Larazotide Acetate for Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Massachusetts General Hospital, Boston, MA
Syndrome+3 More
Larazotide Acetate - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug called AT1001 might help treat children with COVID-19.

See full description

Eligible Conditions

  • Syndrome
  • Covid19
  • Multisystem Inflammatory Syndrome in Children

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

This trial is evaluating whether Larazotide Acetate will improve 2 primary outcomes and 17 secondary outcomes in patients with Syndrome. Measurement will happen over the course of 24 weeks.

24 weeks
Determine proportion of participants with improvement in MIS-C related GI symptoms and no progression of disease
Determine the impact of AT1001 on (all causes)
Determine the impact of AT1001 on change from baseline in activities limitatio
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.
Determine the impact of AT1001 on change from baseline in levels of IgM, IgG, and IgA antibodies against SARS CoV-2.
Determine the impact of AT1001 on change from baseline in levels of NT proBNP and troponin (participants with cardiac organ system involvement).
Determine the impact of AT1001 on change from baseline in levels of inflammatory markers (CRP, d-dimer, ferritin).
Determine the impact of AT1001 on change from baseline in levels of zonulin.
Determine the impact of AT1001 on change in mortality (all causes)
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.
Determine the impact of AT1001 on infectious and inflammatory markers of MIS-C
Determine the impact of AT1001 on length of stay in hospital
Determine the impact of AT1001 on length of stay in hospital (days from baseline to readiness to discharge).
Determine the impact of AT1001 on need for escalation of care (eg, transfer from hospital ward to ICU; supplemental oxygen; mechanical ventilation).
Determine the impact of AT1001 on normalization of SARS-CoV-2 Spike, S1 and nucleocapsid.
Determine the impact of AT1001 on rate of mortality (all causes)
Determine the impact of AT1001 on rate of normalization of SARS-CoV-2 Spike, S1 and nucleocapsid.
Determine the impact of AT1001 on re-presentation to medical care for MISC-related symptoms after discharge.
Evaluate the efficacy and safety of AT1001 versus placebo on mitigating symptoms of MIS-C

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

Larazotide Acetate
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 20 total participants across 2 different treatment groups

This trial involves 2 different treatments. Larazotide Acetate is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

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.
Placebo
Drug
Matching placebo will be administered orally four times a day (QID) to the standard of care for MIS-C.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Larazotide
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 24 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 24 weeks for reporting.

Who is running the study

Principal Investigator
L. Y. M.
Lael Yonker M.D., Pulmonary Attending Physician
Massachusetts General Hospital

Closest Location

Massachusetts General Hospital - Boston, MA

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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);
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.

Patient Q&A Section

Can syndrome be cured?

"Results from a recent clinical trial indicate that a syndrome of chronic illness with cognitive change and neurological and cardiovascular abnormalities may be a curable syndrome." - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"A patient with multiple signs of disease may have a number of conditions responsible for the signs.\n\nThere may be many causes of pain. It may result from a specific medical condition, such as cancer, from inflammation or inflammation-related conditions like rheumatoid arthritis or gout or from musculoskeletal disorders such as osteoarthritis. A symptom which may be related to a specific condition is not always present.\n\nOften there will be no specific treatment for a condition that causes pain, and the pain will need to be addressed." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"Syndromes may be defined as a group of disorders with common symptoms (e.g. fever, vomiting, and weakness), or can be defined as a constellation of signs and symptoms. Examples include Behçets’ disease, essential tremor, essential thrombocythemia, lymphoma, multiple system atrophy, paroxysmal nocturnal hemoglobinuria, and psoriasis. A syndrome also may be defined as a characteristic biological abnormality that is usually seen in a group of individuals with other similar symptoms that are not due to an identifiable disease, disorder, or syndromes.\n\nThis chapter provides an exhaustive classification of syndrome types according to body system (e.g." - Anonymous Online Contributor

Unverified Answer

How many people get syndrome a year in the United States?

"The incidence of the syndrome is low in this cohort of adults. However a larger study is needed to establish these findings in a different population." - Anonymous Online Contributor

Unverified Answer

What causes syndrome?

"Results from a recent clinical trial on the association between childhood trauma and the symptoms of adulthood somatoform disorders suggest the potential use of psychiatric diagnoses in unraveling the neural bases of this syndrome." - Anonymous Online Contributor

Unverified Answer

What are common treatments for syndrome?

"Many syndromes are treated through pharmacological and/or psychological therapies alone. Some types of syndromes such as the anxiety spectrum, are often treated through CBT. The most common medications for syndromes are antidepressants which are often prescribed as an adjunct to CBT. A large number of medications are used to treat syndromes such as the panic spectrum and anxiety spectrum but are not common for all syndromes. A small number of medications are used as a mainstay for syndromes although are not very common and are mainly used for severe and/or persistent cases of these syndromes. One exception is the use of benzodiazepines for the management of insomnia." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating syndrome?

"Syndromes can be divided into two groups: syndromes caused by mutations in genes and syndromes caused by environmental exposures. While some conditions occur frequently in humans, it would be difficult for treatment strategies to be tailored to specific syndromes. Thus, it is essential for scientists to continue searching for new ways to treat syndromes, hoping to one day find a cure." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for syndrome?

"Syndromes are more relevant than tumors alone as a reason for enrolling in clinical trials. A number of important clinical trials have been designed to select patients with rare conditions for new drugs or investigational agents." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving larazotide acetate?

"Larazotide acetate is used to treat patients with Neuroendocrine tumors and cancer. This suggests a broader clinical use for this drug. Please go to [Medscape(https://emedicine.medscape.com/articles/viewarticle/106638) to review further research." - Anonymous Online Contributor

Unverified Answer

What does larazotide acetate usually treat?

"Larazotide acetate is used to treat adult and childhood-onset intractable central neuropathic pain. It can also reduce chronic dyspnea and, in pediatric ALS, it can affect spasticity and fatigue. Treatment with larazotide acetate can improve symptoms of ALS." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets syndrome?

"A patient with the syndrome can be anywhere in their 30s as a result of a delay in diagnosis. This is not only a cosmetic issue for patients and clinicians but also a huge loss of professional productivity from physicians and other healthcare professionals as they are forced to revisit patients unnecessarily. As physicians become increasingly busy treating other patient needs and have less time to consider new cases of suspected syndrome, this issue of overwork must be kept in check through timely recognition and reporting of syndrome cases." - Anonymous Online Contributor

Unverified Answer

Is larazotide acetate typically used in combination with any other treatments?

"LA used in combination with other treatments did not significantly reduce the TFI in the entire treatment arm over placebo. However, there were significant differences in tumor response between the placebo arm and the LA plus radiotherapy arm." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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