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

Phase 2

Withdrawn

• Conditions: Systemic Juvenile Idiopathic Arthritis; Systemic juvenile rheumatoid Arthritis; 10003816

• Registration Number: NL-OMON48933
• Lead Sponsor: Swedish Orphan Biovitrum AG

Brief Summary

Trial never started

Detailed Description

Not available

Study Timeline

• Results First Posted: 2022-03-08
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

1. Having received at least one dose of NI-0501 during a previous NI-0501 study
or under a compassionate use (CU) treatment protocol.
2. Informed Consent signed by the patient or the patient*s legal
representative(s), as applicable, with the assent of patients who are legally
capable of providing it.

Exclusion Criteria

Please see inclusion criteria.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Study Parameters: * Vital signs, including body temperature<br /><br>* Physical examination, including liver and spleen sizes<br /><br>* Laboratory parameters: complete blood count, coagulation tests (aPTT, PT,<br /><br>d-Dimers and fibrinogen), ferritin,CRP, LDH, glucose, triglycerides, ,<br /><br>liver(alanine aminotransferase, aspartate aminotransferase, gamma glutamyl<br /><br>transferase, total bilirubin and alkaline phosphatases) and renal function<br /><br>tests (creatinine, albumin, urea)<br /><br>* Pharmacokinetics: circulating NI-0501 concentration<br /><br>* Pharmacodynamics: circulating IFN&gamma; levels, CXCL9, CXCL10 and exploratory<br /><br>markers of disease activity (e.g. sCD25)<br /><br>* Anti-drug antibodies (ADAs).</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>N/A</p><br>