Ignite Creation Date:
2024-10-25 @ 7:54 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06587191
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
None
First Post:
2024-08-22
Brief Title:
Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Retrospective Non-interventional Study Assessing Efficacy and Safety of Emapalumab in Children With Primary Hemophagocytic Lymphohistiocytosis pHLH
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CGM EMA
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
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 IIIII 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
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 IIIII 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None