A study to investigate a new medication, emapalumab, in children with the disease systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) developing Macrophage Activation Syndrome/secondary Hemophagocytic Lymphohistiocytosis (MAS/sHLH)
- Conditions
- Macrophage activation syndrome / Secondary hemophagocytic lymphohistiocytosis (MAS/sHLH) in patients with Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD)MedDRA version: 21.1Level: LLTClassification code 10053867Term: Macrophage activation syndromeSystem Organ Class: 100000004870Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2016-004223-23-GB
- Lead Sponsor
- Swedish Orphan Biovitrum AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 12
•Patients of both genders.
•Confirmed sJIA or high presumption of sJIA for patients presenting with MAS in the context of the onset of sJIA.
•AOSD patients: confirmed AOSD diagnosis as per Yamaguchi criteria
•Diagnosis of active MAS confirmed by the treating rheumatologist
•Patient presenting an inadequate response to high dose i.v. glucocorticoid treatment
•Tocilizumab, TNF inhibators and canakinumab, if administered, have to be discontinued before emapalumab initiation
•Informed consent provided by the patient (as required by local law), or by the patient’s legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•Diagnosis of suspected or confirmed primary HLH or HLH consequent to a neoplastic disease.
•Active mycobacteria (typical and atypical), Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.
•Clinical suspicion of latent tuberculosis.
•Positive serology for HIV antibodies.
•Presence of malignancy.
•Receipt of a BCG vaccine within 12 weeks prior to screening.
•Receipt of live or attenuated live vaccines (other than BCG) within 6 weeks prior to screening.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method