A Single-Centre, Open Label Study of the Safety and Tolerability of Rilonacept in Subjects Living in Germany with Muckle-Wells Syndrome (MWS), a Cryopyrin-Associated Periodic Syndrome, or Schnitzler’s Syndrome (SchS). - ACCILTRA1
- Conditions
- 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.
- Registration Number
- EUCTR2006-004290-97-DE
- Lead Sponsor
- Charité Universitätsmedizin Berlin, Department of Dermatology and Allergy
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- Not specified
- 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 < 1). A woman will be considered not of childbearing potential if she is post-menopausal for greater than two years or surgically sterile (bilateral tubal ligation, bilateral oophor- ectomy or hysterectomy).
- In men: Willingness to utilize highly effective contraception and to not have their partner[s] become pregnant during the full course of the study. Adequate contraceptive measures include oral contraceptives (stable use for two or more cycles prior to screening); bilateral tubal ligation; vasectomy; condom or diaphragm plus either contraceptive sponge, foam or jelly.
- All subjects will have received a normal Chest X-Ray (CXR) within 6 months prior to enrollment (signing of consent) which notes the absence of calcified granulomas and/or pleural scarring consistent with tuberculosis.
- Subjects are considered eligible according to the following tuberculosis (TB) screening criteria:
*have no history of latent or active TB prior to screening,
*have no signs or symptoms suggestive of active TB,
*have no recent close contacts with a person with active TB,
*have a 5 tuberculin units (TU) of standard purified protein derivative (PPD) skin test of less than 5 mm of induration unless the patient had been previously vaccinated with BCG and a more specific test for mycobactium tuberculosis (e.g., ELISpot plus T-SPOT or QuantiFERON-TB) is negative,
- if PPD test is positive ( > 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
- 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 <3.6 x 103/mm3; platelet count <150,000 mm3; ALT or AST >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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method