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

Start 3 May 2022 · End 25 Jul 2025 · Status Ended · 1 EU/EEA countries · 26 sites

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)

Status Ended

Participants planned 70

Countries 1

Sites 26

Adults patients having non severe HLH

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

Key facts

Sponsor: Assistance Publique Hopitaux De Paris, Assistance Publique Hopitaux De Paris
Participant type: Patients
Age range: 18-64 years, 65+ years
Gender: Male and Female
Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
Trial duration: 3 May 2022 → 25 Jul 2025
Decision date (initial): 2024-07-29
Transition trial: Yes
Low-intervention: No
Rare-disease indication: No
Vulnerable population: No

External identifiers

EU CT number: 2024-513173-44-00
EudraCT number: 2021-000407-20
ClinicalTrials.gov: NCT05063110

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy

Conditions and MedDRA coding

Adults patients having non severe HLH

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

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 16

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
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)
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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Itacitinib

PRD1600053 · Product

Active substance: Itacitinib
Pharmaceutical form: TABLET
Route of administration: ORAL USE
Max daily dose: 400 mg milligram(s)
Max total dose: 72 g gram(s)
Max treatment duration: 6 Month(s)
Authorisation status: Not Authorised
MA holder: INCYTE CORPORATION
Paediatric formulation: No
Orphan designation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation: Assistance Publique Hopitaux De Paris
Address: Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
City: Paris Cedex 10
Postcode: 75475
Country: France

Scientific contact point

Organisation: Assistance Publique Hopitaux De Paris
Contact name: Coordinating Investigator

Public contact point

Organisation: Assistance Publique Hopitaux De Paris
Contact name: Coordinating Investigator

Assistance Publique Hopitaux De Paris

Sponsor organisation: Assistance Publique Hopitaux De Paris
Address: 1 Avenue Claude Vellefaux
City: Paris
Postcode: 75010
Country: France

Locations

1 EU/EEA country · 26 investigational sites

By country

Country	MS status	Planned subjects	Sites
France	Ended	70	26
Rest of world	—	0	—