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

ConditionHaemophagocytic Lymphohistiocytosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeUp to 22
SponsorAssistance Publique - Hôpitaux de Paris

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

Patient aged 0 to 22 years

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

Fever

Splenomegaly

Disqualifiers

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.

History of hypersensitivity to the active substance or to any of the excipients

Trial design

Design model

Single group

Treatments tested in this trial

  • Ruxolitinib

    Drug

    Form: tablets, 50 mg/m2/day in two administrations. Maximum dose is 100 mg/day. Administration in association with Methylprednisolone IV (or Prednisolone PO) starting at 2 mg/kg/day in two administrations. Duration of treatment: until D-1 of conditioning for allogeneic HSCT OR 9weeks for patients who are not eligible for HSCT.

Treatment groups

20 Participants
are divided into 1 treatment group
Group A: RuxolitinibExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Survival until HSCT

Time frame
Day 0 until HSCT, up to 8 weeks

Secondary outcomes

1

Rate of patients achieving a complete response

To evaluate the efficacy of Ruxolitinib

Time frame
Day 7, Day 14, Day 21, Day 28, Week 8, and Day-1 of the conditioning for HSCT
2

Rate of patients achieving a partial response

To evaluate the efficacy of Ruxolitinib

Time frame
Day 7, Day 14, Day 21, Day 28, Week 8, and Day-1 of the conditioning for HSCT
3

Delay to obtain complete response

To evaluate the efficacy of Ruxolitinib

Time frame
Day 0 up to Day-1 of the conditioning for HSCT
4

Delay to obtain partial response

To evaluate the efficacy of Ruxolitinib

Time frame
Day 0 up to Day-1 of the conditioning for HSCT

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Assistance Publique - Hôpitaux de Paris

Lead sponsor

URC-CIC Paris Descartes Necker Cochin

Collaborator

Source ClinicalTrials.gov
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