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.
Participants may receive up to 2 infusions of 2 x 10^6 remestemcel-L within a 5-day period.
Participants who are not currently taking a corticosteroid will receive hydrocortisone, 0.5-1 milligram per kilogram (mg/kg), up to 50 mg IV, at least 30 minutes prior to the infusion of remestemcel-L.
Participants will receive diphenhydramine, 0.5-1 mg/kg, up to 50 mg IV, at least 30 minutes prior to the infusion of remestemcel-L.
Other Name: Benadryl®
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 ( 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 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.
Kenneth M. Borow, MD