Steroid Refractory GVHD
Conditions
Brief summary
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)
Interventions
BIOLOGICALInolimomab (Leukotac)
* 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.
Sponsors
ElsaLys Biotech
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
28 Days to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall response | Day 29 post inclusion | Complete response + very good partial response + partial response |
Outcome results
None listed