Covid19Phase 2NCT05022303
AT1001 for the Treatment of COVID-19 Related MIS-C
A Phase 2 study testing Larazotide Acetate in people with Covid19 and Multisystem Inflammatory Syndrome in Children, run by Massachusetts General Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 21 years
- Conditions the study is for
- Covid19, Multisystem Inflammatory Syndrome in Children
- What is being tested?
- Larazotide Acetate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2021to 15 May 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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,
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: A Phase 2a (Proof of Concept), Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AT1001 for the Treatment of COVID-19 Related Multisystem Inflammatory Syndrome in Children (MIS-C)
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.
Read the detailed description
This is a Phase 2a (Proof of Concept), randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of AT1001 for use in hospitalized pediatric patients diagnosed with MIS-C. Eligible participants (N=20) will be treated with AT1001 or matching placebo orally four times a day (QID) for up to 21 days as an add-on to standard of care.
The study includes three phases:
- Screening/Baseline: The main purpose of this phase is to determine if the participant meets entry criterion after obtaining informed consent and obtain baseline assessments.
- Treatment: Eligible participants will be treated with AT1001 or matching placebo 10 μg/kg/dose QID up to 500 μg/dose (rounded to the nearest 50 μg) for 21 days as an add-on to standard of care for MIS-C.
- Follow-up through 24 weeks: The participant will return for a follow-up visits during weekly clinic visits during Week 1 through Week 3, with monthly telemedicine visits at Week 4, Week 8, Week 16 and Week 20, and clinic visits at Week 12 and Week 24.
Safety monitoring, including physical examination, vitals, and clinical laboratory testing will be performed during the screening phase, periodically during treatment phase and at the follow-up phase. Adverse events and concomitant medications will be recorded during the entire study.
Total duration of the participants' participation in the study is approximately 24 weeks (with 21 days treatment period). Total duration of the study is projected to be 12 months, dependent on enrollment timeline.
- Being tested
- Larazotide Acetate, Placebo
- Medical subject headings
- COVID-19, pediatric multisystem inflammatory disease, COVID-19 related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Larazotide AcetateDrug
Also called AT1001
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.
PlaceboDrug
Matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C. Receives: 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. Receives: Drug: Larazotide Acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 24 weeks
Secondary outcomes (11)
Determine the impact of AT1001 on infectious and inflammatory markers of MIS-C
Secondary aims of this study include determining the impact of AT1001 on infectious and inflammatory markers of MIS-C.
Measured: 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.
Measured: 24 weeks
Determine the impact of AT1001 on length of stay in hospital (days from baseline to readiness to discharge).
Measured: 24 weeks
Determine the impact of AT1001 on re-presentation to medical care for MISC-related symptoms after discharge.
Measured: 24 weeks
Determine the impact of AT1001 on need for escalation of care (eg, transfer from hospital ward to ICU; supplemental oxygen; mechanical ventilation).
Transfer from hospital ward to ICU; supplemental oxygen; mechanical ventilation.
Measured: 24 weeks
Determine the impact of AT1001 on change from baseline in additional organ system(s) involvement during acute presentation/hospitalization, as identified by clinical assessment and clinical laboratory tests.
Measured: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of IgM, IgG, and IgA antibodies against SARS CoV-2.
Measured: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of inflammatory markers (CRP, d-dimer, ferritin).
Measured: 24 weeks
Determine the impact of AT1001 on normalization of SARS-CoV-2 Spike, S1 and nucleocapsid.
Measured: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of zonulin.
Measured: 24 weeks
Determine the impact of AT1001 on change in mortality (all causes)
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2021
- Study start1 Oct 2021
- Primary completion15 May 2024
- Study completion15 May 2024
Why it stopped: Decline in MISC cases
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Covid19Studies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Placebo and drug arm (10 μg/kg/dose up to 500 μg/dose)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lael Yonker, M.D., Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05022303
- Sponsor's protocol ID
- 2021P002143
- Status last verified
- Jun 2024
- First submitted
- 9 Aug 2021
- Last updated
- 13 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Yonker LM, Kane AS, Swank Z, Papadakis L, Kenyon V, Han S, Lima R, Guthrie LB, Alvarez-Carcamo B, Lahoud-Rahme M, Balaguru D, Carroll RW, Lok J, El Saleeby C, Walt DR, Fasano A. Viral spike antigen clearance and augmented recovery in children with post-COVID multisystem inflammatory syndrome treated with larazotide. Sci Transl Med. 2025 Jul 30;17(809):eadu4284. doi: 10.1126/scitranslmed.adu4284. Epub 2025 Jul 30.
