Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis (CGM EMA)

September 30, 2024 updated by: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Retrospective Non-interventional Study Assessing Efficacy and Safety of Emapalumab in Children With Primary Hemophagocytic Lymphohistiocytosis (pHLH)

The goal of this retrospective study is to assess efficacy and safety of emapalumab prescribed for the treatment in children with refractory from of pHLH.

Study Overview

Status

Active, not recruiting

Conditions

Detailed Description

Primary hemophagocytic lymphohistiocytosis (pHLH) encompasses a group of genetically determined disorders, characterized by severe, often fulminant systemic inflammation, cytopenia and multiple organ involvement. The disease manifests predominantly in the early childhood, has high mortality rate and in most cases requires hematopoietic stem cell transplantation (HSCT) as the only currently available curative option. Yet, HSCT outcomes are dependent on the state of remission of the underlying systemic inflammation and infections complications, as well as organ damage due to the side effects of the chemotherapeutic drugs received prior to it. For years the gold standard of HLH treatment has been the dexamethasone and etoposide-based HLH-2004 protocol, yet it failed to uniformly control the disease. An alternative regiment combining antithymocyte globulin and corticosteroids demonstrated good results but barely improved survival. Also, no universally accepted treatment exists for relapsed or refractory hemophagocytic lymphohistiocytosis. Advances in availability of biologic treatments open up new possibilities of HLH therapy, with several targets proposed in recent years. Interleukin 6 inhibitor tocilizumab, interleukin 1 inhibitor anakinra, Janus-kinase inhibitor (JAKinib) ruxolititnib have been used for HLH treatment, with variable results.

Mounting evidence provides support for the pivotal pathogenic role of interferon-γ (IFNg) in hemophagocytic lymphohistiocytosis. Emapalumab is a fully human IgG1 anti-interferon-γ monoclonal antibody that binds free and receptor-bound interferon-γ and inhibits its biologic activity. The data accumulated from the phase II/III clinical trial and reports of the small groups of patients demonstrate its efficacy in pHLL.

The study will collect and analyze information of the effectiveness and safety of emapalumab treatment that was previously prescribed in a cohort of seven pediatric patients with pHLH.

Criteria for inclusion in the study were:

  1. age from 0 to 18 y.
  2. the diagnosis of pHLH ( established according to the Histiocytic society criteria).
  3. treatment with empalumab for at least 2 weeks
  4. signed informed consent for participation in the study Exclusion criteria - not applicable.

Study Type

Observational

Enrollment (Estimated)

7

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Russian Federation
      • Moscow, Russian Federation
        • Research Institute of Pediatric Hematology, Oncology and Immunology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

The disease manifests predominantly in the early childhood, has high mortality rate.

Also, no universally accepted treatment exists for relapsed or refractory hemophagocytic lymphohistiocytosis. For years the gold standard of HLH treatment has been the dexamethasone and etoposide-based HLH-2004 protocol, yet it failed to uniformly control the disease.

Description

Inclusion Criteria:

Criteria for inclusion in the study were:

  1. age from 0 to 18 y.
  2. the diagnosis of pHLH ( established according to the Histiocytic society criteria).
  3. treatment with empalumab for at least 2 weeks
  4. signed informed consent for participation in the study

Exclusion Criteria:

not applicable. signed informed consent for participation in the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Dynamics of severity clinical and laboratory activity using scale H-score.
Time Frame: at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Adverse events
Time Frame: at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
Infection complications before therapy
Time Frame: at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
Infection complications after therapy
Time Frame: at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days
at the day the first dose of study treatment was administered, further at days 14, 28 and at the end of treatment, an average 45 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Investigators

  • Study Director: Anna Scherbina, MD,Phd, Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2024

Primary Completion (Actual)

September 1, 2024

Study Completion (Estimated)

October 1, 2024

Study Registration Dates

First Submitted

August 22, 2024

First Submitted That Met QC Criteria

September 5, 2024

First Posted (Actual)

September 19, 2024

Study Record Updates

Last Update Posted (Actual)

October 1, 2024

Last Update Submitted That Met QC Criteria

September 30, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCHPOI-2024--07

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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