NCT02069899

A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody

A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 58 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).

Detailed description

The aim of this study is to monitor the long-term safety profile of emapalumab in participants who have previously received at least one dose of emapalumab, including survival time after the administration of emapalumab. Moreover, the elimination profile of emapalumab and the immunogenicity will also be assessed. Furthermore, safety, tolerability, efficacy, and pharmacokinetic (PK) profile of emapalumab will be closely monitored in the event that some participants, upon request of the treating physician, will receive emapalumab treatment in the follow-up study.

Conditions

• Hemophagocytic Lymphohistiocytosis

Interventions

Type	Name	Description
DRUG	Emapalumab	Treatment with emapalumab is not planned for all enrolled participants. For participants who will continue receiving emapalumab in the context of this study (NI-0501-05), the dose and timing will be either carried forward from the last administered emapalumab dose as part of the parent study in which the participant was enrolled, or an adjusted dose will be administered, if necessary.

Timeline

• Start date: 2014-08-04
• Primary completion: 2021-05-18
• Completion: 2021-05-18
• First posted: 2014-02-24
• Last updated: 2022-06-28
• Results posted: 2022-06-28

Locations

16 sites across 5 countries: United States, France, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study