Intermediate-size Expanded Access Program (EAP), Mesenchymal Stromal Cells (MSC) for Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)

Sponsor

Mesoblast International Sàrl (Industry)

Overall Status

Available

CT.gov ID

NCT04456439

Collaborator

(none)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Multisystem Inflammatory Syndrome in Children (MIS-C) Associated With Coronavirus Disease (COVID-19)	Biological: Remestemcel-L Drug: Hydrocortisone Drug: Diphenhydramine

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.

Study Design

Study Type:

Expanded Access

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)

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Months to 17 Years

Sexes Eligible for Study:

All

Inclusion Criteria

2 months to 17 years of age, inclusive
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
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

If on mechanical ventilation or ECMO, ≤72 hours post initiation of the respiratory support device

Exclusion Criteria

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.
Females who are pregnant or lactating
Body mass index (BMI) ≥40 kilograms per square meter (kg/m^2)
Known hypersensitivity to dimethyl sulfoxide (DMSO) or to porcine or bovine proteins
Aspartate aminotransferase/alanine transaminase (AST/ALT) ≥5x upper limit of normal (ULN)
Creatinine clearance <30 mL/min
Serum creatinine >2 mg/dL
Any end-stage organ disease which in the opinion of the treating physician may possibly affect the safety of the remestemcel-L treatment.