Ignite Creation Date:
2024-05-06 @ 8:18 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06339177
Status:
RECRUITING
Last Update Posted:
2024-07-08
First Post:
2024-03-29
Brief Title:
Hemophagocytic Lymphohistiocytosis HLH Evaluation and Research of Clinical ImmUnoLogic and TranscriptomE Study
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Hemophagocytic Lymphohistiocytosis HLH Evaluation and Research of Clinical ImmUnoLogic and TranscriptomE Study
Status:
RECRUITING
Status Verified Date:
2024-10-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
Hemophagocytic lymphohistiocytosis HLH is a disease caused by disrupted immune function People with HLH are prone to fevers and illnesses which can be fatal Some people develop a genetic form of this disease pHLH but researchers do not understand why some other people develop a nongenetic form sHLH They also do not have good ways to diagnose and treat sHLH
Objective
To learn about sHLH and why some people get it and others do not
Eligibility
Adults aged 18 years and older with sHLH
Design
Participants will be admitted to the study based on a review of their medical records Those who join will have at least 3 clinical evaluations over 9 to 12 months These may occur during an inpatient hospitalization if they require medical care or in the outpatient clinic
Participants will also have a physical exam at each visit Up to half a cup of blood will be drawn at each visit Participants may also have their blood drawn by their own doctors who will send the samples to the researchers Researchers may also contact these participants by telephone or video calls
The blood will be used for clinical tests as well as research No new treatments will be administered as part of this study however standard medications and treatments may be recommended
Participants may opt to continue their visits once a year for 3 more years Participants may also opt for an extra clinial evaluation 1 week after starting a new treatment
Hemophagocytic lymphohistiocytosis HLH is a disease caused by disrupted immune function People with HLH are prone to fevers and illnesses which can be fatal Some people develop a genetic form of this disease pHLH but researchers do not understand why some other people develop a nongenetic form sHLH They also do not have good ways to diagnose and treat sHLH
Objective
To learn about sHLH and why some people get it and others do not
Eligibility
Adults aged 18 years and older with sHLH
Design
Participants will be admitted to the study based on a review of their medical records Those who join will have at least 3 clinical evaluations over 9 to 12 months These may occur during an inpatient hospitalization if they require medical care or in the outpatient clinic
Participants will also have a physical exam at each visit Up to half a cup of blood will be drawn at each visit Participants may also have their blood drawn by their own doctors who will send the samples to the researchers Researchers may also contact these participants by telephone or video calls
The blood will be used for clinical tests as well as research No new treatments will be administered as part of this study however standard medications and treatments may be recommended
Participants may opt to continue their visits once a year for 3 more years Participants may also opt for an extra clinial evaluation 1 week after starting a new treatment
Detailed Description:
Study Description
The purpose of this natural history study is to study the immunopathogenesis of secondary hemophagocytic lymphohistiocytosis sHLH This will include detailed longitudinal clinical and immunologic characterization of sHLH as well as mechanistic studies evaluating inflammasome activation cytotoxicity and JAK-STAT signaling Participants with sHLH will undergo clinical assessment and management along with three research blood draws with the option for additional blood draws at time points such as post-immunosuppressive treatment or treatment escalation and during longer-term follow-up Participation may be in person or remote with blood collected and processed locally then shipped to the NIH Longitudinal clinical information will be recorded and standard of care will be offered as needed
Primary Objective
To study the immunopathogenesis of sHLH from various etiologies including biomarkers cellular phenotypes and gene expression to determine mechanistic pathways that may be amenable to host-directed therapies
Secondary Objectives
To prospectively and longitudinally characterize the predisposing conditions clinical features acute triggers clinical labs and outcomes of a cohort of individuals meeting sHLH criteria
To compare biomarkers and immune profiles between the classically defined sHLH subgroups malignancy autoimmune immune-therapy infectious-triggered unknown etiology
To evaluate for novel immunologic subsets of sHLH with unsupervised analyses using a multi-omic approach including single-cell transcriptomics and proteomics
To evaluate for rare protein-altering variants in genes associated with cytotoxicity inflammasome activation or immunoregulation via optional co-enrollment in NIAID Centralized Sequencing protocol
Primary Endpoint
Identify immunologic mechanisms involved in the pathogenesis of sHLH from a variety of predisposing conditions
Secondary Endpoints
Characterize the longitudinal clinical course of sHLH including relapse rates and predictors of key clinical outcomes at one year after diagnosis
Identify differences in clinical andor immunologic profiles between sHLH subgroups malignancy autoimmune immune-therapy infectious-triggered unknown etiology
To evaluate for novel immunologic profiles in sHLH using multi-omic unsupervised analyses
Identify new genetic determinants of susceptibility to sHLH in the setting of different predisposing conditions
The purpose of this natural history study is to study the immunopathogenesis of secondary hemophagocytic lymphohistiocytosis sHLH This will include detailed longitudinal clinical and immunologic characterization of sHLH as well as mechanistic studies evaluating inflammasome activation cytotoxicity and JAK-STAT signaling Participants with sHLH will undergo clinical assessment and management along with three research blood draws with the option for additional blood draws at time points such as post-immunosuppressive treatment or treatment escalation and during longer-term follow-up Participation may be in person or remote with blood collected and processed locally then shipped to the NIH Longitudinal clinical information will be recorded and standard of care will be offered as needed
Primary Objective
To study the immunopathogenesis of sHLH from various etiologies including biomarkers cellular phenotypes and gene expression to determine mechanistic pathways that may be amenable to host-directed therapies
Secondary Objectives
To prospectively and longitudinally characterize the predisposing conditions clinical features acute triggers clinical labs and outcomes of a cohort of individuals meeting sHLH criteria
To compare biomarkers and immune profiles between the classically defined sHLH subgroups malignancy autoimmune immune-therapy infectious-triggered unknown etiology
To evaluate for novel immunologic subsets of sHLH with unsupervised analyses using a multi-omic approach including single-cell transcriptomics and proteomics
To evaluate for rare protein-altering variants in genes associated with cytotoxicity inflammasome activation or immunoregulation via optional co-enrollment in NIAID Centralized Sequencing protocol
Primary Endpoint
Identify immunologic mechanisms involved in the pathogenesis of sHLH from a variety of predisposing conditions
Secondary Endpoints
Characterize the longitudinal clinical course of sHLH including relapse rates and predictors of key clinical outcomes at one year after diagnosis
Identify differences in clinical andor immunologic profiles between sHLH subgroups malignancy autoimmune immune-therapy infectious-triggered unknown etiology
To evaluate for novel immunologic profiles in sHLH using multi-omic unsupervised analyses
Identify new genetic determinants of susceptibility to sHLH in the setting of different predisposing conditions
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 001899-I | None | None | None |