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New treatment option for Haemophagocytic Lymphohistiocytosis
Official title Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)
ClinicalTrials.gov ID: NCT05762640
What this study is testing
What is Ruxolitinib?
Ruxolitinib is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for haemophagocytic lymphohistiocytosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 22
You may be able to join 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...
- Presence of at least 5 of the 8 following HLH diagnostic criteria:
- Fever
You likely can't join if
- Previous treatment with ATG, Alemtuzumab, Etoposide, JAK-inhibitors, rifampicin and/or anti-Interferon gamma antibodies. St. John's Wort, or any...
- 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
See the full eligibility criteria
- 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.
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Paris, Île-de-France Region, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 22 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Paris, Île-de-France Region, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.