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

Conditions

• Hemophagocytic Lymphohistiocytosis

Eligibility Criteria

Sex

ALL

Age

6 Weeks - 22 Years

Healthy Volunteers

Not accepted

Interventions

• Ruxolitinib — DRUG
  Given orally (PO) or per nasogastric tube (NGT) twice a day for 8 weeks
• Dexamethasone — DRUG
  Given intravenously (IV) or orally (PO) twice a day for 8 weeks
• Etoposide — DRUG
  Given intravenously (IV) once a week for 8 weeks

Study Details

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 Dates

Start date

Jul 13, 2021

Status verified

Apr 2026

Primary completion

Nov 7, 2025

Completion

Aug 31, 2026

Study Design

Enrollment

10 participants (actual)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Frontline Arm
  Safety Phase: Patients with newly diagnosed HLH will receive ruxolitinib PO or NGT and dexamethasone, PO or IV. Etoposide IV will be added based on disease response. Expansion Phase: Patients with newly diagnosed HLH treatment will begin with ruxolitinib PO or NGT at the MTD dose. Dexamethasone will be administered PO or IV. Etoposide IV will be added based on disease response.
• Experimental: Salvage Arm
  Patients with relapsed/refractory HLH will receive ruxolitinib PO or NGT and dexamethasone PO or IV. Etoposide IV will be added based on disease response.

Primary Outcome Measure

Complete Response (CR)/Complete Response with Incomplete Hematologic Recovery (CRi) [ Time Frame: 8 weeks ]

Locations (13)

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