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

NCT ID: NCT04456439

Last Updated: 2025-10-31

Basic Information

• Recruitment Status: NO_LONGER_AVAILABLE
• Study Classification: EXPANDED_ACCESS

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.

Conditions

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

Interventions

Remestemcel-L

Participants may receive up to 2 infusions of 2 x 10\^6 remestemcel-L within a 5-day period.

Intervention Type: BIOLOGICAL

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.

Intervention Type: 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.

Intervention Type: DRUG

Other Intervention Names

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.

• Minimum Eligible Age: 2 Months
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mesoblast International Sàrl

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kenneth M. Borow, MD

Role: STUDY_DIRECTOR

Mesoblast, Inc.

References

Eckard AR, Borow KM, Mack EH, Burke E, Atz AM. Remestemcel-L Therapy for COVID-19-Associated Multisystem Inflammatory Syndrome in Children. Pediatrics. 2021 May;147(5):e2020046573. doi: 10.1542/peds.2020-046573. Epub 2021 Feb 12.

Reference Type: RESULT

PMID: 33579813 (View on PubMed)

Other Identifiers

MSB-MSC-MISC001

Identifier Type: -

Identifier Source: org_study_id