Treatment of non severe Hemophagocytosis Lymphohistiocytosis with ITACITINIB a Phase II prospective trial

Phase 2

Not yet recruiting

• Conditions: Adults patients having non severe HLH

• Registration Number: 2024-513173-44-00
• Lead Sponsor: Assistance Publique Hopitaux De Paris, Assistance Publique Hopitaux De Paris

Brief Summary

Overall response rate (RR) defined as achieving a Complete (CR) or a Partial response (PR) at day 15 of ITACITINIB treatment on clinical and biological symptoms of primitive/refractory/relapses in non-severe adults HLHs

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-07-29
• Last Update Posted: 2025-03-17

Recruitment & Eligibility

• Status: Authorised, recruitment pending
• Sex: Not specified
• Target Recruitment: 70

Inclusion Criteria

Patients age >= 18 years, with front line or recurrent sporadic HLH, without or awaiting diagnosis of associated pathologies that may benefit from specific treatment, without severity criteria, without organ failure

Major criteria: (i) Cytological features of hemophagocytosis (ii) Fever (iii) Splenomegaly

Minor criteria: (i) Adenopathies (ii) Cytopenia> 2 lineages: - Haemoglobin < 9 g/dl, - Platelets < 100 000/mm3 - Neutrophils < 1000/mm3 (iii) Hypertriglyceridemia > 3 mmol/l and/or hypofibrigenemia < 1.5 g/l (iv) Ferritin>500 μg/l

HScore ≥ 169

Patient is willing to provide written informed consent prior to enrolment and agrees to follow the protocol

Patient known to have systemic juvenile idiopathic arthritis are classified as having HLH if they met the following criteria: (i) Ferritin > 684 ng/mL and (ii) any 2 of the following: - Platelet count < 181 000/mm3, - ASAT > 48 UI/ml, - Triglyceride > 4 mmol/L, - Fibrinogen < 3.6 g/L

Negative pregnancy test for woman of childbearing potential, woman of childbearing potential should have reliable contraception for the duration of the study

Be either affiliated to, or a beneficiary of, a social security category

Exclusion Criteria

Organ failure (at least one of those criteria): - Confusion, glasgow < 13, - Organic kidney failure requiring dialysis or KDIGO > 2, - Liver failure (Factor V < 50%), - Heart failure requiring catecholamines, - Respiratory failure requiring assisted ventilation

HIV infection with positive viral charge (note: in case of negative HIV antibody, it is not necessary to perform the PCR HIV RNA)Protected adults (including individual under guardianship by court order)

Vulnerable adults, under a safeguard of justice measure

Adults deprived of their liberty by judicial or administrative decision

Persons under psychiatric care without their consent

Persons admitted to social institution for purposes other this research

Adults under legal protection (guardianship or curatorship)

Persons unable to express their consent

Fibrinogen < 0.50 g/l, platelets <20G/L

Patient participating in another investigational therapeutic study

Breastfeeding women

Women with a positive pregnancy test or not willing to take contraceptive measures

Known allergies, hypersensitivity, or intolerance to any of the ITACITINIB or excipients, or similar compounds

Current or history of recurrent infections, including HBV, HCV

Participants with active HBV infection that requires treatment or who are at risk for HBV reactivation (ie Positive HBs Ag serology)

Candidates positive for HCV antibody and positive PCR RNA HCV (note: in case of negative result of HCV antibody, it is not necessary to perform the PCR HCV RNA)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Overall response rate (RR) achievement of either a Complete (CR) or a Partial response (PR) at day 15 of ITACITINIB treatment on clinical and biological symptoms of primitive/refractory/relapses in non-severe adults HLHs		Overall response rate (RR) achievement of either a Complete (CR) or a Partial response (PR) at day 15 of ITACITINIB treatment on clinical and biological symptoms of primitive/refractory/relapses in non-severe adults HLHs

Trial Locations

Locations (15)

Assistance Publique Hopitaux De Paris; 🇫🇷 Paris, France

Centre Hospitalier Universitaire D'Angers; 🇫🇷 Angers, France

Centre Hospitalier Universitaire De Bordeaux; 🇫🇷 Bordeaux, France

Centre Hospitalier Universitaire De Lille; 🇫🇷 Lille Cedex, France

Les Hopitaux Universitaires De Strasbourg; 🇫🇷 Strasbourg, France

Centre Hospitalier Universitaire Amiens Picardie; 🇫🇷 Amiens Cedex 1, France

Centre Hospitalier Bretagne Atlantique; 🇫🇷 Vannes, France

Centre Hospitalier Sud Francilien; 🇫🇷 Corbeil Essonnes, France

Hospital Foch; 🇫🇷 Suresnes, France

Centre Hospitalier Universitaire De Rennes; 🇫🇷 Rennes, France

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Assistance Publique Hopitaux De Paris

🇫🇷Paris, France

Sophie GEORGIN-LAVIALLE

Site contact

0156017431

sophie.georgin-lavialle@aphp.fr

Marc MICHEL

Site contact

0149812076

marc.michel@aphp.fr

Olivier HERMINE

Site contact

0144494000

olivier.hermine@aphp.fr

Coralie BLOCH-QUEYRAT

Site contact

0148955495

coralie.bloch-queyrat@aphp.fr

Olivier LAMBOTTE

Site contact

0149812904

olivier.lambotte@aphp.fr

Bruno FAUTREL

Site contact

0142177801

bruno.fautrel@aphp.fr

Sébastien RIVIERE

Site contact

0149283320

sebastien.riviere@aphp.fr

Fanny LANTERNIER

Site contact

0142160159

fanny.lanternier@ap-hp.fr

Julien SCHMIDT

Site contact

0148955241

julien.schimdt@aphp.fr

David BOUTBOUL

Site contact

0142499690

david.boutboul@aphp.fr