Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)

Summary

This phase II trial tests the effects of ruxolitinib in combination with a de-intensified HLH-94 drug regimen has on patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH), a disorder caused by dysregulated immune responses (that is, immune responses that are too strong and cause inflammatory damage to normal tissues). The therapy used for HLH decreases the activity of the immune system. Ruxolitinib is a type of drug called a kinase inhibitor. It works by blocking the signals that cause inflammatory cells to multiply. De-intensified HLH-94 is a treatment regimen that includes 4 weeks of dexamethasone with the dose being decreased each week, and up to 4 weeks of etoposide. This combination is commonly used to treat HLH. Dexamethasone is a steroid medication that works by fighting inflammation. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and deoxyribonucleic acid (DNA) repair and may kill cancer cells and is used to kill the types of white blood cells in HLH that are attacking the body. Giving ruxolitinib in combination with a de-intensified HLH-94 drug regimen may reduce toxic exposure to therapy while maintaining efficacy in patients with HLH.

Conditions

• Hemophagocytic Lymphohistiocytoses

Interventions

DRUG

Ruxolitinib

Administered Orally (PO)

DRUG

Etoposide

Administered IV

DRUG

Dexamethasone

Administered PO or IV

PROCEDURE

Non-interventional Imaging

Participants undergo abdominal ultrasound and/or magnetic resonance imaging (MRI)

PROCEDURE

Research Biopsy

Bone marrow biopsy and lymph node biopsy will be obtained during screening and as clinically indicated throughout the trial.

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

Sponsors & Collaborators

• Incyte Corporation

  collaborator INDUSTRY

• UC Hematological Malignancies Consortium (UCHMC)

  collaborator UNKNOWN

• Jerry Lee, MD, MSc, MPhil

  lead OTHER

Principal Investigators

• Jerry Lee, MD · University of California, San Francisco

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-10-01

Primary Completion

2027-12-31

Completion

2029-11-30

FDA Drug

Yes

Countries

• United States

Study Locations