NCT03985423

A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH

A Phase 2/3, Open-label, Single Arm, Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Emapalumab in Adult Patients With Hemophagocytic Lymphohistiocytosis

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on the background of several clinical conditions (e.g. autoimmune disease, infection, malignancy). Emapalumab (previously referred to as NI-0501) is a monoclonal antibody neutralizing interferon-gamma (IFN-gamma), a key cytokine driving the inflammation and tissue damage seen in HLH. The purpose of this study is to assess the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH.

Detailed description

Study NI-0501-10 is an open-label, single arm, multicenter, Phase 2/3 interventional study. The study enrolls adult patients with hemophagocytic lymphohistiocytosis (HLH), specifically newly diagnosed patients with malignancy-associated HLH (M-HLH), and newly diagnosed or previously treated patients with non-malignancy-associated HLH.

Conditions

• Hemophagocytic Lymphohistiocytoses

Interventions

Type	Name	Description
DRUG	Emapalumab-Lzsg	Patients were administered Emapalumab-Lzsg by intravenous (i.v.) infusion over a period of 1 to 2 hours, at an initial dose of 6 mg/kg and continued at 3 mg/kg, every 3 days for the first 2 weeks (Study Day \[SD\] 15), and then twice-a-week. If the treating physician deemed appropriate, the dose of emapalumab could be increased (up to 10 mg/kg), guided by clinical and laboratory response.

Timeline

• Start date: 2020-06-02
• Primary completion: 2021-06-29
• Completion: 2021-06-29
• First posted: 2019-06-13
• Last updated: 2023-10-06
• Results posted: 2023-10-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study