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

NCT04551131 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-04-24

No results posted yet for this study

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.

Conditions

  • Hemophagocytic Lymphohistiocytosis

Interventions

DRUG

Ruxolitinib

Given orally (PO) or per nasogastric tube (NGT) twice a day for 8 weeks

DRUG

Dexamethasone

Given intravenously (IV) or orally (PO) twice a day for 8 weeks

DRUG

Etoposide

Given intravenously (IV) once a week for 8 weeks

Sponsors & Collaborators

Principal Investigators

  • Melissa Hines, MD · St. Jude Children's Research Hospital

  • Kim E. Nichols, MD · St. Jude Children's Research Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Weeks
Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-13
Primary Completion
2025-11-07
Completion
2026-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04551131 on ClinicalTrials.gov