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

Recruiting now · Phase 2

Conditions studied: Haemophagocytic Lymphohistiocytosis

In brief

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.

Key facts

Study ID

NCT05762640

Run by

Assistance Publique - Hôpitaux de Paris

People needed

20

Starts

2024-11-10

Expected to finish

2028-03-01

Last updated by the study team

2026-04-23

Who can join

Age: any, up to 22. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patient aged 0 to 22 years
• Patient with HLH syndrome confirmed by at least one of the two criteria:
• Confirmed genetic diagnosis of a condition predisposing to primary HLH (see table 1 and table 2) or abnormal expression of perforin, MUNC13-4, SAP or XIAP in FACS and/or positive family history OR
• Presence of at least 5 of the 8 following HLH diagnostic criteria:
• Fever
• Splenomegaly
• Cytopenia (affecting at least two cell lineages)
• Haemoglobin < 9 g/dl (<10 g/dL in neonates)
• Platelets < 100,000/µL
• Absolute neutrophil count (ANC) < 1,000/µL
• Hypertriglyceridemia and/or hypofibrinogenemia
• Fasting triglycerides ≥ 3 mmol/l
• Fibrinogen <1.5 g/L
• Haemophagocytosis found in a histological sample (without evidence of a malignant process or an underlying rheumatic disorder)
• Decreased or absent NK function
• Ferritin ≥ 500 µg/l
• Presence of activated T cells in the immune phenotyping as evidenced by expression of the activation marker DR (superior to the normal value of the laboratory) OR CD25 soluble (sIL-2 receptor) ≥ 2,400 U/mL.
• Patient with no previous specific treatment for HLH syndrome
• For patients of childbearing age : using an effective method of contraception during the trial, and through to 90 days after EOS for male participants and 30 days after EOS for female participants
• Freely given, informed and written consent of legal representative of the participant or consent of the adult participant
• Affiliation to Social Security.

You may not qualify if…

• Previous treatment with ATG, Alemtuzumab, Etoposide, JAK-inhibitors, rifampicin and/or anti-Interferon gamma antibodies. St. John's Wort, or any other strong CYP3A4 inducers.
• Previous treatment with corticosteroids and/or cyclosporine A for more than 14 days
• Isolated CNS disease.
• Contraindication to receive Ruxolitinib:
• History of hypersensitivity to the active substance or to any of the excipients
• Pregnant or lactating female patient
• Contraindication to receive methylprednisolone or prednisolone
• History of hypersensitivity to the active substance or to any of the excipients
• Any infectious condition with the exception of infections, which are the trigger for lymphohistiocytic activation.
• Patient with acute very severe renal impairment (Creatinine Clearance <15 mL/min/1.73m²) who are NOT receiving dialysis.
• Patient with Grade 4 hepatic failure according to the CTCAE v5.0 of 27 November 2017 (Life-threatening consequences; moderate to severe encephalopathy; coma)
• Past or know active tuberculosis
• Known rheumatologic disorder.
• Known active malignancy.
• Patient who is taking another investigational agent or is enrolled in another treatment protocol.
• Patient who cannot tolerate administration of drugs PO or through NG

Where it is running

• Hopital Necker Enfants malades — Paris, Île-de-France Region, France (enrolling)

Full record on ClinicalTrials.gov

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