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A Phase II, single center open label, prospective trial to evaluate the efficacy and safety of mepolizumab for patients with refractory or relapsing Churg Strauss Syndrome - MEPOCHUSS

Conditions
Churg-Strauss-Syndrome
MedDRA version: 9.1Level: LLTClassification code 10009164Term: Churg Strauss syndrome
Registration Number
EUCTR2006-001791-20-DE
Lead Sponsor
niversity Hospital of Schleswig-Holstein (UKSH), Campus Lübeck
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

1. informed consent

2. documented history of Churg Strauss Syndrome:
a. previous or currently active vasculitis either on biopsy or based on the following surrogate parameters proposed by the EMEA group on the classification of primary systemic vasculitides for epidemiological studies [33]: dysmorphic red cells or erythrocyte casts indicative of glomerulonephritis, rapidly progressive neuropathy, alveolar hemorrhage demonstrated on bronchoalveolar lavage, episcleritis.
b. In agreement with recommendations of the EMEA working group on the classification of primary systemic vasculitides for epidemiological studies the criteria of the American College of Rheumatology are used for classification of Churg Strauss Syndrome, as in contrast to other criteria (Lanham, CHCC) the ACR criteria are data-driven and not based on expert opinion alone. Four or more of the seven (ACR) for classification of Churg-Strauss syndrome need to be fulfilled (see [34] for definitions) in order to yield a sensitivity of 85% and specificity of 99.7 %:
i. Asthma
ii. Eosinophilia > 10 % on peripheral blood white cell count
iii. History of allergy
iv. Mononeuropathy or Polyneuropathy
v. Pulmonary infiltrates, non-fixed
vi. Paranasal sinus abnormality
vii. Extravascular eosinophils on tissue biopsy

3. active disease defined as either
a. active vasculitis (BVAS > 3) and/or
b. symptomatic tissue eosinophilia and
c. a prednisolone demand of >- 12.5 mg/day to control disease

4. Subjects must complete screening and baseline assessment as outlined below

5. stable corticosteroid dose of > 12.5 mg prednisolone for at least one week

6. treatment with cyclophosphamide (pulse or daily oral) or methotrexate in a stable dose for at least 4 weeks

7. Male or female, 18-80 years of age. A female patient may be included only if
i. She is not pregnant or nursing
ii. Of non-childbearing potential
iii. Are of child-bearing potential but have a negative pregnancy test and agree to practice birth control

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

1. Life threatening disease or other critical illness deemed inappropriate for inclusion in the study by the principal investigator. Among others, the following manifestations of CSS are considered potentially life-threatening:
a. severe alveolar hemorrhage requiring blood transfusions or mechanical respiratory support
b. rapid progressive glomerulonephritis requiring dialysis
c. severe gastrointestinal involvement (i.e. gangrene, GI-bleeding requiring transfusions)
d. severe CNS involvement (i.e. ischemic stroke)
e. severe cardiac involvement (i.e. life-threatening arrhythmias or cardiac failure as outlined below)

2. Treatment with other immunosuppressive agents within 4 weeks prior to randomisation. Leflunomide must have been washed out according to current guidelines (cholestyramine for 11 days) before entry of the study. MTX or CYC are allowed if their dosage has not been changed within the last 4 weeks.

3. Corticosteroid pulse of > 60 mg within the last three weeks prior to randomisation.

4. known secondary cause of eosinophilia: drug eruption, parasitic infection, HIV infection, history of graft versus host disease, acute/chronic eosinophilic leukemia,

5. no history or clinical features of vasculitis, suggesting a diagnosis of hypereosinophilic syndrome rather than Churg Strauss Syndrome

6. Diagnosis of other primary systemic vasculitis: Wegener’s granulomatosis, microscopic polyangiitis etc.

7. currently active malignant disease

8. abnormal laboratory values:
a. serum creatinine >- 3 times institutional upper normal limit (UNL)
b. AST or ALT > UNL
c. Platelet count < 50.000/µL

9. Impaired cardiac function defined as:
a. Left ventricular ejection fraction < 20 %
b. NYHA class IIIb or IV
c. Myocardial infarction

10. history of allergic reaction due to monoclonal antibodies

11. prior treatment with anti-hIL-5 monoclonal antibody

12. exposure to investigational drug within 30 days prior to randomisation

13. positive pregnancy test

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: Induction of remission, defined as BVAS = 0 and a daily prednisolone dose of -< 7.5 mg.;Secondary Objective: - Change in BVAS score <br>- Change in Disease Extent Index (DEI) score <br>- Permanent End organ damage assessed by the Vasculitis Damage Index (VDI) <br>- Time to remission<br>- Response, defined* as a 50 % reduction of the BVAS score<br>- Time to response<br>- The frequency of relapses, defined* as as a BVAS score of >- 1 in patients who had previously attained remission<br>- Blood eosinophil count<br>- Frequency of all AEs and SAEs<br><br>*according to consensus definitions of the EUVAS/EULAR Working group on recommendations for conducting clinical trials in systemic vasculitis<br>;Primary end point(s): Attaining remission (BVAS Score = 0 and prednisolone dose of -< 7.5 mg/d).
Secondary Outcome Measures
NameTimeMethod
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