intermediate-size expanded access program (eap), mesenchymal stromal cells (msc) for multisystem inflammatory syndrome in children (mis-c) associated with coronavirus disease (covid-19)

Conditions: Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)

Summary: the objectives of this intermediate-size expanded access protocol are to assess the safety and efficacy of remestemcel-l in participants with mis-c associated with covid-19.

this intermediate-size expanded access protocol plans to treat approximately 50 children or adolescents, male and female, with mis-c associated with covid-19. participants who are 2 months to 17 years of age, inclusive, will be enrolled at multiple clinical sites across the united states.

Eligibility criteria:
inclusion criteria 1. 2 months to 17 years of age, inclusive 2. positive for current or recent sars-cov-2 (covid-19) infection by real-time reverse transcription polymerase chain reaction (rt-pcr), serology, or antigen test; or covid-19 exposure within the 4 weeks prior to the onset of symptoms and no alternative plausible diagnoses 3. presenting with: * fever (\>38.0°c or \>100.4°f for ≥24 hours) or reporting subjective fever lasting ≥24 hours * laboratory evidence of inflammation with high sensitivity c-reactive protein (hscrp) ≥4.0 milligrams per deciliter (mg/dl) and associated abnormalities of at least one of the following: * elevated erythrocyte sedimentation rate (esr) * elevated fibrinogen * elevated procalcitonin * elevated d-dimer * elevated ferritin * elevated lactic dehydrogenase (ldh) * elevated interleukin 6 (il-6) * elevated neutrophils * reduced lymphocytes * low albumin * clinically severe multisystem illness requiring hospitalization with evidence for cardiac involvement plus at least one other organ involvement (renal, respiratory, hematologic, gastrointestinal, dermatologic or neurological) * cardiac involvement is defined as reduced left ventricular ejection fraction (\<55%) in addition to at least one of the following: * increased troponin i, * increased n-terminal pro-b-type natriuretic peptide (nt-probnp) or bnp and/or * echocardiographic and/or other imaging evidence of left anterior descending coronary artery (lad) and/or right coronary artery (rca) dilation associated with a z-score \> 2.5 4. if on mechanical ventilation or ecmo, ≤72 hours post initiation of the respiratory support device exclusion criteria 1. documented other microbial cause for mis-c including bacterial sepsis, staphylococcal or streptococcal shock syndromes, or infections associated with myocarditis such as enterovirus. of importance, waiting for results of these investigations should not delay initiation of remestemcel-l therapy. 2. females who are pregnant or lactating 3. body mass index (bmi) ≥40 kilograms per square meter (kg/m\^2) 4. known hypersensitivity to dimethyl sulfoxide (dmso) or to porcine or bovine proteins 5. aspartate aminotransferase/alanine transaminase (ast/alt) ≥5x upper limit of normal (uln) 6. creatinine clearance \<30 ml/min 7. serum creatinine \>2 mg/dl 8. any end-stage organ disease which in the opinion of the treating physician may possibly affect the safety of the remestemcel-l treatment.

Keywords: mis-c covid-19 msc remestemcel-l mesoblast mesenchymal cellular therapy cells