Evaluation of EFficacy and SaFEty of Leukotac (Inolimomab) in Pediatric Patients With SR-aGvHD
Phase 3
- Conditions
- Steroid Refractory GVHD
- Interventions
- Biological: Inolimomab (Leukotac)
- Registration Number
- NCT04289103
- Lead Sponsor
- ElsaLys Biotech
- 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 65
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Inolimomab/Leukotac Inolimomab (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
- Primary Outcome Measures
Name Time Method Overall response Day 29 post inclusion Complete response + very good partial response + partial response
- Secondary Outcome Measures
Name Time Method