Schnitzler’s syndrome (SchS) MedDRA version: 19.1 Level: PT Classification code 10062908 Term: Schnitzler's syndrome System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Trade Name: RoActemra
Product Name: RoActemra
Product Code: Ro 487-7533/F10
Pharmaceutical Form: Solution for injection
Sponsors
Charité - Universitätsmedizin Berlin; Dpt. of Dermatology and Allergy
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Adults (18 years or older) SchS diagnosis based on Strasbourg clinical criteria Active SchS, refractory to treatment with antihistamines, NSAIDS or colchicine, hydroxychloroquine or dapsone Patients who have a symptom score (PGA) of at least 8 (0-20) at baseline If necessary, concurrent/ongoing treatment with a stable dose of systemic corticosteroids not greater than 10mg/d for 14 days prior to screening If necessary, concurrent/ongoing treatment with a stable dose of antihistamines and NSAIDs for 7 days prior to screening Able to read, understand and willing to sign the informed consent form and abide with study procedures Willing, committed and able to return for all clinic visits and complete all study-related procedures, including willingness to have SC injections administered by a qualified person In females of childbearing potential: Negative pregnancy test within 28 days of randomization; males and females willing to use highly effective contraception (Pearl-Index =65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: Major surgery (including joint surgery) within 8 weeks prior to screening or planned major surgery within 6 months following randomization. Concurrent/ongoing treatment with biologics or recent treatment (less than 5 half lives) o With Anakinra within 7 days prior to screening, with canakinumab within 100 days prior to screening o with oral/parental corticosteriods greater than 10 mg/d within 2 weeks prior to screening o with Cyclosporin A Methotrexate, Dapsone, Chloroquine, Hydroxychloroquine, Azathioprine, Cyclophosphamide within 4 weeks prior to screening o other immunosuppressives within 4 weeks or 5 half lives prior to screening, whichever is longer o Previous treatment within six months of randomization with any cell-depleting therapies, including investigational agents or approved therapies, some examples include: CAMPATH, anti-CD4, anti-CD5, anti-CD3, anti-CD19 and anti-CD20. o Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of baseline. Treatment with a live (attenuated) virus vaccine within 4 weeks prior to Baseline visit Significant medical condition rendering the patient immunocompromised or not suitable for a clinical trial Any previous treatment with alkylating agents such as chlorambucil, or with total lymphoid irradiation. History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies. An abnormal chest radiograph consistent with clinical signs of prior or present tuberculosis infection whether or not previously treated with anti-tuberculosis agents 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 Evidence of current HIV, Hepatitis B, or Hepatitis C infection by clinical or serological history Presence of any of the following laboratory abnormalities at enrollment visit: serum creatinine > 1.6 mg/dL (141 µmol/L) in female patients and > 1.9 mg/dL (168 µmol/L) in male patients, WBC 2 x ULN or total bilirubin >ULN, Hemoglobin <8.0 g/dL, neutrophil count <2,000 cells/µl or lymphocyte count <500/ µl Evidence of active, recurrent or latent systemic infection Active systemic inflammatory condition other than SchS including, but not limited to, rheumatoid arthritis History of malignancies within five years prior to screening other than a successfully treated non-metastatic cutaneous, basal, or squamous cell carcinoma and/or in situ cervical cancer Lactating females or pregnant females 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the effect of TCZ on the clinical signs and symptoms of SchS; Secondary Objective: To assess the effect of TCZ on inflammation markers (CRP, ESR, SAA, S100A12) To assess the effect of TCZ on the patient’s quality of life To assess the safety and tolerability following administration of TCZ to patients with SchS ;Primary end point(s): Change in the investigator’s assessment of total disease activity (PGA) between baseline and TCZ treatment;Timepoint(s) of evaluation of this end point: Week 20 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Proportion of patients with complete response (based on physician’s global assessment with no or minimal overall autoinflammatory disease activity and CRP </= 10 mg/l) at week 20 Change in the patient based Schnitzler Activity Score (SchAS) during the treatment period (The SchAS combines the key symptoms of SchS). Change in inflammation markers (CRP, ESR, SAA, S100A12) Change in the patient’s quality of life (assessed by the Dermatology Life Quality Index and SF-36) ;Timepoint(s) of evaluation of this end point: Week 20 | — |
Countries
Germany
Contacts
Public ContactKaroline Krause
Charité - Universitätsmedizin Berlin; Dpt. of Dermatology and Allergy
Outcome results
None listed