NI-0501-06
Research type
Research Study
Full title
A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of NI-0501, an anti-interferon gamma (anti-IFNγ) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary HLH (MAS/sHLH)
IRAS ID
223831
Contact name
Paul Brogan
Contact email
Sponsor organisation
Novimmune SA
Eudract number
2016-004223-23
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Macrophage Activation Syndrome (MAS) is a severe, potentially life-threatening, complication of several chronic rheumatic diseases of childhood. It occurs most commonly with systemic juvenile idiopathic arthritis (sJIA). The immune system is the body’s natural defence system to fight infection. There are several groups of cells that work together and send signals to each other to fight against infections (some of these cells are called Macrophages and T Lymphocytes). In individuals with MAS, these cells within the immune system are uncontrolled and work in overdrive, leading to too much inflammation in the entire body. This inflammation can be in any organ system and can result in organ damage.\n\nBecause MAS is potentially fatal, a timely diagnosis and immediate therapeutic interventions are essential for appropriate management of the disease. The reported mortality rates in MAS reach 20-30%, and it remains the major source of mortality in paediatric rheumatology. There are currently no approved medications for the treatment of MAS.\n\nThis is an open-label, single arm, international, multicentre pilot phase 2 study which will investigate how safe, effective and well tolerated NI-0501 is in controlling sJIA developing into MAS. Participants of both sexes, aged from 0 to 18 will be recruited into this study. The study will be conducted in patients who are partly hospitalised and then provided outpatient care if patients health condition allows. Participation will last for 8 weeks [4 weeks of study medication dosing and 4 weeks of follow up].
REC name
London - Hampstead Research Ethics Committee
REC reference
17/LO/0976
Date of REC Opinion
13 Sep 2017
REC opinion
Further Information Favourable Opinion