NCT06339177 Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study
| NCT ID | NCT06339177 |
| Status | Recruiting |
| Phase | — |
| Sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
| Condition | Lymphohistiocytosis, Hemophagocytic |
| Study Type | OBSERVATIONAL |
| Enrollment | 300 participants |
| Start Date | 2024-07-02 |
| Primary Completion | 2030-10-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 300 participants in total. It began in 2024-07-02 with a primary completion date of 2030-10-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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. ...
Eligibility Criteria
* INCLUSION CRITERIA: * Aged 18 years or older. * Established diagnosis of sHLH defined by meeting any published criteria, per Table 1: * Meeting the HLH-2004 criteria. * HScore of \>168. For those without a bone marrow biopsy to evaluate for hemophagocytosis (worth 35 points in the criteria), HScore\>134 will be used. * For those with underlying rheumatologic disease: meeting the 2016 American College of Rheumatology criteria for macrophage activation syndrome. * Agree to storage and sharing of study data and biospecimens for future research use. Table 1: Published Criteria for HLH HLH-2004 Criteria: Molecular diagnosis of HLH OR At least 5 of 8 below criteria: * Fever (\>38.4 Degrees Celcius) * Splenomegaly * Cytopenias affecting \>=2 of 3 lineages: Hgb \<9 g/dL, platelets \<10\^5/microliter, neutrophils \<10\^6/microliter * Hypertriglyceridemia (\>256 mg/dL) and/or fibrinogen \<1.5 g/L * Hemophagocytosis on biopsy * Serum ferritin \>=500 ng/mL * Increased serum sCD25 (\>2400 U/mL) * Low or absent NK cell activity HScore: Known immunosuppression: 0 (no) or 18 (yes) Temperature (degrees, Celsius): 0 (\<38.4), 33 (38.4-39.4), 49 (\>39.4) Organomegaly: 0 (no), 23 (liver/spleen), 38 (both) Number of cytopenias: 0 (1 lines), 24 (2 lines), 34 (3 lines) Ferritin (ng/mL): 0 (\<2000), 35 (2000-6000), 50 (\>6000) Triglycerides (mg/dL): 0 (\<1.5), 44 (1.5-4), 66 (\>4) Fibrinogen (g/L): 0 (\>2.5), 30 (\<2.5) AST (IU/mL): 0 (\<30), 19 (\>30) Hemophagocytosis: 0 (no) or 35 (yes) Cutoff value=169 ACR 2016-MAS Criteria: A febrile patient with known or suspected sJIA is classified as having macrophage activation syndrome if the following criteria are met: Ferritin \>684 ng/mL AND any 2 of the following: * Platelets \<=181,000/microliter * AST \>48 IU/mL * Triglycerides \>156 mg/dL * Fibrinogen \<=3.6 g/L Abbreviations: ACR, American College of Rheumatology; AST, aspartate transaminase; Hgb, hemoglobin; HLH, hemophagocytic lymphohistiocytosis; MAS, macrophage activation syndrome; NK, natural killer, sJIA, systemic juvenile idiopathic arthritis. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: * Currently pregnant. * Any condition that, in the judgment of the investigator, may put the participant at undue risk or make them unsuitable for participation in the study.
Contact & Investigator
Joseph M Rocco, M.D.
PRINCIPAL INVESTIGATOR
National Institute of Allergy and Infectious Diseases (NIAID)
Frequently Asked Questions
Who can join the NCT06339177 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 120 Years, studying Lymphohistiocytosis, Hemophagocytic. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06339177 currently recruiting?
Yes, NCT06339177 is actively recruiting participants. Contact the research team at joseph.rocco@nih.gov for enrollment information.
Where is the NCT06339177 trial being conducted?
This trial is being conducted at Bethesda, United States, Pittsburgh, United States.
Who is sponsoring the NCT06339177 clinical trial?
NCT06339177 is sponsored by National Institute of Allergy and Infectious Diseases (NIAID). The principal investigator is Joseph M Rocco, M.D. at National Institute of Allergy and Infectious Diseases (NIAID). The trial plans to enroll 300 participants.