Ruxolitinib Combination for Hemophagocytic Lymphohistiocytosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the effects of combining ruxolitinib, a drug that blocks signals causing inflammation, with a less intense version of a common treatment for hemophagocytic lymphohistiocytosis (HLH). HLH occurs when the immune system becomes overly active and attacks the body's own tissues. The trial aims to determine if this combination is effective while reducing side effects. Suitable participants have newly diagnosed HLH with symptoms such as persistent fever and an enlarged spleen. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, certain medications like cyclosporine and tacrolimus are not allowed during the initial induction period, and prior use of some HLH-directed therapies is restricted.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that the treatment combination for hemophagocytic lymphohistiocytosis (HLH) using ruxolitinib, dexamethasone, and etoposide is generally well-tolerated.
Ruxolitinib calms the immune system. Studies have found it safe for people with HLH, with patients experiencing fewer symptoms and better survival rates.
Dexamethasone, a steroid that reduces inflammation, is considered safe and effective for treating HLH. It is often used with ruxolitinib in studies, showing good results.
Etoposide targets certain white blood cells that attack the body. As a standard part of HLH treatment, it has improved survival rates.
Overall, these treatments have been studied for safety in people with HLH and have shown positive results, with manageable side effects.12345Why do researchers think this study treatment might be promising for HLH?
Researchers are excited about the combination of ruxolitinib, dexamethasone, and etoposide for treating Hemophagocytic Lymphohistiocytosis (HLH) because it introduces a novel approach by incorporating ruxolitinib, a JAK1/2 inhibitor, which is not part of the standard treatment. Most existing HLH treatments rely heavily on immunosuppressive and chemotherapy drugs like dexamethasone and etoposide. However, ruxolitinib offers a targeted mechanism by blocking specific pathways (JAK1/2) that are crucial in the inflammatory process of HLH. This precise targeting may lead to better control of inflammation and potentially fewer side effects compared to traditional therapies. The integration of ruxolitinib alongside established drugs could enhance overall treatment efficacy and transform HLH management.
What evidence suggests that this treatment might be an effective treatment for HLH?
In this trial, participants will receive a combination treatment of ruxolitinib, dexamethasone, and etoposide for hemophagocytic lymphohistiocytosis (HLH). Research has shown that ruxolitinib yields promising results for HLH, helping patients live longer and feel better compared to usual treatments. One study found that combining ruxolitinib with dexamethasone was effective and safe, with all patients showing improvement. Dexamethasone is a type of steroid, and etoposide is a chemotherapy drug. These are part of the HLH-94 treatment plan, which has led to better outcomes and higher survival rates. Specifically, etoposide-based therapy has improved patient response and survival. This combination treatment aims to effectively manage the overactive immune system in HLH.12467
Who Is on the Research Team?
Jerry Lee, MD
Principal Investigator
University of California, San Francisco
Are You a Good Fit for This Trial?
Adults with newly diagnosed hemophagocytic lymphohistiocytosis (HLH) who meet specific diagnostic criteria, including fever, spleen enlargement, low blood cell counts, and high levels of certain immune markers. Participants must not be pregnant or breastfeeding and agree to use contraception. Excluded are those with severe organ dysfunction, recent investigational drug use, certain viral infections unless controlled by medication, prior malignancies that may affect the trial's safety assessment or have received other HLH treatments.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Induction Therapy
Participants receive ruxolitinib plus de-intensified HLH-94 induction with dexamethasone and etoposide for 4 weeks, with potential extension based on response
Continuation Therapy
Participants receive continuation therapy with ruxolitinib for up to 6 months
Follow-up
Participants are monitored for safety and effectiveness after treatment completion
What Are the Treatments Tested in This Trial?
Interventions
- Dexamethasone
- Etoposide
- Ruxolitinib
Dexamethasone is already approved in European Union, United States, Canada, Japan for the following indications:
- Inflammation
- Allergic reactions
- Respiratory diseases
- Skin conditions
- Eye diseases
- Immune system disorders
- Inflammatory conditions
- Allergic states
- Respiratory diseases
- Blood disorders
- Neoplastic diseases
- Nervous system disorders
- Inflammation
- Allergic reactions
- Respiratory diseases
- Skin conditions
- Eye diseases
- Inflammatory conditions
- Allergic states
- Respiratory diseases
- Blood disorders
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, San Francisco
Lead Sponsor
Aaron Logan, MD
Lead Sponsor
Jerry Lee, MD, MSc, MPhil
Lead Sponsor
Incyte Corporation
Industry Sponsor
Steven Stein
Incyte Corporation
Chief Medical Officer since 2015
MD from University of Witwatersrand
Hervé Hoppenot
Incyte Corporation
Chief Executive Officer since 2014
MBA from ESSEC Business School