30 closest matches · ranked by relevance
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
Inflammation · Familial Mediterranean Fever · Still's Disease, Adult-Onset
Primary: Mean Change in Daily Scores — -3.09 units on a scale
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 Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
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
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…
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
Familial Amyloid Polyneuropathy
Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of 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…
NLRC4-MAS · XIAP Deficiency
Primary: Prevention of Flares — 2.71; NA Weeks
Mevalonate Kinase Deficiency
Primary: Number of Flares Per Participant During Historical Period and Treatment Period — 5; 0 Number of flares
Fetal Alcohol Spectrum Disorders
Primary: Eyberg Child Behavior Inventory - Intensity at Baseline and 12-week Follow-Up — 68.33; 69.13; 68.40; 64.47 T-score — p=.602
Familial Adenomatous Polyposis
Primary: Polyp Number — 22.6; 18.6 polyps — p=0.58
TNF-receptor Associated Periodic Syndromes (TRAPS)
Primary: Percentage of Participants With Complete or Almost Complete Response at Day 15 — 95 Percentage of participants
Familial Amyloid Polyneuropathy · Familial Amyloidosis
Primary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001
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
Fever · Infection · Inflammation
Primary: Number of Participants With Clinically-assigned Retrospective Phenotype — 655; 0 Participants
Systemic Juvenile Idiopathic Arthritis · Hereditary Periodic Fevers
Primary: Number of Participants With Adverse Events — 7; 29 Participants
Periodic Fevers Syndrome
Primary: Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths — 1; 1; 0; 0 Participants
Friedreich Ataxia
Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027
Hemophagocytic Lymphohistiocytosis
Primary: Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10) — 5040.638; 454.392 picogram/milliliter (pg/mL)
Fibromyalgia
Primary: Pitsburgh Sleep Quality Index — 8.3 score on a questionnarie
Acute Febrile Illness
Primary: The Etiologies (Bacteria, Viruses, Parasites, Fungi and Others) of Fever Illness Expressed in Percentages of Enrolled Subjects. — 621; 362; 15; 5 Participants
Sepsis
Primary: Mortality — 11 Participants
Adenomatous Polyposis Coli
Primary: Change in Marked Polyp Size — 16; 4; 0 Size of marked polyps
Familial Hypercholesterolaemia
Primary: Percent Change From Baseline in LDL-C — -37.86; -43.67; -42.88 Percentage change — p=<0.001
Familial Chylomicronemia Syndrome · Lipoprotein Lipase Deficiency · Hyperlipoproteinemia Type 1
Primary: Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) — -550.00 mg/dL
Familial Partial Lipodystrophy · Hypertriglyceridemia · Fatty Liver
Primary: Change in Fasting Serum Triglyceride (at 12 Weeks) — -0.44; -20.27 percent change — p=0.517
Familial Chylomicronemia Syndrome
Primary: Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3 — 17.6; -76.5 percent change — p=< 0.0001