AT1001 for the Treatment of COVID-19 Related MIS-C

NCT05022303 | Terminated Phase 2 FDA Drug

• 1 other identifier: 2021P002143
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The primary aim of this study is to evaluate the efficacy and safety of AT1001 versus placebo in pediatric patients with SARS-CoV-2 infection who experience early signs of MIS-C and are at high risk of progression. AT1001 10 μg/kg/dose up to 500 μg/dose (rounded to the nearest 50 μg) or matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C.

Conditions

Covid19; Multisystem Inflammatory Syndrome in Children

Outcome Measures

Primary Outcomes (2)

• Evaluate the efficacy and safety of AT1001 versus placebo on mitigating symptoms of MIS-C

  • To evaluate the efficacy and safety of AT1001 versus placebo in pediatric patients with SARS-CoV-2 infection who experience early signs of MIS-C and are at high risk of progression.

  24 weeks

• Determine proportion of participants with improvement in MIS-C related GI symptoms and no progression of disease

  Improvement in GI symptoms is defined as: * Improvement in PedsQL GI Symptoms Scales score ≥48 hours, as determined by patient or caregiver response. * AND improvement in clinical manifestation of GI symptoms, as documented by complete physical exam or clinical assessment and clinical laboratory tests or imaging. No Progression of MIS-C is defined as: * No involvement of additional organ involvement, as identified by clinical assessment and clinical laboratory tests or imaging. * AND no new onset of new or worsening GI symptoms for ≥48 hours including nausea, vomiting, diarrhea, loss of appetite, and/or abdominal pain, as determined by patient or caregiver symptom report or worsening PedsQL GI Symptoms Scales scores.

  24 weeks

Secondary Outcomes (11)

• Determine the impact of AT1001 on infectious and inflammatory markers of MIS-C

  24 weeks

• Determine the impact of AT1001 on improvement in MIS-C symptoms for ≥48 hours, as determined by patient or caregiver symptom report on MIS-C Symptom Questionnaire.

  24 weeks

• Determine the impact of AT1001 on length of stay in hospital (days from baseline to readiness to discharge).

  24 weeks

• Determine the impact of AT1001 on re-presentation to medical care for MISC-related symptoms after discharge.

  24 weeks

• Determine the impact of AT1001 on need for escalation of care (eg, transfer from hospital ward to ICU; supplemental oxygen; mechanical ventilation).

  24 weeks

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C.

Drug: Placebo

Larazotide Acetate

EXPERIMENTAL

AT1001 10 μg/kg/dose up to 500 μg/dose (rounded to the nearest 50 μg) will be administered orally four times a day (QID) to the standard of care for MIS-C.

Drug: Larazotide Acetate

Interventions

Larazotide Acetate DRUG

AT1001 10 μg/kg/dose up to 500 μg/dose (rounded to the nearest 50 μg) will be administered orally four times a day (QID) to the standard of care for MIS-C.

Also known as: AT1001

Larazotide Acetate

Placebo DRUG

Matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C.

Placebo

Eligibility Criteria

• Age: 1 Month - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Pediatric patients with or without comorbidity
• Age ≥ 1 month to \< 21 years
• Confirmed MIS-C by signs and symptoms as detailed by the CDC Health Advisory (https://www.cdc.gov/mis-c/hcp/; May 14, 2020)
• Persistent fever/chills (\>38.0°C for ≥24 hours, or report of subjective fever lasting ≥24 hours); AND
• One or more laboratory parameters (evidence of inflammation); AND,
• i) elevated C-reactive protein (CRP) ii) elevated erythrocyte sedimentation rate (ESR) iii) elevated ferritin iv) elevated lactic acid dehydrogenase (LDH) v) elevated d-dimer vi) elevated fibrinogen vii) elevated procalcitonin viii) elevated interleukin 6 (IL-6) ix) increased neutrophils x) reduced lymphocytes xi) low albumin c) Evidence of clinically severe illness requiring hospitalization, with multisystem (\>2) organ involvement (cardiac, renal, respiratory, hematologic, gastrointestinal, dermatologic, or neurological)-MUST include GI symptoms, such as nausea, vomiting, diarrhea and/or abdominal pain; AND, d) No alternative plausible diagnoses; AND e) Positive for current or recent SARS-CoV-2 infection by RT-PCR, serology, or antigen test
• Subject (or legal authorized representative) capable of understanding and signing an informed consent form and assent form, when appropriate.

You may not qualify if:

• Female participants pregnant and/or lactating.
• Female participant has childbearing potential and is unwilling to use an acceptable method of birth control for the duration of the study.
• Participant has a significant co-morbid disease that by the Investigator's determination would make the participant unsuitable for enrollment, including unstable medical conditions.
• Participation in any other clinical investigation using an experimental drug within 30 days prior to screening or intends to participate in another clinical study while participating in AT1001 MIS-C 101 study.
• Have participated in a blood/plasma donation or blood loss greater than 400 mL within 90 days, or greater than 200 mL within 30 days prior to Screening.
• Known hypersensitivity to any of the formulation components of AT1001.

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

COVID-19; pediatric multisystem inflammatory disease, COVID-19 related

Interventions

larazotide acetate

Condition Hierarchy (Ancestors)

Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Participant and investigator blinded, Pharmacy unblinded.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pulmonary Attending Physician

Model Details: Placebo and drug arm (10 μg/kg/dose up to 500 μg/dose)

Study Record Dates

• First Submitted: August 9, 2021
• First Posted: August 26, 2021
• Study Start: October 1, 2021
• Primary Completion: May 15, 2024
• Study Completion: May 15, 2024
• Last Updated: June 13, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)