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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03985423
Recruitment Status : Active, not recruiting
First Posted : June 13, 2019
Last Update Posted : May 10, 2021
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
NovImmune SA

Brief 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 HLH.

Condition or disease Intervention/treatment Phase
Hemophagocytic Lymphohistiocytoses Drug: Emapalumab-Lzsg Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 2, 2020
Estimated Primary Completion Date : November 2021
Estimated Study Completion Date : November 2021

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Emapalumab Drug: Emapalumab-Lzsg
Emapalumab administered intravenously at the initial dose of 6 mg/kg, and continued at the dose of 3 mg/kg every 3 days until Study Day 15, and twice-a-week afterwards

Primary Outcome Measures :
  1. Overall Response [ Time Frame: Week 4 ]
    Achievement of either a Complete or Partial Response

Secondary Outcome Measures :
  1. Best response on treatment [ Time Frame: Week 4; End of Treatment Visit (on average of 12 weeks) ]
  2. Overall response [ Time Frame: End of Treatment Visit (on average of 12 weeks) ]
  3. Time to Complete or Partial Response [ Time Frame: Week 4; End of Treatment visit (on average of 12 weeks) ]
  4. Duration of response [ Time Frame: Up to 1 year after last emapalumab administration ]
  5. HLH relapse [ Time Frame: Up to 1 year after last emapalumab administration ]
  6. Incidence, severity, causality and outcomes of AEs (serious and non-serious) [ Time Frame: Up to 1 year after last emapalumab administration ]
  7. Serum concentrations of emapalumab [ Time Frame: Up to 1 year after last emapalumab administration ]
  8. Serum biomarker levels [ Time Frame: Up to 1 year after last emapalumab administration ]
    Levels of INF-gamma, CXCL9, sCD25, IL-06

  9. Incidence of anti-drug antibodies (ADAs) against emapalumab [ Time Frame: Up to 1 year after last emapalumab administration ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male and female patients of age 18 and older at the time of HLH diagnosis
  • Fulfilment of 5 of the 8 HLH-2004 clinical criteria
  • Patients diagnosed with malignancy-associated HLH must be treatment naïve; patients diagnosed with HLH driven by any other etiology or idiopathic can be either treatment naïve or treatment experienced
  • Patients with non-malignancy-associated or idiopathic HLH who have already received conventional therapy for HLH must have failed prior treatment as per the treating physician's judgement
  • Informed consent signed by the patient or by the patient's legally authorized representative(s) (as required by local law)
  • Willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug, if female and of childbearing potential.

Exclusion Criteria:

  • Primary HLH
  • Current or scheduled administration of therapies known to trigger the cytokine release syndrome (e.g. chimeric antigen receptor (CAR)-modified T cells, bispecific T cell-engaging antibodies)
  • Current or scheduled administration of PD-1/PD-L1/CTLA-4 inhibitors
  • Life-expectancy associated with the underlying disease (triggering HLH) < 3 months
  • Ongoing participation in an investigational trial, or administration of any investigational treatment within 30 days
  • History of hypersensitivity or allergy to any components of emapalumab
  • Active mycobacteria, Histoplasma capsulatum, or Leishmania infections
  • Evidence of latent tuberculosis
  • Receipt of a bacille Calmette-Guerin (BCG) vaccine within 12 weeks prior to Screening
  • Receipt of a live or attenuated live (other than BCG) vaccine within 6 weeks prior to Screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03985423

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United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
NovImmune SA
Swedish Orphan Biovitrum
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Responsible Party: NovImmune SA Identifier: NCT03985423    
Other Study ID Numbers: NI-0501-10
First Posted: June 13, 2019    Key Record Dates
Last Update Posted: May 10, 2021
Last Verified: May 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by NovImmune SA:
interferon gamma
adult HLH
malignancy-associated HLH
Additional relevant MeSH terms:
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Lymphohistiocytosis, Hemophagocytic
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases