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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04456439
Expanded Access Status : Available
First Posted : July 2, 2020
Last Update Posted : October 23, 2020
Sponsor:
Information provided by (Responsible Party):
Mesoblast, Ltd. ( Mesoblast International Sàrl )

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
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.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
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)



Intervention Details:
  • Biological: Remestemcel-L
    Participants may receive up to 2 infusions of 2 x 10^6 remestemcel-L within a 5-day period.
    Other Name: Ryoncil™
  • 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®

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04456439


Contacts
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Contact: Elizabeth Burke, ANP-C 646-315-1725 elizabeth.burke@mesoblast.com
Contact: Kenneth M. Borow, MD 610-299-7855 ken.borow@mesoblast.com

Sponsors and Collaborators
Mesoblast International Sàrl
Investigators
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Study Director: Kenneth M. Borow, MD Mesoblast, Inc.
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Responsible Party: Mesoblast International Sàrl
ClinicalTrials.gov Identifier: NCT04456439    
Other Study ID Numbers: MSB-MSC-MISC001
First Posted: July 2, 2020    Key Record Dates
Last Update Posted: October 23, 2020
Last Verified: October 2020
Keywords provided by Mesoblast, Ltd. ( Mesoblast International Sàrl ):
MIS-C
COVID-19
MSC
remestemcel-L
Mesoblast
mesenchymal
cellular therapy
cells
Additional relevant MeSH terms:
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Coronavirus Infections
Syndrome
Disease
Pathologic Processes
Coronaviridae Infections
Nidovirales Infections
RNA Virus Infections
Virus Diseases
Diphenhydramine
Promethazine
Hydrocortisone
Anti-Inflammatory Agents
Sleep Aids, Pharmaceutical
Hypnotics and Sedatives
Central Nervous System Depressants
Physiological Effects of Drugs
Anesthetics, Local
Anesthetics
Sensory System Agents
Peripheral Nervous System Agents
Antiemetics
Autonomic Agents
Gastrointestinal Agents
Histamine H1 Antagonists
Histamine Antagonists
Histamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Anti-Allergic Agents
Antipruritics