PubMed 40737433
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a (Proof of Concept), Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AT1001 for the Treatment of COVID-19 Related Multisystem Inflammatory Syndrome in Children (MIS-C)
- ClinicalTrials.gov ID
- NCT05022303
- Acronym / sponsor ID
- 2021P002143
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Decline in MISC cases
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 1 Oct 2021 (actual)
- Primary completion
- 15 May 2024 (actual)
- Study completion
- 15 May 2024 (actual)
- First posted
- 26 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a Phase 2a (Proof of Concept), randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of AT1001 for use in hospitalized pediatric patients diagnosed with MIS-C. Eligible participants (N=20) will be treated with AT1001 or matching placebo orally four times a day (QID) for up to 21 days as an add-on to standard of care.
The study includes three phases:
- Screening/Baseline: The main purpose of this phase is to determine if the participant meets entry criterion after obtaining informed consent and obtain baseline assessments.
- Treatment: Eligible participants will be treated with AT1001 or matching placebo 10 μg/kg/dose QID up to 500 μg/dose (rounded to the nearest 50 μg) for 21 days as an add-on to standard of care for MIS-C.
- Follow-up through 24 weeks: The participant will return for a follow-up visits during weekly clinic visits during Week 1 through Week 3, with monthly telemedicine visits at Week 4, Week 8, Week 16 and Week 20, and clinic visits at Week 12 and Week 24.
Safety monitoring, including physical examination, vitals, and clinical laboratory testing will be performed during the screening phase, periodically during treatment phase and at the follow-up phase. Adverse events and concomitant medications will be recorded during the entire study.
Total duration of the participants' participation in the study is approximately 24 weeks (with 21 days treatment period). Total duration of the study is projected to be 12 months, dependent on enrollment timeline.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Placebo and drug arm (10 μg/kg/dose up to 500 μg/dose)
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C. |
| Larazotide Acetate | Experimental | Drug: Larazotide AcetateAT1001 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. |
Who may qualify
- Ages
- 1 month to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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)
- Subject (or legal authorized representative) capable of understanding and signing an informed consent form and assent form, when appropriate.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Larazotide AcetateAlso: AT1001AT1001 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 |
| PlaceboMatching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C. | Drug | Placebo |
Primary outcome
Evaluate the efficacy and safety of AT1001 versus placebo on mitigating symptoms of MIS-C
Time frame: 24 weeks
• 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.
Determine proportion of participants with improvement in MIS-C related GI symptoms and no progression of disease
Time frame: 24 weeks
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.
11 secondary outcomes
Determine the impact of AT1001 on infectious and inflammatory markers of MIS-C
Time frame: 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.
Time frame: 24 weeks
Determine the impact of AT1001 on length of stay in hospital (days from baseline to readiness to discharge).
Time frame: 24 weeks
Determine the impact of AT1001 on re-presentation to medical care for MISC-related symptoms after discharge.
Time frame: 24 weeks
Determine the impact of AT1001 on need for escalation of care (eg, transfer from hospital ward to ICU; supplemental oxygen; mechanical ventilation).
Time frame: 24 weeks
Determine the impact of AT1001 on change from baseline in additional organ system(s) involvement during acute presentation/hospitalization, as identified by clinical assessment and clinical laboratory tests.
Time frame: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of IgM, IgG, and IgA antibodies against SARS CoV-2.
Time frame: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of inflammatory markers (CRP, d-dimer, ferritin).
Time frame: 24 weeks
Determine the impact of AT1001 on normalization of SARS-CoV-2 Spike, S1 and nucleocapsid.
Time frame: 24 weeks
Determine the impact of AT1001 on change from baseline in levels of zonulin.
Time frame: 24 weeks
Determine the impact of AT1001 on change in mortality (all causes)
Time frame: 24 weeks
Results and safety
The study was stopped early (reason given: Decline in MISC cases). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2024.
- Derived from this study: PubMed 40737433Yonker LM, Kane AS, Swank Z, Papadakis L, Kenyon V, Han S, Lima R, Guthrie LB, Alvarez-Carcamo B, Lahoud-Rahme M, Balaguru D, Carroll RW, Lok J, El Saleeby C, Walt DR, Fasano A. Viral spike antigen clearance and augmented recovery in children with post-COVID multisystem inflammatory syndrome treated with larazotide. Sci Transl Med. 2025 Jul 30;17(809):eadu4284. doi: 10.1126/scitranslmed.adu4284. Epub 2025 Jul 30.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Larazotide Acetate in people with Covid19 and Multisystem Inflammatory Syndrome in Children, run by Massachusetts General Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Decline in MISC cases). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the efficacy and safety of AT1001 versus placebo on mitigating symptoms of MIS-C (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05022303, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2024.