Safety and Tolerability of Rilonacept in Muckle-Wells Syndrome (MWS) or Schnitzler Syndrome (SchS) (NCT01045772) | Clinical Trial Compass
CompletedPhase 2
Safety and Tolerability of Rilonacept in Muckle-Wells Syndrome (MWS) or Schnitzler Syndrome (SchS)
Germany10 participantsStarted 2009-01
Plain-language summary
This 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
* Able to read, understand and willing to sign the informed consent form and abide with study procedures
* 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).
* In men: Willingness to utilize highly effective contraception and to not have their partner\[s\] become pregnant during the full course of the study
* All subjects will have received a normal Chest radiograph (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, if active tuberculosis is ruled out with appropriate measurements (e.g. PPD skin test, QuantiFERON-TB)
Exclusion Criteria:
* Treatment with a live (attenuated) virus vaccine during three months prior to visit 2
* Current or recent treatment (less than 5 half lives) with a TNF inhibitor
* Co…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety and tolerability parameters (including physical examination, PPD tuberculosis skin test, review of recent chest radiograph, electrocardiograms, routine safety laboratory assessments, clinical observations, vital signs, and adverse event reporting)