Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis

NCT06587191 | Active Not Recruiting

• 1 other identifier: NCHPOI-2024--07
• Study Type: observational
• Target: 7
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Primary Hemophagocytic Lymphohistiocytosis

Keywords

primary hemophagocytic lymphohistiocytosis; children; treatment; emapalumab

Outcome Measures

Primary Outcomes (1)

• Dynamics of severity clinical and laboratory activity using scale H-score.

  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 Outcomes (3)

• Adverse events

  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

  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

  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

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

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

You may not qualify if:

• not applicable. signed informed consent for participation in the study

Sponsors & Collaborators

• Federal Research Institute of Pediatric Hematology, Oncology and Immunology: lead

Study Sites (1)

Research Institute of Pediatric Hematology, Oncology and Immunology

Moscow, Russia

Location

MeSH Terms

Conditions

Lymphohistiocytosis, Hemophagocytic

Condition Hierarchy (Ancestors)

Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Anna Scherbina, MD,Phd

  Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2024
• First Posted: September 19, 2024
• Study Start: August 1, 2024
• Primary Completion: September 1, 2024
• Study Completion: October 1, 2024
• Last Updated: October 1, 2024

Record last verified: 2024-08

Locations

RU (1)