Biologics in refractory vasculitis
- Conditions
- Patients with relapsing or refractory non-ANCA-associated vasculitis (NAAV). Specific diseases to be included in this trial are: giant cell arteritis (GCA), Takayasu's arteritis (TA), polyarteritis nodosa (PAN), relapsing polychondritis, IgA vasculitis (of adults and children), cryoglobulinaemia, Cogan's syndrome and primary central nervous system (CNS) vasculitisCirculatory System1. Other giant cell arteritis2. Polyarteritis nodosa and related conditions3. Relapsing polychondritis4. Cryoglobulinaemia5. Interstitial and deep keratitis
Recruitment & Eligibility
- Status
- Ongoing
- Sex
- All
- Target Recruitment
- 140
1. Aged at least 5 years
2. Have given, or their parent/legal guardian aged =16 years old has given, written informed consent
3. Diagnosis of NAAV
4. Refractory disease defined by:
4.1. Active disease, BVAS v3/ PVAS with = 1 severe (new/worse) or = 3 non-severe (new/worse) items despite 12 weeks of conventional therapy prior to screening visit OR
4.2. Inability to reduce prednisolone below 15 mg/day or (0.2 mg/kg/day in case of children) without relapse in the 12 weeks prior to screening visit
1. Previous treatment failure/contraindication to =2 trial IMPs
2. Increase in the dose or frequency of background immunosuppressive (e.g. methotrexate) or anti-cytokine therapy within 30 days of screening visit
3. Use of intravenous immunoglobulins within 30 days, or cyclophosphamide or lymphocyte depleting biologic (e.g. rituximab) within 6 months of screening visit
4. Have an active systemic bacterial, viral or fungal infection, or tuberculosis
5. Hepatitis B (HB) core antibody (Ab) or HB surface antigen-positive or hepatitis C antibody positive or human immunodeficiency virus (HIV) antibody test positive
6. History of malignancy within five years prior to screening visit or any evidence of persistent malignancy, except fully excised basal cell or squamous cell carcinomas of the skin, or cervical carcinoma in situ which has been treated or excised in a curative procedure
7. Pregnant or breastfeeding (Section 11.9)
8. Severe disease, which in the opinion of the physician prevents randomization to placebo
9. Recent or upcoming major surgery within 45 days of screening visit
10. Leukocyte count < 3.5 x 109 cells/l, platelet count < 100 x 109 cells/l, neutrophil count of < 1 x 109 cells/l
11. ALT or ALP > 3 times the upper limit of normal
12. Symptomatic congestive heart failure (NYHA class III/IV) requiring prescription medication within 90 days of screening visit
13. Demyelinating disorders
14. History or presence of any medical condition or disease which, in the opinion of the Investigator, may place the participant at unacceptable risk because of trial participation
15. Administration of live or live-attenuated vaccines within 45 days of screening
16. Have received an investigational medicinal product (IMP) within 5 half-lives or 30 days prior to screening
17. Diagnosis of adenosine deaminase type 2 (DADA2)
18. Hypersensitivity to the active IMP substance or to any of the formulation excipients
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method