Clinical trial NCT06047210: Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis
Official title: An Observational, Multicenter Study to Evaluate Levels of Interferon Gamma (IFNγ) and Other Inflammatory Mediators in Adult Patients With Hemophagocytic Lymphohistiocytosis (A-HLH)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophagocytic Lymphohistiocytoses
Keywords: Hemophagocytic Lymphohistiocytosis
Interferon Gamma
IFNγ
Biomarkers
Inflammatory Markers
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: Brigham and Women's Hospital
name: Harvard Medical School (HMS and HSDM)
First posted: 2023-09-21
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female adult (\>=18 years old) patients who are diagnosed with HLH and meet the inclusion criteria.

Brief summary: This observational study is designed to provide a better understanding of the potential use of interferon gamma (IFNγ) and IFNγ-inducible chemokines as markers of HLH disease activity in adults, and the potential of IFNγ to represent a therapeutic target.

Detailed description: This is a observational study designed to determine the levels of pro-inflammatory markers in adult patients diagnosed with HLH and to assess the relationship between the biomarkers and disease activity in these patients during the HLH course. Inflammatory markers include IFNγ and IFNγ-induced chemokines C-X-C chemokine ligand 9 and C-X-C chemokine ligand 10.

In addition to the blood samples for the biomarker analysis, relevant information gathered by the treating physician will be collected in a data collection form. Whenever possible, collection of serum samples for biomarker analysis and relevant information should occur at HLH diagnosis, at regular time intervals during the treatment course (not more than once a week) up to resolution of HLH.

Blood samples will also be sent to the study sponsor (NovImmune S.A.) for cytokine testing. Before patient's samples are sent to the sponsor for testing, patient's name and any personal identifying information will be coded to protect participant's privacy.

Eligibility criteria: Inclusion Criteria:

* Patients \>=18 years old diagnosed with active HLH as established by the treating physician.
* HLH forms of unknown origin or secondary to infections or rheumatologic disorders.
* Should the diagnosis of primary HLH or malignancy become apparent after inclusion, the data collected will be analyzed separately as additional cohorts.
* The patient must have consented to the use of their clinical data for research purposes at the site.

Exclusion Criteria:

* Patients with primary HLH (diagnosed by the presence of homozygous mutations in a known HLH causative gene) and secondary HLH due to malignancy are excluded.

Primary outcomes:
measure: Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 9 (CXCL9)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Interferon Gamma (IFNg)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Interleukin 10 (IL-10)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Interleukin 1 Beta (IL-1B)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Interleukin 6 (IL-6)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Neopterin
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Tumor Necrosis Factor Alpha (TNFa)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Total Human Interferon Gamma (hIFNg)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Soluble CD163 (sCD163)
timeFrame: 11 months
measure: Mean Serum Concentration of Inflammatory Biomarker - Soluble IL-2 Receptor Alpha (sIL2Ra)
timeFrame: 11 months
measure: Mean Serum Concentration of White Blood Cells
timeFrame: 11 months
measure: Mean Serum Concentration of Red Blood Cells
timeFrame: 11 months
measure: Mean Serum Concentration of Hemoglobin
timeFrame: 11 months
measure: Mean Serum Concentration of Hematocrit
timeFrame: 11 months
measure: Mean Serum Concentration of Platelets
timeFrame: 11 months
measure: Mean Serum Concentration of Neutrophils
timeFrame: 11 months
measure: Mean Serum Concentration of Lymphocytes
timeFrame: 11 months
measure: Mean Serum Concentration of Monocytes
timeFrame: 11 months
measure: Mean Serum Concentration of Eosinophils
timeFrame: 11 months
measure: Mean Serum Concentration of Basophils
timeFrame: 11 months
measure: Mean Plasma Concentration of D-Dimer
timeFrame: 11 months
measure: Mean Plasma Concentration of Fibrinogen
timeFrame: 11 months
measure: Mean Activated Partial Thromboplastin Time (aPTT)
timeFrame: 11 months
measure: Mean Prothrombin Time
timeFrame: 11 months
measure: Mean Serum Concentration of C-Reactive Protein
timeFrame: 11 months
measure: Mean Serum Concentration of Ferritin
timeFrame: 11 months
measure: Mean Plasma Concentration of Blood Urea Nitrogen
timeFrame: 11 months
measure: Mean Serum Concentration of Creatinine
timeFrame: 11 months
measure: Mean Serum Concentration of Albumin
timeFrame: 11 months
measure: Mean Serum Concentration of Sodium
timeFrame: 11 months
measure: Mean Serum Concentration of Fasting Triglycerides
timeFrame: 11 months
measure: Mean Serum Concentration of Total Bilirubin
timeFrame: 11 months
measure: Mean Serum Concentration of Conjugated Bilirubin
timeFrame: 11 months
measure: Mean Serum Concentration of Alanine Aminotransferase
timeFrame: 11 months
measure: Mean Serum Concentration of Aspartate Aminotransferase
timeFrame: 11 months
measure: Mean Serum Concentration of Alkaline Phosphatase
timeFrame: 11 months
measure: Mean Serum Concentration of Lactate Dehydrogenase
timeFrame: 11 months
measure: Mean Serum Concentration of Immunoglobulin G (IgG)
timeFrame: 11 months
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
Study officials:
affiliation: Brigham and Women's Hospital
name: Nancy Berliner, MD
role: PRINCIPAL_INVESTIGATOR
References: