AT1001 for the Treatment of Long COVID

NCT05747534 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2023P000240
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 2

Brief Summary

The primary objective of this study is to evaluate the safety and efficacy of Larazotide (AT1001) versus placebo in children and adults 7 to ≤50 years of age who present with symptoms of Long COVID in the presence of SARS-CoV-2 antigenemia. AT1001 (n=100) or placebo (n=50) will be administered orally four times a day (QID) for 21 days.

Conditions

Long COVID; Long COVID-19; Post Acute COVID-19 Syndrome; Post Acute Sequelae of COVID-19

Keywords

SARS-CoV-2; Long COVID; Pediatrics

Outcome Measures

Primary Outcomes (1)

• Adverse Event Profiling and Time to Symptom Resolution

  The primary objective of this study is to evaluate the safety and efficacy of Larazotide (AT1001) versus placebo in children and young adults 7 to ≤50 years of age who present with symptoms of Long COVID. Safety will be assessed by means of adverse event monitoring, and efficacy will be evaluated using the Symptom Burden Questionnaire™ for Long COVID (SBQ™-LC) survey. Additional surveys will be used to monitor symptoms, quality of life and limitation of activities before, during and after treatment with Larazotide or Placebo.

  8 weeks

Secondary Outcomes (1)

• Cytokine profiling, Antigen Testing and Humoral and Cellular Responses

  21 days

Study Arms (2)

Larazotide Acetate (AT1001)

EXPERIMENTAL

Subjects will receive 250 or 500 µg of Larazotide Acetate orally four times a day (QID) for 21 days. Subjects \<25.0 kg will receive 250 µg dose of Larazotide Acetate (AT1001), and subjects ≥25.0 kg will receive 500 µg dose of Larazotide Acetate (AT1001).

Drug: Larazotide Acetate

Placebo

PLACEBO COMPARATOR

Subjects will receive 250 or 500 µg of placebo orally four times a day (QID) for 21 days. Subjects \<25.0 kg will receive 250 µg dose of placebo and subjects ≥25.0 kg will receive 500 µg dose of placebo.

Drug: Placebo

Interventions

Larazotide Acetate DRUG

AT1001 (Larazotide) is a locally acting, non-systemic, octapeptide inhibitor of the zonulin receptor that has shown efficacy in a large variety of animal models of inflammation. The effectiveness of AT1001 in controlling paracellular permeability as a tight junction regulator has been widely demonstrated in animal models both in vitro and in vivo. In MIS-C, prolonged presence of SARS-CoV-2 in the GI tract leads to release of zonulin, a biomarker of intestinal permeability, with subsequent trafficking of SARS-CoV-2 antigens into the bloodstream, leading to hyperinflammation (Yonker, et. al. 2021). Five children treated with AT1001 (through an Emergency Investigational New Drug request authorized by the FDA) displayed a decrease in plasma SARS-CoV-2 Spike antigen levels, inflammatory markers, and symptom improvement superior to that achieved with the current standard of treatment for MIS-C (ie. immunoglobulin, systemic steroids) (Yonker, et. al. 2021) (Yonker, et. al. 2022)

Also known as: AT1001

Larazotide Acetate (AT1001)

Placebo DRUG

Matching placebo will be administered orally four times a day (QID) to participants in the placebo arm.

Placebo

Eligibility Criteria

• Age: 7 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 7 to ≤50 years
• History of SARS-CoV-2 infection, documented by positive PCR and/or antigen test
• Ongoing, worsening, new, or recurrent symptoms present ≥4 weeks after SARS-CoV-2 infection.Symptoms include but are not limited to fatigue, malaise, headache, cognitive impairment, neuropsychiatric symptoms, decreased exercise tolerance, post exertional malaise, dyspnea, cough, chest pain, palpitations, tachycardia, gastrointestinal symptoms, musculoskeletal symptoms, fever, lightheadedness, insomnia and other sleep disturbances, anosmia or dysgeusia, pain, paresthesia, menstrual cycle irregularities, erectile dysfunction.

You may not qualify if:

• Age ≤6 years or \>50 years at time of enrollment
• Pregnancy and/or lactation
• Female participant of childbearing age unwilling to use an acceptable method of birth control for the duration of the study
• Inability to tolerate drug
• Unstable medical conditions or significant co-morbid disease that, by the investigator's determination would make the participant unsuitable for enrollment
• Participation in any other clinical investigation using an experimental drug within 30 days prior to screening
• Intent to participate in another clinical study while participating in this clinical trial
• Blood/plasma donation and 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.
• Abnormal baseline liver function as indicated by AST or ALT ≥3 times the upper limit of normal (ULN), or total bilirubin ≥2x ULN for age
• Abnormal baseline renal function, defined as glomerular filtration rate ≤50 mL/min/1.73m2

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Boston Children's Hospital, Boston, MA, USA: collaborator

Study Sites (2)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Post-Acute COVID-19 Syndrome

Interventions

larazotide acetate

Condition Hierarchy (Ancestors)

COVID-19; Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases; Post-Infectious Disorders; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Lael M Yonker, MD

CONTACT

617-724-2890; lyonker@mgh.harvard.edu

Lauren Guthrie, MPH

CONTACT

6176437175; lauren.guthrie@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind: 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: Drug arm Placebo arm Subjects \<25.0 kg will receive 250 µg of AT1001 or placebo, and subjects ≥25.0 kg will receive 500 µg of AT1001 or placebo

Study Record Dates

• First Submitted: February 24, 2023
• First Posted: February 28, 2023
• Study Start: May 31, 2023
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): March 31, 2027
• Last Updated: July 28, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)