A Study to Investigate the Safety and Efficacy of an Anti-IFN-gamma mAb in Children With Systemic Juvenile Idiopathic Arthritis (sJIA) Developing Macrophage Activation Syndrome/ Secondary Hemophagocytic Lymphohistiocytosis (MAS/sHLH)
| Status: | Recruiting |
|---|---|
| Conditions: | Arthritis, Endocrine |
| Therapuetic Areas: | Endocrinology, Rheumatology |
| Healthy: | No |
| Age Range: | Any - 18 |
| Updated: | 3/23/2019 |
| Start Date: | March 2, 2018 |
| End Date: | May 2021 |
| Contact: | Maria Ballabio, MD |
| Email: | mballabio@novimmune.com |
| Phone: | +41 61 201 1324 |
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)
Macrophage Activation Syndrome (MAS) is a rare, life-threatening condition characterized by
uncontrolled hyperinflammation which may develop on the background of systemic Juvenile
Idiopathic Arthritis (sJIA). NI-0501 (Emapalumab) is a monoclonal antibody neutralizing
interferon-gamma (IFN-gamma), a key cytokine which contributes to the inflammation and tissue
damage seen in MAS. The purpose of this study is to assess the safety, tolerability and
efficacy of NI-0501 in sJIA patients developing MAS, presenting an inadequate response to
high dose glucocorticoid treatment.
uncontrolled hyperinflammation which may develop on the background of systemic Juvenile
Idiopathic Arthritis (sJIA). NI-0501 (Emapalumab) is a monoclonal antibody neutralizing
interferon-gamma (IFN-gamma), a key cytokine which contributes to the inflammation and tissue
damage seen in MAS. The purpose of this study is to assess the safety, tolerability and
efficacy of NI-0501 in sJIA patients developing MAS, presenting an inadequate response to
high dose glucocorticoid treatment.
Inclusion Criteria:
- Patients of both genders, aged <18 years.
- Confirmed sJIA or high presumption of sJIA.
- Diagnosis of active MAS.
- Patient presenting an inadequate response to high dose i.v. glucocorticoid treatment.
- 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.
Exclusion Criteria:
- Diagnosis of suspected or confirmed primary HLH or HLH consequent to a neoplastic
disease.
- Patients treated with Tocilizumab, Canakinumab or TNF inhibitors within 5 times of
their defined half-life.
- 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.
We found this trial at
2
sites
Cincinnati, Ohio 45229
Principal Investigator: Alexei Grom, MD
Phone: 513-636-4676
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Genova, 16147
Principal Investigator: Angelo Ravelli, MD
Phone: +3901056362578
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