Primary Hemophagocytic LymphohistiocytosisCGM EMANCT06587191
Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis
An observational study of Primary Hemophagocytic Lymphohistiocytosis, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 day to 18 years
- Conditions the study is for
- Primary Hemophagocytic Lymphohistiocytosis
- How many people
- 7 participants
- Where
- 1 siteRussia
- Study dates
- 1 Aug 2024to 1 Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- Criteria for inclusion in the study were:
- 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
Who cannot join
- not applicable. signed informed consent for participation in the study
Trial locations and contacts
1 site in 1 country.
Russia1 site
Research Institute of Pediatric Hematology, Oncology and Immunology
Moscow
About this study
Official title: 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.
Read the 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:
- 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 Exclusion criteria - not applicable.
- Keywords
- primary hemophagocytic lymphohistiocytosis, children, treatment, emapalumab
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What is being measured
The primary outcome is the main question the study is designed to answer.
Dynamics of severity clinical and laboratory activity using scale H-score.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 Aug 2024
- Primary completion1 Sep 2024
- Study completion1 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hemophagocytic LymphohistiocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anna Scherbina, MD,Phd, Study director, Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06587191
- Sponsor's protocol ID
- NCHPOI-2024--07
- Acronym
- CGM EMA
- Status last verified
- Aug 2024
- First submitted
- 22 Aug 2024
- Last updated
- 1 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Non-interventional Study Assessing Efficacy and Safety of Emapalumab in Children With Primary Hemophagocytic Lymphohistiocytosis (pHLH)
- ClinicalTrials.gov ID
- NCT06587191
- Acronym / sponsor ID
- CGM EMA / NCHPOI-2024--07
- Keywords
- primary hemophagocytic lymphohistiocytosis, children, treatment, emapalumab
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyUniversity, hospital or other
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 1 Sep 2024 (actual)
- Study completion
- 1 Oct 2024 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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:
- 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 Exclusion criteria - not applicable.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 day to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- not applicable. signed informed consent for participation in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
3 secondary outcomes
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
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
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anna Scherbina, MD,Phd study director
Chief HSCT department at Federal Research Center for pediatric hematology, oncology and immunology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Hemophagocytic Lymphohistiocytosis, run by Federal Research Institute of Pediatric Hematology, Oncology and Immunology at 1 site in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2024.