Direct Answer
Larazotide is an investigational 8-amino-acid tight-junction-regulating peptide studied in randomized trials for celiac disease.
Summary Table
Evidence Level
Moderate
AI Summary
Larazotide (larazotide acetate, AT-1001) is a synthetic 8-amino-acid peptide that acts as a tight-junction regulator, taken orally to reduce intestinal permeability. Evidence is moderate: it was studied in multiple randomized, placebo-controlled trials in celiac disease, including gluten-challenge studies and a trial in patients with persistent symptoms despite a gluten-free diet. A later phase 3 program did not meet its primary endpoint, and larazotide is not approved by any regulator. Effects are indication-specific and not established for general use.
Key Information
Classification
Key Takeaways
- Larazotide is an investigational tight-junction-regulating peptide studied in celiac disease
- Multiple randomized trials were conducted, but the phase 3 primary endpoint was not met
- It is not approved by any regulator
Scientific Overview
In Plain English
Larazotide is a lab-made peptide taken by mouth that was tested to help people with celiac disease by tightening the junctions between gut cells. It went through several randomized trials, but a late-stage (phase 3) study did not meet its main goal, and it is not an approved medicine.
Scientific Details
Larazotide acetate (AT-1001) is an orally administered 8-amino-acid peptide that regulates intestinal epithelial tight junctions, reducing paracellular permeability. It was evaluated in several randomized, placebo-controlled trials in celiac disease, including gluten-challenge studies and a trial in patients with persistent symptoms despite a gluten-free diet, with some symptom-related signals. A subsequent phase 3 program did not meet its primary endpoint, and larazotide has not achieved regulatory approval.
How It Works
Larazotide is described as a tight-junction regulator associated with reduced intestinal paracellular permeability, intended to limit passage of gluten-derived peptides across the intestinal epithelium. This mechanism is characterized in laboratory studies and evaluated in randomized clinical trials for celiac disease.
Mechanism of Action
Tight-junction regulation
human
Larazotide is associated with reduced intestinal epithelial permeability by acting on tight-junction assembly, studied in the context of celiac disease.
Evidence Level
Human Evidence
Multiple randomized, placebo-controlled trials in celiac disease evaluated larazotide during gluten challenge and for persistent symptoms; a phase 3 program did not meet its primary endpoint.
Animal Evidence
Preclinical work characterized effects on intestinal barrier permeability.
Limitations
Despite randomized trials, the phase 3 primary endpoint was not met, and larazotide is not approved. Evidence is specific to celiac disease and does not establish benefit for other uses.
Why This Grade
Graded moderate: supported by multiple randomized, placebo-controlled trials in a defined indication, but the phase 3 primary endpoint was not met and there is no regulatory approval.
References
- Larazotide acetate for persistent symptoms of celiac disease despite a gluten-free diet: a randomized controlled trial. Gastroenterology.Human Studydoi:10.1053/j.gastro.2015.02.008 →
- A randomized, double-blind study of larazotide acetate to prevent the activation of celiac disease during gluten challenge. American Journal of Gastroenterology.Human Studydoi:10.1038/ajg.2012.211 →
- Larazotide acetate in patients with coeliac disease undergoing a gluten challenge: a randomised placebo-controlled study. Alimentary Pharmacology & Therapeutics.Human Studydoi:10.1111/apt.12147 →
Alternative Names
- Larazotide acetate
- AT-1001
- INN-202
Risks & Safety
- Not an approved medicine; it did not meet its phase 3 primary endpoint
- Reported adverse events in trials were generally mild (including gastrointestinal symptoms and headache), but long-term safety is not established outside trials
- Benefit is specific to celiac-disease research and not established for general use
- Material sold outside clinical trials is not the regulated investigational product and is not quality-controlled
Claim Boundaries
ION BLUE is an educational research aggregator. This content summarizes published scientific literature. It is not medically reviewed, is not medical advice, and is not a recommendation to use any substance. Some peptides discussed are investigational drugs not approved for human use. Consult a licensed healthcare provider. This entry summarizes investigational clinical research and is not medical advice or a recommendation to obtain or use larazotide.
This page summarizes published research and is for informational purposes only; it is not medical advice.