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Nonapeptide-1

Melanostatine-5, White 05

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Studies 4
Trials 100
Completed PHASE2, PHASE3 INTERVENTIONAL 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

View on ClinicalTrials.gov Updated Dec 15, 2025

Brief 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.

Interventions

Name: Emapalumab
Type: DRUG
Description: 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.

Primary Outcomes

Measure: Number of Participants With Adverse Event (AE)
TimeFrame: From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days)
Description: Adverse events were defined as any undesirable experience occurring in a participant during the study, whether or not considered related to emapalumab.

Trial Information

NCT ID

NCT02069899

Status

Completed

Study Type

INTERVENTIONAL

Phases

PHASE2, PHASE3

Sponsor

Swedish Orphan Biovitrum

Last Updated

December 15, 2025