The objectives of this intermediate-size expanded access protocol are to assess thesafety 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.
Biological: Remestemcel-L
Participants may receive up to 2 infusions of 2 x 10^6 remestemcel-L within a 5-day
period.
Drug: Hydrocortisone
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.
Drug: Diphenhydramine
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 (<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.
Elizabeth Burke, ANP-C
646-315-1725
elizabeth.burke@mesoblast.com
Kenneth M. Borow, MD
610-299-7855
ken.borow@mesoblast.com
Kenneth M. Borow, MD, Study Director
Mesoblast, Inc.