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)

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-IFN?) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) developing Macrophage Activation Syndrome/secondary HLH (MAS/sHLH)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-004223-23-IT

Enrollment

Registered

2017-01-25

Start date

2017-04-14

Completion date

Unknown

Last updated

2018-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Macrophage activation syndrome / Secondary hemophagocytic lymphohistiocytosis (MAS/sHLH) in patients with Systemic Juvenile Idiopathic Arthritis (sJIA) MedDRA version: 19.1 Level: LLT Classification code 10053867 Term: Macrophage activation syndrome System Organ Class: 100000004851

Interventions

Product Code: NI-0501 Pharmaceutical Form: Concentrate for solution for infusion

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Patients of both genders, aged =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: • To describe the pharmacokinetic profile of NI-0501 in sJIA patients with MAS •To confirm the proposed dosing regimen of NI-0501 •To evaluate the safety and tolerability profile of intravenous administrations of NI-0501 •To preliminary assess the efficacy of NI-0501 •To assess the levels of relevant biomarkers •To assess the immunogenicity of NI-0501;Secondary Objective: Not applicable;Primary end point(s): Safety and tolerability: •Incidence, severity, causality and outcomes of AEs (serious and non-serious), with particular attention being paid to infections. •Evolution of laboratory parameters, in particular CBC, LFTs, inflammatory markers (ferritin and CRP) and coagulation parameters. •Number of patients withdrawn from the study due to safety reasons. Pharmacokinetics and pharmacodynamics: •PK profile of NI-0501. •Levels of circulating free IFN? at predose, and total IFN? after initiation of NI-0501. •Levels of the main IFN?-induced chemokines and other potential disease biomarkers. •Levels (if any) of circulating antibodies against NI-0501 to determine immunogenicity. ;Timepoint(s) of evaluation of this end point: At multiple timepoints over 8 weeks	—

Measure

Time frame

Main Objective: • To describe the pharmacokinetic profile of NI-0501 in sJIA patients with MAS •To confirm the proposed dosing regimen of NI-0501 •To evaluate the safety and tolerability profile of intravenous administrations of NI-0501 •To preliminary assess the efficacy of NI-0501 •To assess the levels of relevant biomarkers •To assess the immunogenicity of NI-0501;Secondary Objective: Not applicable;Primary end point(s): Safety and tolerability: •Incidence, severity, causality and outcomes of AEs (serious and non-serious), with particular attention being paid to infections. •Evolution of laboratory parameters, in particular CBC, LFTs, inflammatory markers (ferritin and CRP) and coagulation parameters. •Number of patients withdrawn from the study due to safety reasons. Pharmacokinetics and pharmacodynamics: •PK profile of NI-0501. •Levels of circulating free IFN? at predose, and total IFN? after initiation of NI-0501. •Levels of the main IFN?-induced chemokines and other potential disease biomarkers. •Levels (if any) of circulating antibodies against NI-0501 to determine immunogenicity. ;Timepoint(s) of evaluation of this end point: At multiple timepoints over 8 weeks

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Secondary

Measure	Time frame
Secondary end point(s): Efficacy: •Number of patients achieving MAS remission by Week 8 after initiation of NI-0501 treatment. •Time to MAS remission. •Number of patients for whom at any time during the study glucocorticoids can be tapered i) to the same (or lower) dose being administered before the occurrence of MAS ((in those patients who are already treated for sJIA) or ii) by 50% (or less) of the dose administered at NI-0501 treatment start (in those patients who present with MAS at sJIA onset). •Time to glucocorticoids tapering (as above described). •Survival time. •Number of patients withdrawn from the study due to lack of efficacy. ;Timepoint(s) of evaluation of this end point: At multiple timepoints over 8 weeks	—

Measure

Time frame

Secondary end point(s): Efficacy: •Number of patients achieving MAS remission by Week 8 after initiation of NI-0501 treatment. •Time to MAS remission. •Number of patients for whom at any time during the study glucocorticoids can be tapered i) to the same (or lower) dose being administered before the occurrence of MAS ((in those patients who are already treated for sJIA) or ii) by 50% (or less) of the dose administered at NI-0501 treatment start (in those patients who present with MAS at sJIA onset). •Time to glucocorticoids tapering (as above described). •Survival time. •Number of patients withdrawn from the study due to lack of efficacy. ;Timepoint(s) of evaluation of this end point: At multiple timepoints over 8 weeks

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Countries

France, Germany, Italy, Netherlands, Spain, United Kingdom

Contacts

Public ContactClinical Science

Novimmune SA

NI-0501.clinscience@novimmune.com+41228397142

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026