RecruitingNot Applicable

Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study

United States300 participantsStarted 2024-07-02

Plain-language 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. ...

Who can participate

Age range

18 Years – 120 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* 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…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Identify immunologic mechanisms involved in the pathogenesis of sHLH from a variety of predisposing conditions.

Timeframe: Through end of study.

Trial details

NCT IDNCT06339177

SponsorNational Institute of Allergy and Infectious Diseases (NIAID)

Sponsor typeNIH

Study typeOBSERVATIONAL

Primary completion2030-10-01

Contact for this trial

Joseph M Rocco, M.D.

(301) 312-2858 joseph.rocco@nih.gov

View on ClinicalTrials.gov More Lymphohistiocytosis, Hemophagocytic trials