Blood and Lymph Conditions

All Conditions

Rare Diseases

Lymphohistiocytosis, Hemophagocytic

Histiocytosis

Histiocytosis, Non-Langerhans-Cell

Lymphatic Diseases

Non-Langerhans-Cell Histiocytosis

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Interferons

Interferon-gamma

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.

Emapalumab-Lzsg

Radmila Kanceva

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.

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.

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

Achievement of either a Complete or Partial Response

Complete Response is adjudicated if the following are observed:

* No fever = body temperature \<37.5°C
* Normal spleen size
* No cytopenia = Absolute Neutrophil Counts \>=1.0x10\^9/L and platelet count \>=100x10\^9/L \[absence of G-CSF and transfusion support must be documented for at least 4 days to report no cytopenia\]
* No hyperferritinemia = serum level is \<2000 µg/L
* No evidence of coagulopathy, i.e., normal D-Dimer and/or normal (\>150 mg/dL) fibrinogen levels
* No neurological and CSF abnormalities attributed to HLH
* No sustained worsening of sCD25 (as indicated by at least two consecutive measurements that are \>2-fold higher than baseline)

Partial Response is adjudicated if there is an improvement (\>50% change from baseline or normalization) of at least 3 HLH clinical and laboratory criteria (including Central Nervous System abnormalities).

As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description.

Levels of interferon-gamma, C-X-C chemokine ligand 9, soluble CD25, interleukin-6.

Light Chain Bioscience - Novimmune SA

Swedish Orphan Biovitrum

The All Treated analysis set included all patients who received at least 1 dose or 1 incomplete dose of emapalumab. Only TEAEs are reported.

Patients were administered emapalumab 6 mg/kg IV infusion for 1 to 2 hours, and continued at 3 mg/kg every 3 days for the first 2 weeks, and then twice-a-week until clinically satisfactory response was achieved.

If the treating physician deemed appropriate, the dose of emapalumab could be increased (up to 10 mg/kg), guided by clinical and laboratory response.

Emapalumab

Age, Continuous

>= 18 and 64 years

>= 65 and 84 years

>= 85 years

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race

Race/Ethnicity, Customized

United States

Region of Enrollment

Weight

Height

The All Treated analysis set included all patients who received at least 1 dose or 1 incomplete dose of emapalumab.

Since the study was prematurely terminated, and considering the low number of patients enrolled, no analysis related to primary and secondary efficacy endpoints and no Pharmacokinetic and Pharmacodynamic summary statistics were performed. Accordingly, no conclusion on the efficacy of emapalumab on this patient population could be withdrawn from the study.

Radmila Kanceva, MD

Since the study was prematurely terminated, no data was collected for the primary efficacy endpoint.

Overall Response

Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints.

Best Response on Treatment

Overall Survival

Time to Complete Response or Partial Response

Duration of Response

Hemophagocytic Lymphohistiocytosis Relapse

Since the study was prematurely terminated, no data was collected for the secondary safety endpoints.

Incidence, Severity, Causality and Outcomes of Serious Adverse Events and Non-serious Adverse Events

Since the study was prematurely terminated, no data was collected for the secondary Pharmacokinetic endpoints.

Serum Concentrations of Emapalumab

Since the study was prematurely terminated, no data was collected for the secondary Pharmacodynamic endpoints.

Serum Biomarker Levels

Incidence of Anti-Drug Antibodies Against Emapalumab

Patients were administered emapalumab 6 milligram per kilogram (mg/kg) intravenous (IV) infusion for 1 to 2 hours, and continued at 3 mg/kg every 3 days for the first 2 weeks, and then twice-a-week until clinically satisfactory response was achieved.

If the treating physician deemed appropriate, the dose of emapalumab could be increased (up to 10 mg/kg), guided by clinical and laboratory response.

Spots Global Cancer Trial Database for A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

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