Actively Recruiting

Phase 2
Age: 0 - 22Years
All Genders
NCT05762640

Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)

Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-04-23

20

Participants Needed

1

Research Sites

172 weeks

Total Duration

On this page

Sponsors

A

Assistance Publique - Hôpitaux de Paris

Lead Sponsor

U

URC-CIC Paris Descartes Necker Cochin

Collaborating Sponsor

AI-Summary

What this Trial Is About

The purpose of this project is to study the survival of patients until Haematopoietic Stem Cell Transplantation following the use of Ruxolitinib as first-line treatment associated to corticosteroids in primary HLH.

CONDITIONS

Official Title

Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)

Who Can Participate

Age: 0 - 22Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient aged 0 to 22 years
  • Diagnosis of HLH confirmed by either a genetic diagnosis predisposing to primary HLH or abnormal expression of specific proteins or positive family history
  • Alternatively, presence of at least 5 of 8 HLH diagnostic criteria including fever, splenomegaly, cytopenia affecting at least two blood cell types, low hemoglobin, low platelets, low neutrophil count, abnormal triglyceride or fibrinogen levels, haemophagocytosis in tissue samples, decreased NK function, high ferritin, or activated T cells
  • No previous specific treatment for HLH syndrome
  • Use of effective contraception for patients of childbearing age during the trial and specified periods after
  • Informed consent given by legal representative or adult participant
  • Affiliation to Social Security
Not Eligible

You will not qualify if you...

  • Previous treatment with ATG, Alemtuzumab, Etoposide, JAK-inhibitors, rifampicin, anti-Interferon gamma antibodies, St. John's Wort, or strong CYP3A4 inducers
  • Previous corticosteroid or cyclosporine A treatment for more than 14 days
  • Isolated central nervous system disease
  • Contraindication to Ruxolitinib or corticosteroids including hypersensitivity
  • Pregnant or lactating female patients
  • Any infectious condition except infection triggering HLH
  • Severe renal impairment without dialysis
  • Grade 4 hepatic failure
  • Past or active tuberculosis
  • Known rheumatologic disorder or active malignancy
  • Use of another investigational agent or enrollment in another treatment protocol
  • Inability to tolerate oral or nasogastric drug administration

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Hopital Necker Enfants malades

Paris, Île-de-France Region, France, 75015

Actively Recruiting

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Research Team

D

Despina MOSHOUS, MD, PhD

CONTACT

L

Laure CHOUPEAUX

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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