JAK Inhibitor in Acquired Hemophagocytic synDrome in the Intensive Care Unit

Sponsor
Assistance Publique - Hôpitaux de Paris
Study ID
NCT06244862
Phase
PHASE2
Status
Unknown

Conditions

  • Hemophagocytic Syndromes

Eligibility Criteria

Sex
ALL
Age
18 Years - 100 Years
Healthy Volunteers
Not accepted

Interventions

  • Ruxolitinib — DRUG
    Oral ruxolitinib twice a day (10 mg x 2 during 28 days) in association with standard of care in HS.

Study Details

Hemophagocytic syndrome (HS) is a rare condition that can be responsible for severe organ failure. Therapeutic guidelines are mainly based on observational studies and expert opinions: no therapeutic advance has been developed for years, explaining why mortality in HS remains high (Intensive Care Unit mortality ranging from 40 to 70%). If etoposide remains the gold standard in critically ill HS patients, nearly 20% of patients are refractory to this therapy: treatment escalation is common, most often requiring the administration of intensive treatments generating high toxicity. Ruxolitinib is the first approved JAK inhibitor. It has been associated with improvement of HS manifestations and survival in a pre-clinical murine model. Data in humans are scarce but promising. The aim is to demonstrate that ruxolitinib, in association with standard of care, may reverse organ failure (as represented by Sequential Organ Failure Assessment (SOFA) score) better than standard of care alone in critically ill patients with acquired HS.

Key Dates

Start date
Feb 1, 2024
Status verified
Dec 2023
Primary completion
Aug 8, 2025
Completion
Feb 1, 2026

Study Design

Enrollment
42 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Interventional medicinal product

Primary Outcome Measure

Survival with a decrease in SOFA score ≥ 3 points [ Time Frame: At day 7 ]

Central Contacts