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Active clinical trials for "Hereditary Autoinflammatory Diseases"

Results 11-20 of 40

Assessment of microRNAs Role in Familial Mediterranean Fever FMF Pathophysiology

Familial Mediterranean Fever

Familial Mediterranean Fever (FMF) genetic diagnosis is well established for homozygous patients. On the other hand, although heterozygous individuals are theoretically healthy carriers, 1/3 of them will develop clinical symptoms of FMF and could benefit from prophylactic treatment. This suggests that the disorder expression mechanisms are not fully elucidated to date. The preliminary results obtained at the Institute for Regenerative Medicine and Biotherapy (IRMB) suggest the involvement of an epigenetic mechanism in FMF pathogenesis, and our laboratory has strong arguments as to the involvement of microRNAs (in particular miR-326) which are negative regulators of gene expression. This study is exploratory and aims to validate the role of miRNAs in the clinical expression of FMF in patients, thus to explore the epigenetic mechanisms that may explain the variability of expression of this disorder.

Not yet recruiting8 enrollment criteria

Clinical and Molecular Evaluation of Childern With Familial Meditterranean Fever and Their Siblings...

Familial Mediterranean Fever

Familial Mediterranean fever (FMF ,recurrent polyserositis ,periodic disease) is an autosomal recessive auto inflammatory disease which primarily affect population surrounding the Mediterranean basin (Arabs , Turks ,Armenians, Jews ).Despite its striking symptoms pattern FMF was first described as distinct entity only in 1945. It is characterized by recurrent attacks of fever , peritonitis ,pleurisy , arthritis , or erysipelas like skin disease. The most dangerous complication of this disease is secondary amyloidosis . FMF diagnosis is mainly clinical, and the genetic testing is indicated to support it . Uncommonly, amyloidosis may develop in individuals carrying two Familial Mediterranean fever gene (MEFV ) mutations without overt clinical symptoms of FMF, a condition designated as phenotype II. Furthermore, two MEFV mutations may be harbored without signs or symptoms of FMF nor of reactive amyloidosis. This 'silent' homozygous or compound heterozygote state is termed phenotype III.

Not yet recruiting9 enrollment criteria

Heat Intolerance in the Group of FMF Patients

Familial Mediterranean FeverHeat Intolerance1 more

There now causation between Heat Intolerance and FMF that were showed in studies till now. We suggest that the prevalence of Heat Intolerance in the group of the FMF patients will be significantly higher than in the group of healthy individuals, that participated in the study of Heller Institute of Medical Research. The aim of the study is verification of causation between these pathologies. The information obtained by the study may allow us to determine the sequence of events associated with FMF attack development, and perhaps take us one step further in the understanding of the pathogenesis of the disease. 15 FMF patients with double mutations MEFV, mail sex, from the age from 18 to 30 without attacks during not less than 2 month will participate in the study. To identify an individuals susceptibility to exercise heat test, a Heat Tolerance Test (HTT) will perform, according to HTT Protocol of Heller Institute of Medical Research.

Enrolling by invitation6 enrollment criteria

Efficacy and Safety of Canakinumab in Patients With Colchicine Resistant Familial Mediterranean...

Familial Mediterranean Fever

Establish the safety and efficacy of 3 months treatment with canakinumab in patients with colchicine resistant Familial Mediterranean Fever.

Completed17 enrollment criteria

Tocilizumab for the Treatment of Familial Mediterranean Fever

Familial Mediterranean Fever

Adult patients with Familial Mediterranean Fever, who have active disease

Completed56 enrollment criteria

The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With...

Familial Mediterranean Fever

It was aimed to examine the efficacy and safety of once daily dosage schema of colchicine in pediatric patients with FMF compared to twice daily dosage schema. In this 24-week, multicentric, randomized, controlled, noninferiority trial, pediatric patients newly diagnosed with FMF, carrying homozygote or compound heterozygote mutation and did not receive any treatment, were included. Patients were randomly assigned using block randomization method to receive treatment with once or twice daily doses. Clinical and laboratory characteristics and medication side effects were recorded and compared between groups. The study complied with Good Clinical Practice and the Consolidated Standards for Reporting of Trials (CONSORT) statement.

Completed13 enrollment criteria

Study of Efficacy and Safety of Canakinumab in Patients With Hereditary Periodic Fevers

Hereditary Periodic Fevers

This study is to determine whether canakinumab is able to induce and maintain a clinically meaningful reduction of disease activity in participants with Hereditary Periodic Fevers (HPF) compared to placebo.

Completed0 enrollment criteria

Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant...

Colchicine Resistant/Intolerant Familial Mediterranean Fever

A study designed to evaluate the role of treatment with a biological agent - Canakinumab in pediatric (age 4-20) Familial Mediterranean Fever (FMF) patients that are intolerant or resistant for colchicine treatment. The study hypothesis is that Canakinumab will reduce attack frequency and severity.

Completed23 enrollment criteria

A Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic...

Systemic Juvenile Idiopathic ArthritisHereditary Periodic Fevers

The objective of this extension protocol is to collect safety data (serious and non-serious adverse events) and to provide continuous canakinumab to patients in France who completed study CACZ885G2301E1(NCT00891046), CACZ885G2306 (NCT02296424) or CACZ885N2301 (NCT02059291) until a decision regarding reimbursement in France is effective for canakinumab (Ilaris®) in these indications.

Completed11 enrollment criteria

Efficacy and Safety Study of ACZ885 in Patients With Active Recurrent or Chronic TNF-receptor Associated...

TNF-receptor Associated Periodic Syndromes (TRAPS)

This trial will assess the safety and efficacy of ACZ885 in patients with active recurrent or chronic TNF-receptor associated periodic syndrome (TRAPS).

Completed22 enrollment criteria

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