Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Hemophagocytic Lymphohistiocytosis
In brief
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.
Key facts
- Study ID
- NCT04551131
- Run by
- St. Jude Children's Research Hospital
- People needed
- 10
- Starts
- 2021-07-13
- Expected to finish
- 2026-08-01
- Last updated by the study team
- 2026-04-24
Who can join
Age: 0 and older, up to 22. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Phoenix Children's Hospital — Phoenix, Arizona, United States
- Children's Hospital of Orange County — Orange, California, United States
- University of California San Francisco — San Francisco, California, United States
- Children's National Medical Center — Washington D.C., District of Columbia, United States
- John Hopkins University — Baltimore, Maryland, United States
- Dana-Farber Cancer Institute — Boston, Massachusetts, United States
- Cohen Children's Medical Center — New Hyde Park, New York, United States
- Levine Children's Hospital — Charlotte, North Carolina, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States
- University of Texas Southwestern Medical Center — Dallas, Texas, United States
- Texas Children's Hospital — Houston, Texas, United States
- Children's Wisconsin/Medical College of Wisconsin — Milwaukee, Wisconsin, United States
Full record on ClinicalTrials.gov
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