30 closest matches · ranked by relevance
Hereditary Periodic Fevers
Primary: Percentage of Participants With Resolution of Initial Flare and Absence of New Flares up to the End of the Randomized Treatment Epoch (16 Weeks) — 61.29; 6.25; 35.14…
Familial Mediterranean Fever
Primary: To Assess the Efficacy of Rilonacept in Decreasing the Number of Acute FMF Attacks. — 0.77; 2.00 number of attacks per month — p=0.027
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
Inflammation · Familial Mediterranean Fever · Still's Disease, Adult-Onset
Primary: Mean Change in Daily Scores — -3.09 units on a scale
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
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…
Periodic Fevers Syndrome
Primary: Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths — 1; 1; 0; 0 Participants
Systemic Juvenile Idiopathic Arthritis · Hereditary Periodic Fevers
Primary: Number of Participants With Adverse Events — 7; 29 Participants
Mevalonate Kinase Deficiency
Primary: Number of Flares Per Participant During Historical Period and Treatment Period — 5; 0 Number of flares
Hereditary Angioedema · Angioneurotic Edema · Genetic Disorders
Primary: Time to Beginning of Relief of Symptoms — 62; 508; 61 minutes — p=0.003
Hereditary Haemorrhagic Telangiectasia (HHT)
Primary: Number of Participants That Achieving Personal Recommended Intake of Iron — 43 Participants
Hereditary Angioedema · Angioneurotic Edema
Primary: Time to Beginning of Relief of Symptoms — 68; 122; 258; 62.5 minutes — p=0.001
TNF-receptor Associated Periodic Syndromes (TRAPS)
Primary: Percentage of Participants With Complete or Almost Complete Response at Day 15 — 95 Percentage of participants
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 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…
Hereditary Angioedema
Primary: Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) — 0.46 hours
Familial Cold Autoinflammatory Syndrome
Primary: Ratio of Fold Change From Pre-challenge to the Highest Post-challenge Value of White Cell Count (WCC) Between Treatment and Screening Period — 0.82 ratio of fold change
NLRC4-MAS · XIAP Deficiency
Primary: Prevention of Flares — 2.71; NA Weeks
Hyperkalemic Periodic Paralysis · Hypokalemic Periodic Paralysis
Primary: HYP Attack Rate — 0.9; 4.8 attacks per week
Hereditary Angioedema
Primary: Time to Start of Relief of Symptoms From HAE Attack — 1.17; 0.5; 1.5 Hours — p=0.0025
Hereditary Haemorrhagic Telangiectasia
Primary: HHT Patients Who by the Questionnaire Did or Did Not Self Report a Medical Complication During Flight — 77 percentage of patients
Muckle Wells Syndrome
Primary: Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part) — 0; 81.3 percent of participants
Migraine
Primary: Progesterone Receptor Gene Polymorphism PROGINS in Women With Menstrual Migraine and Women Without Migraine — 208; 112; 64; 39 participants — p=0.513
Hereditary Angioedema
Primary: Time-Normalized Number of Hereditary Angioedema (HAE) Attacks Per Month During Treatment Period — 0.27; 2.01 number of HAE attacks per month — p=<0.001
Fever · Infection · Inflammation
Primary: Number of Participants With Clinically-assigned Retrospective Phenotype — 655; 0 Participants
Genetic Disorders · Hereditary Angioedema
Primary: HAE Attacks/Week — 0.9; 0.4 attacks/week
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Hereditary Angioedema (HAE)
Primary: Time to Peak Concentration (Tmax) of a Single Subcutaneous (SC) Dose of Icatibant — 0.42; 0.55; 0.57 Hour (h)
Erythropoietic Protoporphyria · EPP · X-Linked Protoporphyria
Primary: The Hospital Anxiety and Depression Scale (HADS) — 4.6; 1.9 score on a scale
Hereditary Angioedema
Primary: Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) — 0; 0; 0; 0 Participants