30 closest matches · ranked by relevance
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
Activated PI3K-delta Syndrome
Primary: Number of Participants With Any Serious Adverse Events (SAEs) and Any Non-serious Adverse Events (Non-SAEs) — 5; 0 Participants
COVID-19
Primary: Overall Survival as Measured by Number of Participants Alive Through 28 Days — 10; 8 Participants
Familial Cold Autoinflammatory Syndrome (FCAS) · Familial Cold Urticaria · Muckle-Wells Syndrome (MWS)
Primary: Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) — 2.4; 3.1; 2.1; 0.5 Units of a Scale — p=<.0001
Mevalonate Kinase Deficiency
Primary: Number of Flares Per Participant During Historical Period and Treatment Period — 5; 0 Number of flares
NLRC4-MAS · XIAP Deficiency
Primary: Prevention of Flares — 2.71; NA Weeks
Lymphoma, T-Cell, Peripheral · Lymphoma, T-Cell, Cutaneous
Primary: Safety of RP6530 — 0; 0; 0; 2 Participants
B-Cell Malignancies
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 1; 3; 3; 32 Participants
Down Syndrome · Alopecia Areata · Atopic Dermatitis / Eczema
Primary: Number of Serious Adverse Events (SAE) Definitely Related to Tofacitinib Treatment. — 0 Serious Adverse Events
Peripheral T-cell Lymphoma
Primary: Overall Response Rate (ORR) as Assessed by the Investigator Using the Lugano Criteria — 53.8; 53.8 percentage of participants
DIRA
Primary: Number of Subjects Who Maintained Inflammatory Remission With Rilonacept — 6 Participants
Inflammation · Familial Mediterranean Fever · Still's Disease, Adult-Onset
Primary: Mean Change in Daily Scores — -3.09 units on a scale
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
COVID-19
Primary: Number of Participants Requiring Mechanical Ventilation or Dying — 3; 3 Participants
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
Cryopyrin-associated Periodic Syndromes · Familial Cold Autoinflammatory Syndrome · Muckle-Wells Syndrome
Primary: Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 — 94.1 Percentage of participants
22q13 Deletion Syndrome · Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1 — 15.78; 11.22; 7.61; 9.70 score on a scale
Cryopyrin-Associated Periodic Syndromes · Familial Cold Autoinflammatory Syndrome · Muckle Wells Syndrome
Primary: The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and…
Aicardi Goutieres Syndrome
Primary: Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks — 5.9 score on a scale — p=<0.0005
Primary Immune Deficiency Disorder · Hematopoietic Stem Cell Transplantation
Primary: Overall Survival — 86; 86; 87 percentage of survival probability — p=0.95
Diabetes Mellitus, Type 2
Primary: Alterations in PKC-zeta mRNA in Vastus Lateralis Skeletal Muscles — 1.76; 3.58; 2.14; 2.22 arbitrary units/ng rRNA — p=.001
Food Allergy · Anaphylaxis Food
Primary: Number of Doses of Ibrutinib for Maximal Suppression of Skin Prick Test Size to Foods — 2 doses — p=<0.0001
Primary Immunodeficiency
Primary: Annual Rate of Occurrence of Serious Bacterial Infections (SBI) — 0; 0.01; 0; 0.03 Rate of events per subject per year
ALPS · Hypersplenism · Lymphadenopathy
Primary: Number of Participants With Response — 0 participants
Neutropenia
Primary: Blood Neutrophil Counts. — 4.48 10^9 per Liter — p=<0.05
Non Hodgkin Lymphoma · Follicular Lymphoma · Indolent Lymphoma
Primary: Discontinuation Rate Due to Zydelig-related Adverse Events at 1 Year — 5 Participants
Cryopyrin-associated Periodic Syndromes · Familial Cold Autoinflammatory Syndrome · Muckle-Wells Syndrome
Primary: The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin…
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Follicular Lymphoma · Small Lymphocytic Lymphoma · Lymphoplasmacytic Lymphoma
Primary: Overall Response Rate — 57.6 percentage of participants — p=< 0.0001
Papillon-Lefevre Disease
Primary: Serum Concentrations of Interleukin (IL)-1 Beta — 1100.00; 896.21; 65559; 45734 pg/ml