NCT05762640 Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)
| NCT ID | NCT05762640 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Haemophagocytic Lymphohistiocytosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2024-11-10 |
| Primary Completion | 2027-05 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 20 participants in total. It began in 2024-11-10 with a primary completion date of 2027-05.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria * Patient aged 0 to 22 years * Patient with HLH syndrome confirmed by at least one of the two criteria: 1. 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 2. 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. Exclusion Criteria * 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
Contact & Investigator
Despina MOSHOUS, MD, PhD
STUDY CHAIR
Hôpital Necker-Enfants Malades
Frequently Asked Questions
Who can join the NCT05762640 clinical trial?
This trial is open to participants of all sexes, up to 22 Years, studying Haemophagocytic Lymphohistiocytosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05762640 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05762640 currently recruiting?
Yes, NCT05762640 is actively recruiting participants. Contact the research team at despina.moshous@aphp.fr for enrollment information.
Where is the NCT05762640 trial being conducted?
This trial is being conducted at Paris, France.
Who is sponsoring the NCT05762640 clinical trial?
NCT05762640 is sponsored by Assistance Publique - Hôpitaux de Paris. The principal investigator is Despina MOSHOUS, MD, PhD at Hôpital Necker-Enfants Malades. The trial plans to enroll 20 participants.