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Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis

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.
 
ClinicalTrials.gov Identifier: NCT03312751
Recruitment Status : Active, not recruiting
First Posted : October 18, 2017
Last Update Posted : November 5, 2021
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Brief Summary:

The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, with special focus on long-term outcome and quality of life assessments. Emapalumab can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the available standard of care.

Emapalumab is to be administered until the start of conditioning, with an anticipated duration ranging from a minimum of 4 weeks to approximately 12 weeks and not exceeding 6 months.

After treatment completion, patients will continue in the study for long-term follow-up until 1 year after either HSCT or last emapalumab infusion (if HSCT is not performed).


Condition or disease Intervention/treatment Phase
Primary Hemophagocytic Lymphohistiocytosis Drug: Emapalumab Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 34 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFNγ) Monoclonal Antibody, and to Assess Its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients With Primary Hemophagocytic Lymphohistiocytosis
Actual Study Start Date : February 5, 2019
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: Emapalumab Drug: Emapalumab
Emapalumab will be administered by intravenous infusion, twice weekly.




Primary Outcome Measures :
  1. Overall Response at Week 8 or End of Treatment (if earlier) [ Time Frame: Up to Week 8 ]
    Achievement of either Complete or Partial Response or HLH Improvement, at Week 8 or EOT (whichever occurs earlier)


Secondary Outcome Measures :
  1. Overall Survival [ Time Frame: Up to 18 months ]
    Including survival to HSCT and post-HSCT

  2. Number of patients proceeding to HSCT [ Time Frame: Up to 18 months ]
    Number of patients proceeding to HSCT

  3. Change in PedsQL Score [ Time Frame: Up to 18 months ]
    Assessment of the quality of life using the PedsQL "Pediatric Quality of Life Inventory"

  4. Incidence, severity, causality and outcomes of AEs (serious and non-serious) [ Time Frame: Up to 18 months ]
    Incidence of adverse events

  5. Immunogenicity [ Time Frame: Up to 18 months ]
    Incidence of circulating antibodies against emapalumab


Other Outcome Measures:
  1. Overall Response at start of conditioning [ Time Frame: Up to 6 months ]
    Achievement of either Complete or Partial Response or HLH Improvement, at start of conditioning

  2. Serum concentrations of emapalumab [ Time Frame: Up to 18 months ]
    Serum concentrations of emapalumab

  3. Pharmacodynamic parameters [ Time Frame: Up to 18 months ]
    Levels of total IFNγ, markers of its neutralization (CXCL9 and CXCL10) and other relevant disease biomarkers e.g. sCD25



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Primary HLH patients with active disease.
  • Treatment naïve patients or patients having already received HLH conventional therapy, but having failed or unable to tolerate current standard of care.
  • Informed consent signed by the patient or by the patient's legally authorized representative.
  • Guidance on contraception

Exclusion Criteria:

  • Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
  • Active Mycobacteria, Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter or Leishmania infections.
  • Evidence of latent tuberculosis.
  • Presence of malignancy.
  • Concomitant disease or malformation severely affecting cardiovascular, pulmonary, central nervous system (CNS), liver, or renal function, that in the opinion of the Investigator may significantly affect the likelihood to respond to treatment and/or the assessment of emapalumab safety and/or efficacy
  • History of hypersensitivity or allergy to any component of the study regimen
  • Receipt of a 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
  • Pregnant or lactating female patients.

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 ClinicalTrials.gov identifier (NCT number): NCT03312751


Locations
Show Show 28 study locations
Sponsors and Collaborators
Swedish Orphan Biovitrum
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Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT03312751    
Other Study ID Numbers: NI-0501-09
First Posted: October 18, 2017    Key Record Dates
Last Update Posted: November 5, 2021
Last Verified: November 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 Swedish Orphan Biovitrum:
Interferon-gamma
NI-0501
primary HLH
emapalumab
Additional relevant MeSH terms:
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Lymphohistiocytosis, Hemophagocytic
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Lymphatic Diseases