A study to investigate a new medication, NI-0501, in children with the disease systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary Hemophagocytic Lymphohistiocytosis (MAS/sHLH)

Phase 1

• Conditions: Macrophage activation syndrome / Secondary hemophagocytic lymphohistiocytosis (MAS/sHLH) in patients with Systemic Juvenile Idiopathic Arthritis (sJIA); MedDRA version: 19.1Level: LLTClassification code 10053867Term: Macrophage activation syndromeSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2016-004223-23-IT
• Lead Sponsor: ovimmune SA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-01-25
• Last Update Posted: 19 March 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

•Patients of both genders, aged <18 years
•Confirmed sJIA or high presumption of sJIA.
•Diagnosis of active MAS confirmed by the treating rheumatologist
•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
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified