NCT06160791

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

Recruiting now Phase 2 Last updated 13 April 2026

What this trial tests

Phase 2 trial testing Ruxolitinib in Hemophagocytic Lymphohistiocytoses in 36 participants. Currently enrolling.

Timeline

1 October 2024

Primary endpoint
31 December 2027

30 November 2029

Quick facts

Lead sponsor	Jerry Lee, MD, MSc, MPhil
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	36
Start date	1 October 2024
Primary completion	31 December 2027
Estimated completion	30 November 2029
Sites	3 locations across United States

Drugs / interventions tested

• Ruxolitinib (RUXOLITINIB) — full drug profile →
• Etoposide (etoposide) — full drug profile →
• Dexamethasone (dexamethasone) — full drug profile →
• Non-interventional Imaging
• Research Biopsy
• Biospecimen Collection — full drug profile →

Conditions studied

• Hemophagocytic Lymphohistiocytoses — all drugs for Hemophagocytic Lymphohistiocytoses →

Sponsor

Jerry Lee, MD, MSc, MPhil

Who can join

18 and older, any sex, with Hemophagocytic Lymphohistiocytoses. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Hemophagocytic lymphohistiocytosis: current treatment advances, emerging targeted therapy and underlying mechanisms.
   Wu Y, Sun X, Kang K, Yang Y, et al · · 2024 · cited 47× · PMID 39511607 · DOI 10.1186/s13045-024-01621-x
2. Late-onset hemophagocytic lymphohistiocytosis associated with monoallelic STX11 mutation in an adult: a case report and rationale for early allogeneic hematopoietic cell transplantation.
   Zaucha JM, Mital A, Taszner M, Ogłoza A, et al · · 2025 · PMID 40970921 · DOI 10.1007/s00277-025-06599-1