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

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ClinicalTrials.gov Identifier: NCT03985423
Recruitment Status : Not yet recruiting
First Posted : June 13, 2019
Last Update Posted : June 13, 2019
Sponsor:
Information provided by (Responsible Party):
NovImmune SA

Tracking Information
First Submitted Date  ICMJE May 23, 2019
First Posted Date  ICMJE June 13, 2019
Last Update Posted Date June 13, 2019
Estimated Study Start Date  ICMJE September 2019
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 12, 2019)
Overall Response [ Time Frame: Week 4 ]
Achievement of either a Complete or Partial Response
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: June 12, 2019)
  • Best response on treatment [ Time Frame: Week 4; End of Treatment Visit (on average of 12 weeks) ]
  • Overall response [ Time Frame: End of Treatment Visit (on average of 12 weeks) ]
  • Time to Complete or Partial Response [ Time Frame: Week 4; End of Treatment visit (on average of 12 weeks) ]
  • Duration of response [ Time Frame: Up to 1 year after last emapalumab administration ]
  • HLH relapse [ Time Frame: Up to 1 year after last emapalumab administration ]
  • Incidence, severity, causality and outcomes of AEs (serious and non-serious) [ Time Frame: Up to 1 year after last emapalumab administration ]
  • Serum concentrations of emapalumab [ Time Frame: Up to 1 year after last emapalumab administration ]
  • Serum biomarker levels [ Time Frame: Up to 1 year after last emapalumab administration ]
    Levels of INF-gamma, CXCL9, sCD25, IL-06
  • Incidence of anti-drug antibodies (ADAs) against emapalumab [ Time Frame: Up to 1 year after last emapalumab administration ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH
Official Title  ICMJE 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
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophagocytic Lymphohistiocytoses
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Emapalumab
Intervention: Drug: Emapalumab-Lzsg
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: June 12, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2022
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Veronica Asnaghi, MD +41 61 201 13 31 vasnaghi@novimmune.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03985423
Other Study ID Numbers  ICMJE NI-0501-10
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party NovImmune SA
Study Sponsor  ICMJE NovImmune SA
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account NovImmune SA
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP