Muckle-Wells SyndromePhase 2ACCILTRA1NCT01045772
Safety and Tolerability of Rilonacept in Muckle-Wells Syndrome (MWS) or Schnitzler Syndrome (SchS)
A Phase 2 study testing rilonacept in people with Muckle-Wells Syndrome and Schnitzler Syndrome, run by Charite University, Berlin, Germany at 1 site in Germany.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Muckle-Wells Syndrome, Schnitzler Syndrome
- What is being tested?
- rilonacept
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2009to Sep 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
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- 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)
Who cannot join
- 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
- 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, lymphatic or hematological disease that would adversely affect the subject's participation or evaluation in this study
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- Active systemic inflammatory condition including, but not limited to, rheumatoid arthritis, systemic lupus erythematosis, polymyalgia rheumatics, 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 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
- Enrollment in another investigational treatment or device study or use of an investigational agent, or no completion of 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
- Present 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
- Subjects who are detained officially or legally to an official institute
- Deafness
- Dementia due to cerebral amyloidosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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 (CAPS), or Schnitzler Syndrome (SchS)
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).
Read more
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.
- Conditions
- Muckle-Wells Syndrome, Schnitzler Syndrome
- Being tested
- rilonacept
- Keywords
- Schnitler syndrome, Muckle-Wells syndrome, anti-IL-1
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes, Schnitzler Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rilonaceptDrug
Also called Arcalyst
160mg of rilonacept 1x/week
| Study group | Role | What they receive |
|---|---|---|
| IL-1 trap | Experimental | Not described Receives: Drug: rilonacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 1 year
Secondary outcomes (1)
Change of the mean MWAS (Muckle-Wells Activity Score) and SCHAS (Schnitzler Activity Score) from the 21-day baseline phase (day -21 to day 0) to the last 21 days of the first 4 weeks initial treatment phase (day 7 to 28) of the study.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2010 (estimated)
- Study startJan 2009
- Primary completionSep 2010
- Study completionSep 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muckle-Wells SyndromeStudies of the same condition, open and under way.
- More trials run by Charite University, Berlin, GermanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Charite University, Berlin, Germany
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Karoline Krause, Charite University, Berlin, Germany
- Investigators
- Marcus Maurer, MD, Principal investigator, Allergie-Centrum-Charité, Charité University Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01045772
- Sponsor's protocol ID
- ACCILTRA1
- Other IDs
- EudraCT Number: 2006-004290-97
- Acronym
- ACCILTRA1
- Status last verified
- May 2012
- First submitted
- 7 Jan 2010
- Last updated
- 31 May 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Krause K, Weller K, Stefaniak R, Wittkowski H, Altrichter S, Siebenhaar F, Zuberbier T, Maurer M. Efficacy and safety of the interleukin-1 antagonist rilonacept in Schnitzler syndrome: an open-label study. Allergy. 2012 Jul;67(7):943-50. doi: 10.1111/j.1398-9995.2012.02843.x. Epub 2012 May 15.
PubMed 22583335
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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 (CAPS), or Schnitzler Syndrome (SchS)
- ClinicalTrials.gov ID
- NCT01045772
- Acronym / sponsor ID
- ACCILTRA1 / ACCILTRA1
- Conditions
- Muckle-Wells Syndrome, Schnitzler Syndrome
- Keywords
- Schnitler syndrome, Muckle-Wells syndrome, anti-IL-1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Charite University, Berlin, GermanyUniversity, hospital or other
- Study start
- Jan 2009
- Primary completion
- Sep 2010 (actual)
- Study completion
- Sep 2010 (actual)
- First posted
- 11 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IL-1 trap | Experimental | Drug: rilonacept |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (21 listed)
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rilonaceptAlso: Arcalyst160mg of rilonacept 1x/week | Drug | IL-1 trap |
Primary outcome
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)
Time frame: 1 year
1 secondary outcome
Change of the mean MWAS (Muckle-Wells Activity Score) and SCHAS (Schnitzler Activity Score) from the 21-day baseline phase (day -21 to day 0) to the last 21 days of the first 4 weeks initial treatment phase (day 7 to 28) of the study.
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2012.
- Derived from this study: PubMed 22583335Krause K, Weller K, Stefaniak R, Wittkowski H, Altrichter S, Siebenhaar F, Zuberbier T, Maurer M. Efficacy and safety of the interleukin-1 antagonist rilonacept in Schnitzler syndrome: an open-label study. Allergy. 2012 Jul;67(7):943-50. doi: 10.1111/j.1398-9995.2012.02843.x. Epub 2012 May 15.
Related trials for Muckle-Wells Syndrome
RecruitingPhase 1
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
Cryopyrin-Associated Periodic Syndromes, Amyloidosis
Recruiting
Circadian Rhythm Deregulation in Patients With CAPS
Cryopyrin Associated Periodic Syndrome, Familial Cold Urticaria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Marcus Maurer, MD principal investigator
Allergie-Centrum-Charité, Charité University Berlin, Germany
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rilonacept in people with Muckle-Wells Syndrome and Schnitzler Syndrome, run by Charite University, Berlin, Germany at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01045772, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2012.