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Hemophagocytic Lymphohistiocytosis clinical trials at UCSF

1 research study open to eligible people

Hemophagocytic lymphohistiocytosis is a rare disease affecting the immune system. UCSF is doing a clinical trial to study a new treatment plan. The goal is to see how well it works and if it has any side effects. The treatment combines two medications.

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  • Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

    open to eligible people ages up to 22 years

    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.

    San Francisco, California and other locations

Our lead scientists for Hemophagocytic Lymphohistiocytosis research studies include .

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