The medical condition to be investigated was first described by Muckle and Wells 1962. The Muckle-Wells syndrome (MWS) is rare and characterized by chronic recurrent urticaria, periodic arthritis, sensorineural deafness, general signs of inflammation and secondary amyloidosis. Schnitzler syndrome (SchS) is rare and characterized by chronic, nonpruritic urticaria and a monoclonal immunoglobulin M gammopathy. Symptoms are recurrent fever, bone pain, muscle pain, arthralgia or arthritis.
Conditions
Interventions
Trade Name: Rilonacept
Pharmaceutical Form: Solution for injection
Sponsors
Charité Universitätsmedizin Berlin, Department of Dermatology and Allergy
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Adult (18 years or older) - Symptomatic MWS diagnosis based on family history of MWS and evidence of a genetic mutation in CIAS1, or Symptomatic SchS diagnosis based on diagnostic criteria as defined in Appendix 4 - Able to read, understand and willing to sign the informed consent form and abide with study procedures. Written informed consent includes permission to confirm mutation in CIAS1 gene via DNA sequence analysis (only MWS subjects). - Able to read, understand and complete study-related questionnaires (subjects must complete their diaries for = 11 of the 21 days prior to Visit 2) - Willing, committed and able to return for all clinic visits and complete all study-related procedures, including willingness to self-administer SC injections or to have SC injections administered by a qualified person. - In females of childbearing potential: Negative pregnancy test; males and females willing to use highly effective contraception (Pearl-Index or equal to 5 mm induration), and active TB has been ruled out, the patient is still eligible if appropriate treatment for latent TB has been initiated either prior to or simultaneously with the first administration of study drug. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Treatment with a live (attenuated) virus vaccine during three months prior to Baseline visit (Visit 2) - Current or recent treatment (less than 5 half lives) with a TNF inhibitor - Concurrent/ongoing treatment with Anakinra (Kineret) - An abnormal chest radiograph consistent with clinical signs of prior or present tuberculosis infection whether or not previously treated with anti-tuberculosis agents - A history of listeriosis, active tuberculosis, persistent chronic or active infection(s) requiring treatment with parenteral antibiotics, parenteral antivirals, or parenteral antifungals within four weeks, or oral antibiotics, oral antivirals, or oral antifungals within four weeks prior to the Screening visit - Significant concomitant illness such as, but not limited to, cardiac, renal, neurological, endocrinological, metabolic, or lymphatic disease that would adversely affect the subject’s participation or evaluation in this study - Active systemic inflammatory condition including, but not limited to, rheumatoid arthritis, systemic lupus erythematosis, polymyalgia rheumatica, vasculitis, or myositis - History of fibromyalgia or chronic fatigue syndrome - Evidence of current HIV, Hepatitis B, or Hepatitis C infection by clinical or serological history - History of malignancies including malignant hematological disorders other than a successfully treated non-metastatic cutaneous, basal, or squamous cell carcinoma and/or in situ cervical cancer within five years of the Screening visit - History of a demyelinating disease or multiple sclerosis - Severe respiratory disease, including, but not limited to severe bronchiectasis, chronic obstructive pulmonary disease, bullous lung disease, uncontrolled asthma, or pulmonary fibrosis - Known hypersensitivity to Chinese hamster ovary (CHO) cell derived therapeutics or proteins or any components of rilonacept - Presence of any of the following laboratory abnormalities at enrollment visit: creatinine >1.5 x Upper Limit of Normal (ULN), WBC 2.0 x ULN - Lactating females or pregnant females - Males not willing to use highly effective contraception - Enrollment in another investigational treatment or device study or use of an investigational agent, or less than 4 weeks or 5 half-lives, whichever is longer, since end of another investigational device or drug trial - Subjects for whom there is concern about compliance with the protocol procedures - Any medical condition which, in the opinion of the Investigator, would interfere with participation in the study or place the subject at risk - History of substance abuse (drug or alcohol) or any other factor (e.g., serious psychiatric condition) that could limit the subject’s ability to comply with study procedures - Deafness - Dementia due to cerebral amyloidosis - Persons who are detained officially or legally to an official institute
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the tolerability and safety of rilonacept in subjects with MWS/ SchS;Secondary Objective: To assess the effects of rilonacept on the clinical signs and symptoms of MWS, and/or SchS To assess the effects of rilonacept on laboratory measures such as acute phase reactants, ESR (erythrocyte sedimentation rate) and SPE (Serum Protein Electrophoresis) To assess the effects of rilonacept on the clinical signs and symptoms of MWS and SchS when used for long term treatment ;Primary end point(s): Change in the mean MWAS / SCHAS (Muckle-Wells Activity Score / Schnitzler Activity Score) from the baseline phase (day -21 to day 0) to the last 21 days of the first 4 weeks treatment phase (day 7 to 28) of the study. - The MWAS combines the five key symptoms of MWS, i.e. rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue - The SCHAS combines five symptoms, i.e. urticarial rash, periodic fever, joint pain, bone/muscle pain and fatigue - Daily MWAS and SCHAS values will be documented using DHAFs (Daily Health Assessment Forms) - Values for the Disease Activity Scores (DAS) can range from 0 to 50 per day (5 symptoms, 0-10 each) Mean daily DAS will be calculated by dividing the daily activity scores by 5. | — |
Countries
Germany
Outcome results
None listed