A study to investigate the long-term safety and efficacy of a new drug, NI-0501, in children with a disease that is called " Haemophagocytic Lymphohistiocytosis".

A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody.

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-005753-23-IT

Enrollment

Registered

2013-04-29

Start date

2013-06-19

Completion date

Unknown

Last updated

2014-11-10

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

Conditions

haemophagocytic lymphohistiocytosis MedDRA version: 17.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: NI-0501 Product Code: NI-0501 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: N/A Current Sponsor code: NI-0501 Other descriptive name: Human anti-interfe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Having received at least one dose of NI-0501 during a previous NI-0501 study. 2. Having signed the Informed Consent by the patient or the patient's legal representative(s), as applicable, with the assent of patients who are legally capable of providing it. 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 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: None.

Design outcomes

Primary

Measure	Time frame
Main Objective: - To monitor the long-term safety profile of patients who have received at least one dose of NI-0501. - To assess HLH patients' survival after NI-0501 treatment. - To study the elimination profile of NI-0501. - To assess the immunogenicity of NI-0501.;Secondary Objective: Not applicable;Primary end point(s): • Safety: - Incidence, intensity, possible relationship to NI-0501 and outcomes of Adverse Events (serious and non-serious) - Evolution of vital signs, physical examination and laboratory values over time • Efficacy (when relevant): - Clinical Response mostly based on HLH diagnostic criteria and existence of neurological symptomatology - Survival before HSCT, 100 days and one year after post HSCT • Pharmacokinetics: NI-0501 elimination profile (clearance and elimination half-life) • Pharmacodynamics: NI-0501 activity on IFN? neutralization • Immunogenicity: presence of ADA • Exploratory endpoints: e.g. additional markers of disease activity;Timepoint(s) of evaluation of this end point: See E.5.1	—

Measure

Time frame

Main Objective: - To monitor the long-term safety profile of patients who have received at least one dose of NI-0501. - To assess HLH patients' survival after NI-0501 treatment. - To study the elimination profile of NI-0501. - To assess the immunogenicity of NI-0501.;Secondary Objective: Not applicable;Primary end point(s): • Safety: - Incidence, intensity, possible relationship to NI-0501 and outcomes of Adverse Events (serious and non-serious) - Evolution of vital signs, physical examination and laboratory values over time • Efficacy (when relevant): - Clinical Response mostly based on HLH diagnostic criteria and existence of neurological symptomatology - Survival before HSCT, 100 days and one year after post HSCT • Pharmacokinetics: NI-0501 elimination profile (clearance and elimination half-life) • Pharmacodynamics: NI-0501 activity on IFN? neutralization • Immunogenicity: presence of ADA • Exploratory endpoints: e.g. additional markers of disease activity;Timepoint(s) of evaluation of this end point: See E.5.1

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Secondary

Measure	Time frame
Secondary end point(s): None.;Timepoint(s) of evaluation of this end point: See E.5.2	—

Countries

Austria, Czech Republic, Germany, Italy, Spain, United States

Contacts

Public ContactClinical Trials Information

NovImmune SA

+41228397142

Outcome results

None listed

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