Evaluation of EFficacy and SaFEty of Leukotac (Inolimomab) in Pediatric Patients With SR-aGvHD

Study Overview
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Brief Summary
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A Phase 3, Multicenter, open label, study to Evaluate the EFficacy and SaFEty of Leukotac® (inolimomab) in pediatric patients with steroid resistant acute Graft versus Host Disease (SR-aGvHD)

Official Title
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A Phase 3, Multicenter, Open Label, Study to Evaluate the EFficacy and SaFEty of Leukotac® (Inolimomab) in Pediatric Patients With Steroid Resistant Acute Graft Versus Host Disease (SR-aGvHD)

Conditions
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Steroid Refractory GVHD

Intervention / Treatment
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* Biological: Inolimomab (Leukotac)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who develop a first episode of aGvHD stage ≥ II (Przepiorka et al., 1995), resistant to a first line therapy with steroids (lack of improvement after 5 days or progression after 3 days of treatment with corticosteroids at 2 mg/Kg methylprednisolone equivalent dose) Age 28 days to < 18 years old Allo-HSCT with any type of donor, stem cell source, GVHD prophylaxis or conditioning regimen Patients receiving Allo-HSCT for any indication (i.e. malignant or non-malignant disease) Signature of informed and written consent by the patient and/or by the patient's legally acceptable representative(s) Exclusion Criteria: Isolated stage 1 skin SR-aGvHD Overlap chronic GvHD as defined by the NIH Consensus Criteria (Jagasia MH, 2015) Acute GvHD after donor lymphocytes infusion (DLI) Relapsed/persistent malignancy requiring rapid immune suppression withdrawal Other systemic drugs than corticosteroids for GvHD treatment (including extra-corporeal photopheresis). Drugs already being used for GvHD prevention (e.g. calcineurin inhibitors) are allowed. Active, uncontrolled bacterial, viral, or fungal infection requiring systemic therapy Known allergy or intolerance to Leukotac of one of its ingredients Pregnancy: positive urinary or blood test in female of childbearing potential; lactation; absence of effective contraceptive method for female and male of childbearing potential Other ongoing interventional protocol that might interfere with

Ages Eligible for Study
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Minimum Age: 28 Days
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Inolimomab/Leukotac<br>Patient screening phase - Patients diagnosed with aGvHD will be identified. Only patients with SR-aGvHD will be finally included in the study. Treatment phase - Leukotac will be given up to D28 Primary Follow-up phase - Patient's response (CR and PR) will be evaluated at D29 post inclusion. Patients will then be followed for survival, long-term safety and chronic GvHD occurrence during 6 months after inclusion | Biological: Inolimomab (Leukotac)<br>* Patient screening phase - Patients diagnosed with aGvHD will be identified. Only patients with SR-aGvHD will be finally included in the study. Treatment phase - Leukotac will be given up to D28 Primary Follow-up phase - Patient's response (CR and PR) will be evaluated at D29 post inclusion. Patients will then be followed for survival, long-term safety and chronic GvHD occurrence during 6 months after inclusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response | Complete response + very good partial response + partial response | Day 29 post inclusion |