Ruxolitinib-Based Regimen for Hemophagocytic Lymphohistiocytosis
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking rifampin, St. John's Wort, or another investigational agent. If you are on these, you may need to stop them before joining the trial.
What data supports the effectiveness of the drug Ruxolitinib for treating hemophagocytic lymphohistiocytosis?
Research shows that Ruxolitinib, when used in combination with other drugs, has been effective in treating hemophagocytic lymphohistiocytosis (HLH), with studies reporting high response rates and improved survival in patients. It has been particularly noted for its ability to modulate cytokines (proteins important in cell signaling) and has shown promise in both primary and secondary forms of HLH.12345
Is the Ruxolitinib-based regimen safe for treating hemophagocytic lymphohistiocytosis?
Ruxolitinib has been used in various studies for treating hemophagocytic lymphohistiocytosis (HLH) and is generally considered safe, with patients tolerating it well. The most common side effects are related to blood cell counts, but it has shown potential to reduce the intensity of chemotherapy needed.23567
What makes the Ruxolitinib-based drug regimen unique for treating hemophagocytic lymphohistiocytosis?
The Ruxolitinib-based regimen is unique because it includes Ruxolitinib, a drug that modulates cytokines (proteins important in cell signaling) and has a safer profile compared to traditional high-dose treatments. This regimen can reduce the intensity of chemotherapy needed and is effective as a bridge to stem-cell transplantation, especially in children with primary hemophagocytic lymphohistiocytosis.12357
What is the purpose of this trial?
This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH) or as Salvage therapy for patients with relapsed/refractory HLH.Primary Objective* To determine the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with newly diagnosed HLH.Secondary Objectives* To describe the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with relapsed/refractory HLH.* To describe the overall response and outcome for patients with newly diagnosed or relapsed/refractory HLH who are treated with this response-adapted ruxolitinib-containing regimen.Exploratory Objectives* To estimate the pharmacokinetic (PK) parameters of ruxolitinib, assess covariates of ruxolitinib pharmacokinetics, and test whether the drug's effectiveness is correlated with systemic drug exposure.* To query specific immunologic biomarkers and determine whether the levels of these biomarkers correlate with disease response and outcome.
Research Team
Melissa Hines, MD
Principal Investigator
St. Jude Children's Research Hospital
Kim E. Nichols, MD
Principal Investigator
St. Jude Children's Research Hospital
Eligibility Criteria
This trial is for patients aged 6 weeks to 22 years with newly diagnosed or relapsed/refractory Hemophagocytic Lymphohistiocytosis (HLH). They must weigh at least 3 kg and be able to take oral medication or agree to an NG tube. Participants should meet specific HLH diagnostic criteria but not have received certain prior treatments, except steroids or anakinra. Pregnant individuals, those with severe organ dysfunction, active malignancy, or on other investigational drugs are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Safety Phase
Identify a feasible and safe dose of ruxolitinib in combination with dexamethasone and etoposide for newly diagnosed HLH patients
Expansion Phase
Administer ruxolitinib at the maximally tolerated dose with dexamethasone, adding etoposide based on response
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
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?
St. Jude Children's Research Hospital
Lead Sponsor
North American Consortium for Histiocytosis
Collaborator
Cures Within Reach
Collaborator
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