Official Title
Intermediate-size Expanded Access of Remestemcel-L, Human Mesenchymal Stromal Cells, for Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)
Brief 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.

Detailed Description

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.

Available
Intermediate-size Population
Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)

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®

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.

Eligibility Gender
All
Eligibility Age
Minimum: 2 Months ~ Maximum: 17 Years
Contacts

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.

Mesoblast International Sàrl
NCT Number
Keywords
MIS-C
Covid-19
MSC
remestemcel-L
Mesoblast
mesenchymal
Cellular therapy
cells
MeSH Terms
COVID-19
Coronavirus Infections
Syndrome
Diphenhydramine
Promethazine
Remestemcel-l
Hydrocortisone