks of LFG316 Treatment as Measured by Response Rate — 10; 0…
Autoimmune Hepatitis
Primary: Safety/Tolerability Measured by Clinical Laboratory Tests, Metabolics, Cytokines, Anti-PIF Antibody, Periodic Physical Examination, Including Vital Signs Measurements…
Immune Thrombocytopenic Purpura
Primary: Set of Antibodies Most Frequently Bound to Red Blood Cells (RBCs) in Subjects Experiencing Clinically Significant Intravascular Hemolysis
Factor XIII Deficiency
Primary: The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) — 0…
Alpha₁-Antitrypsin Deficiency
Primary: AUC(0-7 Days) Based on Antigenic Content — 203.20; 198.38 mg*h/mL
Hyperlipoproteinemia Type I · Hypertriglyceridemia
Primary: Fasting Serum Triglycerides — 705; 1827; 1511; 1007 mg/dL
Sepsis
Primary: Mortality — 11 Participants
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) — 66.7; 41.7; 16.7; 0 Percentage of participants
COVID19
Primary: Number of Participants With Inflammatory Marker Reduction of at Least 50% at Any Efficacy Time Point — 5; 8 Participants — p=0.16
Biliary Atresia
Primary: Feasibility of IVIG Treatment — 23 Participants
Emphysema · Alpha 1-antitrypsin Deficiency (AATD)
Primary: Subjects With Treatment-Emergent Adverse Events (TEAEs) — 23; 18 participants