Haemophagocytic lymphohistiocytosis – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:37 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Haemophagocytic lymphohistiocytosis – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Comparing the timing of etoposide and dexamethasone treatment for patients with severe sporadic hemophagocytic lymphohistiocytosis in intensive care. https://clinicaltrials.eu/trial/comparing-the-timing-of-etoposide-and-dexamethasone-treatment-for-patients-with-severe-sporadic-hemophagocytic-lymphohistiocytosis-in-intensive-care/ Wed, 29 Apr 2026 15:09:00 +0000 https://clinicaltrials.eu/trial/comparing-the-timing-of-etoposide-and-dexamethasone-treatment-for-patients-with-severe-sporadic-hemophagocytic-lymphohistiocytosis-in-intensive-care/ This study focuses on patients with Hemophagocytic lymphohistiocytosis, a rare and serious condition where the immune system becomes overactive and causes excessive inflammation. This inflammation can lead to organ failure, which occurs when vital organs like the liver, kidneys, or lungs stop working correctly. The study is being conducted to compare two different ways of starting a treatment called etoposide in patients who are receiving intensive care. All patients in the study also receive dexamethasone, a medication used to reduce inflammation.

The first group will receive etoposide very early, within 12 hours of being admitted to the study. The second group will follow a delayed strategy, where the medication is only started if the patient’s condition does not improve after 48 hours. During the course of the study, researchers will monitor how these different timing strategies affect the development or worsening of organ problems. The study will also look at various aspects of recovery, such as the length of time spent in the hospital and the need for support from machines like a ventilator, which helps a person breathe.

]]> A study of ruxolitinib as first treatment for children with haemophagocytic lymphohistiocytosis (HLH) https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-ruxolitinib-and-corticosteroids-for-treating-hemophagocytic-lymphohistiocytosis-in-children/ Wed, 29 Apr 2026 15:04:00 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-ruxolitinib-and-corticosteroids-for-treating-hemophagocytic-lymphohistiocytosis-in-children/ This study focuses on haemophagocytic lymphohistiocytosis (HLH), a severe condition affecting the immune system in children where the body produces too many activated immune cells. The study will evaluate the effectiveness of ruxolitinib (also known as Jakavi) when used as a first treatment option along with corticosteroids in children who have primary HLH.

The medication being tested, Jakavi, comes in tablet form in different strengths (5 mg, 10 mg, 15 mg, and 20 mg) and is taken by mouth. The purpose of this research is to determine how well patients survive until they can receive a Haematopoietic Stem Cell Transplantation, which is a procedure where damaged bone marrow is replaced with healthy cells.

The treatment period will last up to 8 weeks, during which patients will receive daily doses of the medication. The maximum daily dose that may be given is 100 mg, and the total amount of medication received during the entire study will not exceed 5,750 mg. This is a Phase 2 study that will help researchers understand how effective this treatment approach is for children with this serious condition.

]]> Study on Etoposide for Patients with Severe Hemophagocytic Lymphohistiocytosis in Intensive Care https://clinicaltrials.eu/trial/study-on-etoposide-for-patients-with-severe-hemophagocytic-lymphohistiocytosis-in-intensive-care/ Wed, 29 Apr 2026 15:02:40 +0000 https://clinicaltrials.eu/trial/study-on-etoposide-for-patients-with-severe-hemophagocytic-lymphohistiocytosis-in-intensive-care/ This clinical trial is focused on studying a condition called Hemophagocytic Lymphohistiocytosis (HLH), which is a severe disorder where the body’s immune system becomes overly active and starts attacking its own tissues. The study is investigating the use of a medication called Etoposide, which is commonly used in cancer treatment, to see how it can help manage this condition when it becomes severe and requires intensive care.

The purpose of the study is to compare two different strategies for starting treatment with Etoposide in patients with severe HLH. One strategy involves starting the treatment early, within 12 hours of the patient being included in the study. The other strategy involves delaying the start of the treatment until 48 hours later, but only if the patient’s condition does not improve. This approach will help researchers understand which timing is more effective in managing the condition and preventing further organ damage.

Participants in the study will be monitored closely to see how their condition progresses with each treatment strategy. The study will look at various outcomes, such as the time it takes for symptoms to improve, the number of days patients spend on ventilators, and the overall length of their hospital stay. The study aims to provide valuable insights into the best way to use Etoposide for treating severe HLH in intensive care settings.

]]> Study on Itacitinib for Treating Non-Severe Hemophagocytic Lymphohistiocytosis in Adults https://clinicaltrials.eu/trial/study-on-itacitinib-for-treating-non-severe-hemophagocytic-lymphohistiocytosis-in-adults/ Wed, 29 Apr 2026 15:01:18 +0000 https://clinicaltrials.eu/trial/study-on-itacitinib-for-treating-non-severe-hemophagocytic-lymphohistiocytosis-in-adults/ This clinical trial is focused on studying a condition called Hemophagocytic Lymphohistiocytosis (HLH), specifically in adults with a non-severe form of the disease. HLH is a rare disorder where the body’s immune system becomes overly activated, leading to symptoms like fever, enlarged spleen, and low blood cell counts. The trial will use a medication called Itacitinib, which is taken in tablet form. Itacitinib is being tested to see if it can help improve the symptoms of HLH by reducing the excessive immune response.

The purpose of this study is to evaluate how well Itacitinib works in treating non-severe HLH. Participants in the trial will take Itacitinib and be monitored for changes in their symptoms. The study will look at how many participants experience a complete or partial improvement in their condition after 15 days of treatment. This will help researchers understand the effectiveness of Itacitinib in managing HLH symptoms.

