Active clinical trials for "Familial Mediterranean Fever"

The primary objective of this study was to evaluate safety and tolerability of ACZ885 in this extension study. This extension study offered the opportunity for participants who completed Epoch 4 of the preceding CACZ885N2301 (NCT02059291) study to continue to be treated with ACZ885 until approval in Japan of the drug in Periodic Fever Syndromes or until development of ACZ885 in Periodic Fever Syndromes was suspended.

FMF is the most common periodic fever with a worldwide patient population estimated as 150,000, mainly located in the Eastern Mediterranean basin. colchicine is the established therapy of choice ,however, around 20.000 patients worldwide fail to respond or cannot tolerate therapeutic doses, thereby suffering from recurrent debilitating, severe, painful attacks of peritonitis, pleuritis and synovitis and are at risk to die from reactive amyloidosis .Mutation-induced reduction in pyrin/ marenostrin activity is thought to underlie the disease by leading to NALP3 inflammasome activation ,and thereby to IL-1β related burst of inflammation. The IL-1 receptor antagonist Kineret (Anakinra), seems to be the most appropriate response to the uncontrolled IL-1β elevation. Indeed, an increasing number of reports over the last few years indicate a good response to Kineret (Anakinra), in colchicine-resistant FMF ,also in children ,however, no controlled study has thoroughly evaluated the efficacy and safety of this treatment. Study outline: The study aims to run at the FMF centre in Sheba Medical Center, covering more than 10,000 patients. The study will evaluate the effect of recombinant IL-1 receptor antagonist, Kineret (Anakinra), on the frequency of FMF attacks in patients that, despite maximum tolerable dose of colchicine, present with more than one attack per month. The study is designed as a randomised, placebo-controlled, double-blind study. 50 patients will be randomised to treatment with either Kineret (Anakinra), or placebo treatment for 4 months.

Familial Mediterranean fever (FMF) is a genetic disease resulting in recurrent attacks of fever, abdominal pain, chest pain, arthritis and rash. There are 5-15% of patients who continue to have FMF attacks despite treatment with colchicine or who cannot tolerate colchicine. Currently there are no alternatives to colchicine. Pyrin, the protein that has a defect in FMF has an important role in the regulation of a molecule called interleukin (IL)-1 beta production and activity. This molecule is very important in the process of inflammation in FMF. Therefore we propose to use IL-1 Trap (Rilonacept), a medication that binds and neutralizes IL-1. We will enroll in this study 17 subjects from the age of 4 years, including adults with active FMF despite colchicine therapy. Subjects will receive in random order two 3-month courses of Rilonacept at 2.2 mg/kg (maximum 160 mg) by weekly subcutaneous injection and two 3-month courses of placebo injection. If patients have at least two FMF attacks during a treatment course they will be able to get if they choose the other treatment until the end of that treatment course. Our hypothesis is that Rilonacept will decrease the number of acute FMF attacks and will be safe to use. This study may confirm the importance of IL-1 in the cause of FMF. Funding source - FDA Office of Orphan Products Development

Autoinflammatory diseases are illnesses characterized by episodes of inflammation that, unlike autoimmune disorders, lack the production of high titer autoantibodies or antigen-specific T cells. There is growing genetic and clinical evidence that Interleukin-1 (IL-1) plays a pathogenic role in several of these diseases. This exploratory study aims to examine the utility of the experimental drug candidate, IL 1 Trap (Regeneron Pharmaceuticals, Inc.) in the treatment of adult subjects with the autoinflammatory disorders Neonatal Onset Multisystem Inflammatory Disease (NOMID), Muckle-Wells Syndrome (MWS), and Familial Cold Autoinflammatory Syndrome (FCAS), Familial Mediterranean Fever (FMF), and adult Still's disease. FMF is associated with mutations in pyrin encoding MEFV. NOMID, MWS and FCAS are associated with mutations in cryopyrin-encoding CIAS1. This pilot study is designed to address: 1) the utility of IL 1 Trap in the treatment of subjects with diseases known to respond to IL-1 blockade (NOMID/MWS/FCAS) as shown by response to treatment with anakinra [Kineret]; 2) the response to IL-1 blockade of subjects with Adult Still's disease and colchicine-resistant FMF once the efficacy of IL-1 Trap has been established in NOMID/MWS/FCAS subjects; and 3) the biochemistry and genetics of autoinflammatory diseases and IL-1 related inflammation. IL-1 Trap is a recombinant fusion protein with picomolar affinity for IL-1 and a half-life of approximately 7.5 days in humans. This agent is currently in Phase 2 clinical studies for the treatment of rheumatoid arthritis and initial studies have shown activity against clinical and biochemical indicators of inflammation. Compared with anakinra, this agent may exhibit improved dosing convenience, potential for fewer injection site reactions, and improved efficacy due to the extremely high affinity of IL-1Trap for its target. In this study, biochemical, genetic, and clinical correlates of autoinflammatory disease will initially be measured at baseline following a withdrawal of any TNF or IL-1 inhibitor medications where applicable. Subjects will receive a course of therapy with IL-1 Trap that is predicted to provide an estimated 3-4 weeks of anti-inflammatory activity. Clinical, biochemical, and genetic correlates of inflammation will be measured at appropriate intervals to ascertain response and to further elucidate disease mechanisms. Subjects will be eligible, based on clinical response, to enter a 1- year extension phase with IL-1 Trap. Those subjects who complete the 1-year extension phase, and maintain improved clinical and laboratory parameters compared to baseline values, may continue to receive study medication at their current dose until the study drug is commercially available. Investigator comment: This protocol (from the NIH standpoint) is a continuation of the ongoing protocol 05-AR-0014, with a new change in study sponsor, the NIH replacing Regeneron as sponsor. this protocol therefore still contains background and procedural information that refer to patients with FMF and FCAS and or MWS and Still's disease, however only patients with Still's disease will be newly enrolled from this point on, enrollment for the FCAS and or MWS patients has already been completed and it has been decided to not enroll any more FMF patients because the number of subjects is too low to reach reasonable conclusions, in addition it has been difficult to recruit patients that are eligible. The background section and study procedures have largely been left as in the currently IRB approved protocol.

