Mepolizumab in Episodic Angioedema With Eosinophilia
EosinophiliaAngioedemaBackground: Gleich syndrome is also called episodic angioedema with eosinophilia (EAE). People with EAE have episodes of swelling. They may also have itching, hives, fever, and weight gain. During episodes, the body has very high numbers of white blood cells, especially a kind called eosinophils. Researchers think a drug called mepolizumab could help. Objective: To see if mepolizumab causes EAE symptoms to be less severe and happen less often. Eligibility: People ages 18 or older with EAE. Design: Participants will be screened under NIH protocol 94-I-0079. Participants will have 8 visits over about 6 months. The timing of some visits will depend on each participant s EAE episodes. Visits will include: Medical history Physical exam Blood and urine tests Optional bone marrow collection at first or second visit. For this, a needle will be inserted through the participant s hip bone into the marrow. Participants will get mepolizumab 3 times over about 3 months. They will get their first dose when their eosinophils are at their lowest point. They will get the drug by IV. A needle will guide a thin plastic tube into an arm vein. The drug will be given through the tube over about 30 minutes. Participants will keep a daily online log for about 3 months. The log will track their weight, temperature, and EAE symptoms. During the whole study, they will complete 2 online questionnaires about their symptoms. They will fill out 1 daily and 1 monthly. Participants will have blood and urine tests 2-3 times a week. For these, they will go to their local doctor.
Study of Dexpramipexole Chronic Sinusitis With Nasal Polyps and Eosinophilia
Chronic Sinusitis With Nasal Polyps and EosinophiliaPhase 2, open-label, multi-center study to evaluate the clinical effects of oral administration of dexpramipexole for 6 months in subjects with chronic sinusitis with nasal polyps and eosinophilia.
Safety and Tolerability of Rilonacept in Muckle-Wells Syndrome (MWS) or Schnitzler Syndrome (SchS)...
Muckle-Wells SyndromeSchnitzler SyndromeThis is a single-center open label study of the IL-1 transfusion protein rilonacept in subjects with Muckle-Wells syndrome (MWS), or Schnitzler syndrome (SchS) in Germany. Prospective subjects will be recruited from a patient population previously characterized in an observational study, and from referrals within the German CAPS community; SchS subjects will be recruited through the Charité Patient pool. The Baseline phase will begin with the Screening visit (day -21 = Visit 1) and continue for three weeks; DHAFs (Daily Health Assessment Forms) will be collected from all subjects from Day - 21 to Day 0. DHAF information including MWAS (Muckle-Wells Activity Score), or SCHAS (Schnitzler Activity Score) values from this period will be used for the baseline phase evaluation. Inclusion to receive rilonacept will occur on day 0 (= Visit 2). On day 0 eligible subjects will receive a loading dose of two subcutaneous (S.C.) injections of rilonacept for a total of 320 mg. Subsequent study drug injections of rilonacept 160 mg will be administered once a week for four weeks. After subjects complete this initial 4-week treatment phase, they will be eligible to receive rilonacept 160 mg once weekly for 48 weeks during the extended treatment phase. DHAFs will be used to assess symptoms throughout the study. Overall a max. of 12 subjects with either MWS or SchS will be enrolled.
Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients...
Cryopyrin-associated Periodic SyndromesFamilial Cold Autoinflammatory Syndrome2 moreTo date there are no approved effective therapies for the treatment of cryopyrin-associated periodic syndromes (CAPS) including Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), or Neonatal Onset Multisystem Inflammatory Disease (NOMID) in Japan. The study will assess the efficacy and safety of canakinumab in Japanese patients with cryopyrin-associated periodic syndromes (CAPS). In previous and currently ongoing CAPS studies (CACZ885A2102, CACZ885D2201, CACZ885D2304, CACZ885D2306), it has been observed that treatment with canakinumab in patients with CAPS contributed to ensure absence of relapse, to improve signs and symptoms and to prevent secondary disease complications. However, no Japanese patients have been included in those studies. This study will allow access for Japanese patients to a new potentially efficacious treatment for CAPS patients with a convenient dosing regimen.
Efficacy of Imatinib Mesylate in Hypereosinophilic Syndromes
Hypereosinophilic SyndromeChronic Eosinophilic Leukemia1 moreThe study was performed to assess: 1) clinical activity of Imatinib in patients with HES, CEL and CIH; 2) correlation between Imatinib activity and specific disease subtype; 3) long-term outcome of HES, CEL and CIH patients treated with Imatinib; 4) safety and tolerability of Imatinib administration.
Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome
Muckle Wells SyndromeThis study is designed to provide efficacy and safety data for ACZ885 (a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody) administered as an injection subcutaneously (s.c.) in patients with Muckle-Wells Syndrome. Part I is an 8-week open-label, active treatment period to identify ACZ885 responders. Part II is a double-blind, placebo-controlled period to assess primarily the efficacy of ACZ885 compared to placebo. Part III is an open-label, active treatment period where patients will receive ACZ885 every 8 weeks after withdrawal or completion of Part II.
Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes:...
Cryopyrin-Associated Periodic SyndromesFamilial Cold Autoinflammatory Syndrome2 moreThis will provided long-term safety and efficacy data for ACZ885 (a fully human anti-interleukin-1β [anti-IL-1β] monoclonal antibody) given as an injection subcutaneously in patients who participated in the CACZ885A2102 (NCT00487708), CACZ885D2201 (NCT00685373) or CACZ885D2304(NCT00465985) studies or newly identified patients with the following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome or Neonatal Onset Multisystem Inflammatory Disease. The duration of this study was 6 months with a maximum duration of 2 years
Montelukast in the Treatment of Duodenal Eosinophilia
Eosinophilic GastroenteritisDyspepsiaThis is a single site study of the effect of montelukast on eosinophil and mast cell counts and activation in the lining of the duodenum in 24 children with dyspepsia in association with duodenal eosinophilia in association with measuring the concentration of the medication in the lining of the duodenum. Patients will be endoscoped with biopsies obtained from the duodenum as part of routine clinical care. Participants in the study will then receive montelukast daily and the endoscopy with biopsies will be repeated on day 21 to measure cell counts and activation and tissue montelukast levels. Cell counts and measures of activation will be compared to pre-treatment levels.
Mepolizumab for Eosinophilic Fasciitis
Eosinophilic FasciitisThis is a proof of concept pilot study to investigate the efficacy of mepolizumab in the treatment of eosinophilic fasciitis.
A Study to Assess Subcutaneous AK002 in Eosinophilic Gastritis and/or Eosinophilic Duodenitis
Eosinophilic GastritisEosinophilic DuodenitisThis is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, and pharmacodynamic effect of subcutaneous lirentelimab (AK002), given monthly for 6 doses, in subjects with moderate to severe Eosinophilic Gastritis and/or Eosinophilic Duodenitis who have an inadequate response with, lost response to, or were intolerant to standard therapies.