Throughout the study, participants will receive regular check-ups to assess their health and the impact of the treatment. The trial is designed to gather important information about the potential benefits of Itacitinib for people with non-severe HLH, contributing to the development of better treatment options for this condition.

]]> Study of emapalumab in children and adults with Macrophage Activation Syndrome (MAS) occurring in Still’s Disease or Systemic Lupus Erythematosus https://clinicaltrials.eu/trial/study-of-emapalumab-for-treating-macrophage-activation-syndrome-in-children-and-adults-with-stills-disease-or-systemic-lupus-erythematosus/ Wed, 29 Apr 2026 14:59:17 +0000 https://clinicaltrials.eu/trial/study-of-emapalumab-for-treating-macrophage-activation-syndrome-in-children-and-adults-with-stills-disease-or-systemic-lupus-erythematosus/ This study focuses on treating Macrophage Activation Syndrome (MAS), a severe inflammatory condition that can occur in patients with Still’s Disease (including both children and adults) and Systemic Lupus Erythematosus. The condition being studied causes the immune system to become overactive, leading to inflammation throughout the body. The study will test a medication called emapalumab, which is given through an intravenous infusion.

The purpose of this research is to determine if emapalumab can effectively treat MAS in patients who have not responded well enough to standard treatments with high-dose steroids. The medication works by targeting specific proteins in the body that are involved in inflammation. The study will include both children and adults between 6 months and 80 years of age.

During the study, participants will receive emapalumab through an intravenous infusion for up to 28 days. The maximum daily dose will be 6 milligrams per kilogram of body weight. Patients will be monitored for 8 weeks after starting the treatment to evaluate how well the medication works. The study will look at how the treatment affects inflammation levels in the body and whether it allows patients to reduce their steroid medication use.

]]> Study on Ruxolitinib and Drug Combination for Critically Ill Adults with Acquired Hemophagocytic Syndrome https://clinicaltrials.eu/trial/study-on-ruxolitinib-and-drug-combination-for-critically-ill-adults-with-acquired-hemophagocytic-syndrome/ Wed, 29 Apr 2026 14:26:33 +0000 https://clinicaltrials.eu/trial/study-on-ruxolitinib-and-drug-combination-for-critically-ill-adults-with-acquired-hemophagocytic-syndrome/ This clinical trial is focused on studying a condition known as acquired hemophagocytic syndrome (HS), which is a serious disorder where the body’s immune system becomes overly active and starts attacking its own tissues. The study is investigating the use of a medication called ruxolitinib, which is a type of drug known as a JAK inhibitor. This medication is being tested to see if it can help improve the condition of patients with HS who are critically ill and admitted to the intensive care unit (ICU). The goal is to determine if ruxolitinib, when used alongside standard treatments, can better reverse organ failure compared to standard treatments alone.

In this study, patients will receive either ruxolitinib or a placebo, in addition to the usual care they would receive for HS. The treatment period for ruxolitinib is up to 28 days. Other medications involved in the study include lidocaine hydrochloride monohydrate, methylprednisolone acetate, etoposide, and betamethasone sodium phosphate, which are used for various supportive treatments. The study will monitor the patients’ health, focusing on improvements in organ function and overall survival, as well as any changes in symptoms related to HS.

The trial will assess the safety of ruxolitinib in these patients and track various health indicators, such as temperature, blood cell counts, and levels of certain proteins in the blood that indicate inflammation. The primary measure of success will be a significant improvement in the patients’ organ function scores after seven days of treatment. The study aims to provide valuable insights into the potential benefits of using ruxolitinib for treating critically ill patients with acquired hemophagocytic syndrome.

]]> Gene Therapy Study for Familial Hemophagocytic Lymphohistiocytosis Using MUNC-CD34 and MUNC-T3 in Patients with UNC13D Gene Mutations https://clinicaltrials.eu/trial/gene-therapy-study-for-familial-hemophagocytic-lymphohistiocytosis-using-munc-cd34-and-munc-t3-in-patients-with-unc13d-gene-mutations/ Wed, 29 Apr 2026 14:22:55 +0000 https://clinicaltrials.eu/trial/gene-therapy-study-for-familial-hemophagocytic-lymphohistiocytosis-using-munc-cd34-and-munc-t3-in-patients-with-unc13d-gene-mutations/ This clinical trial is focused on studying a rare disease called Familial Hemophagocytic Lymphohistiocytosis (FHL), which is caused by mutations in the UNC13D gene. The study is testing a new treatment involving gene therapy. This treatment uses a special type of cell therapy with two medications: MUNC13.4-CD34 suspension and MUNC13.4-T3 suspension. These medications are made from the patient’s own cells, which are modified outside the body using a lentiviral vector, a tool that helps deliver the correct gene into the cells. The modified cells are then given back to the patient through an intravenous infusion.

The purpose of the study is to evaluate the safety and effectiveness of this gene therapy in patients with FHL. Participants will receive a single dose of their own modified cells. The study will monitor the patients closely to see how their bodies respond to the treatment and to check for any side effects. The trial will also look at how well the treatment works in controlling the disease over time.

Throughout the study, researchers will observe the patients for any changes in their health, focusing on the safety of the treatment and its ability to help manage FHL. The study aims to provide valuable information on whether this new gene therapy can be a safe and effective option for patients with this genetic condition.

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