Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis
Status:
Recruiting
Trial end date:
2026-08-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborators:
Incyte Corporation North American Consortium for Histiocytosis