The purpose of study is to see if adding probiotics to corticosteroid treatment for children with PFAPA could improve the health and daily of patients through reduction in febrile period frequency and length, along with concomitant reduction of associated symptoms. Current standard of care incorporates the administration of corticosteroids; however, while limiting the symptoms associated with PFAPA, corticosteroid use has been shown to increase the frequency at which these symptoms occur. Investigators hypothesize that administration of probiotics along with corticosteroids will work to decrease the frequency at which the febrile episodes occur. Additionally, probiotics may decrease the maximal fever experienced during these episodes, amount of corticosteroid needed to control the symptoms, average length of the episodes, and the number of patients who ultimately undergo tonsillectomy due to unsuccessful treatment with medication.

A 12-week Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects with Familial Mediterranean Fever followed by an additional Open-label Extension Phase.

Colchicine is the drug of choice to treat patients with Familial Mediterranean Fever (FMF ), some of the patients treated with colchicine may suffer from gastrointestinal (GIT) adverse effect such as diarrhea and abdominal pain especially in the higher dose. 5-10% of the patients with FMF that have been treated with colchicine may have partial or no response to this therapy. Aim of our study: the aim of our study is :.1to evaluate the efficacy of probiotics in reducing the number of adverse effect in patients with FMF that are being treated with colchicine and suffering from GIT adverse effect. .2 To evaluate the efficacy of probiotics in reducing the number of FMF attacks in children with FMF that has been treated with colchicine with only partial response. Methods: the study will be done among children (5-17 years old) with FMF that are currently followed in the pediatric rheumatology clinic at Mayer children hospital Israel Haifa that are being treated with colchicine and suffering from either GIT adverse effect or partial response to colchicine. The study is design to be double blind placebo control, in the first 3 month patients will be with no therapy and will be required to record all there FMF episodes and their GIT adverse effect, in the second period the patients will be randomly divided into two groups 1.patients that will received placebo (group 1) and the 2. Probiotics group - patients will received probiotics (Bio -25 including 11 types of bacteria L.acidophilus, B.bifidum , L.rhamnosus, L.lactis, L.casei, B.breve, B.thermophilus, B.longum, L.paracseis, L.plantarum, B.infantis), both for three month, during this period patient will be required to record their gastrointestinal symptoms and other symptoms that may be related to FMF.

Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or crushing adult-dose tablets. An age-appropriate sprinkle formulation will allow for more accurate dosing in pediatric patients. The primary objective of this study is to evaluate and compare the steady-state pharmacokinetics of multiple oral doses of colchicine sprinkle capsules administered to pediatric and adult FMF patients. Secondary objectives include evaluation of the safety and tolerability of this regimen in pediatric and adult FMF patients and measurement of the levels of acute phase reactants (i.e, serum amyloid A [SAA], erythrocyte sedimentation rate [ESR], C-reactive protein [CRP]) at baseline and after dosing.

Behcet's disease (BD) is a chronic systemic inflammatory disease which is characterized by oral and genital ulcers, uveitis and skin lesions. Musculoskeletal involvement such as arthritis, arthralgia, enthesitis and sacroiliitis can be seen in the course of BD. Familial Mediterranean fever (FMF) is the most frequent autoinflammatory disease (AID) and characterized by selflimited episodes of fever and polyserositis. It is the most common monogenic periodic fever syndrome that affects mainly the people of Mediterranean descent. Regular physical activity involving aerobic and strengthening programs in inflammatory rheumatic diseases has a long-term anti-inflammatory effect. Eccentric exercises cause an acute increase in TNF alpha level and should be avoided especially at the beginning. It has been stated that patients should be encouraged about aerobic exercise programs where the intensity is gradually increased, starting with low intensity. Pilates method is the mind and body centering technique developed by Joseph Hubertus Pilates, based on providing lumbopelvic stability. FMF and BH have common characteristics such as ethnicity, etiopathogenetic mechanisms, symptoms, and treatment. When we look at the literature, there is no study on the effectiveness of exercise applications in FMF and Behçet patients. The purpose of our study; It is the comparison of the effectiveness of physical activity in patients diagnosed with Behçet and FMF and healthy individuals. In this context, with the short form of the International Physical Activity Evaluation Questionnaire, Behçet's patients whose physical activity level is inactive will be Group 1, FMF patients Group 2 and healthy controls with similar demographic characteristics will be Group 3. Pilates based exercise program will be applied to all 3 groups for 8 weeks, 2 times a week.

This study is Safety, Tolerability and Pharmacokinetic of Recombinant anti-IL-1β Humanized Monoclonal Antibody injection in Healthy Subjects. Pharmacokinetics, Pharmacodynamics and Anti-drug antibody (ADA) data will be collected; Drug safety, tolerability and immunogenicity for healthy subjects will be evaluated.