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Investigational oral peptide · no approved US product found
Larazotide
Some lower larazotide doses showed symptom gains. More was not consistently better. The Phase 3 trial stopped, with no posted results on benefit.
- Higher doses did not consistently produce better symptom results.
- The Phase 3 trial stopped because too many more people would be needed to test its effect.
- The current US sources checked did not show an approved product.
Larazotide acetate is a peptide with eight amino acids. Sources also call it AT-1001 or INN-202. CeDLara names the sponsor’s Phase 3 celiac-disease trial. AT1001 can mean a different drug in other trials.
Fabry-disease records use it for migalastat hydrochloride. That drug is unrelated.
Celiac-disease trials tested separate oral schedules three times daily. Amounts were 0.25, 0.5, 1, 2, 4, and 8 mg. The results did not follow a steady dose-response pattern.
Some lower doses showed symptom results. Higher doses did not consistently do so. Permeability endpoints were negative or could not be interpreted.
Phase 3 CeDLara tested 0.25 mg and 0.50 mg three times daily. It stopped after an interim check of the needed sample size. Too many more people would be needed to detect a statistically significant clinical effect.
Its registry posts no benefit results. Current US sources showed no approved larazotide product.
What matters first
- More was not consistently better: The 12-week trial enrolled people with persistent symptoms. Its 0.5 mg arm, taken three times daily, met the main endpoint. The 1 mg and 2 mg arms did not differ from placebo on any endpoint. Earlier trials also found selected symptom results at lower doses. Those results did not consistently extend to higher doses.
- The permeability tests did not show benefit: Both gluten-challenge studies measured LAMA. Neither found a significant difference from placebo. In the 14-day study, gluten challenge did not significantly raise LAMA above the gluten-free control.
- CeDLara stopped after a sample-size check: The sponsor did not name a specific safety event as the cause. The reviewed registry posts no Phase 3 benefit result.
- Phase 3 endpoint descriptions conflict: The registry names the proportion of binary responders as the main endpoint. This was based on lower CeD PRO abdominal-domain scores. The SEC-filed interim release instead names mean change from baseline in that score. Do not silently replace one with the other.
- Counts describe different things: The sponsor first planned 525 people, or 175 per arm. The registry reports actual enrollment of 307. Neither count says how many people completed the trial.
- Keep the MIS-C child study separate: Twelve children were enrolled while in the hospital. They were randomized to added larazotide or matching placebo with standard care. The sources do not give the arm split. They had an acute disease. This small study does not set a celiac or long-COVID schedule. It does not set a schedule for general GI use or prevention.
- Amounts cannot recreate the trial preparation: The sponsor described enteric-coated multiparticulate beads. These released the drug in stages. The sources do not give the capsule count or bead concentration. They do not give the fraction absorbed or a compounding method.
Identity, aliases, and trial preparation
FDA’s substance record names larazotide acetate and UNII FO8S2IW40N. It maps AT-1001 to that substance. FDA says a UNII does not imply regulatory review or approval.
Celiac trial registries also use INN-202. CeDLara is a study name, not an approved product.
A broad ClinicalTrials.gov intervention query returned 29 records. Many were Fabry-disease trials. In those records, AT1001 means migalastat hydrochloride, an unrelated small molecule.
The full search count cannot be called a larazotide pipeline. Each alias needs the substance identity and trial number.
The sponsor’s 2022 annual report describes a peptide with eight amino acids. It was in enteric-coated multiparticulate beads inside a hard gelatin capsule. The mixed beads released the drug in stages.
Part was released when the beads entered the duodenum. The rest was released about 30 minutes later.
This describes a trial preparation. The studies give the amount per use. They do not give a bead concentration or capsule count.
They do not give the fraction absorbed. They do not show how to recreate the formulation.
Celiac-disease evidence
Fourteen-day gluten challenge
The 14-day trial randomized 86 people with diet-controlled celiac disease. They took larazotide acetate 0.25, 1, 4, or 8 mg, or placebo, three times daily. They received either 2.4 g/day gluten or no gluten.
The main endpoint was the urinary lactulose/mannitol ratio, called LAMA.
The gluten challenge did not significantly raise LAMA above the gluten-free control. Among challenged people, larazotide did not differ from placebo on LAMA. Some lower doses seemed to limit worsening stomach and gut symptoms.
The higher dose did not. No serious adverse events were reported. The main measure varied.
The symptom findings were exploratory and did not follow a steady dose-response pattern. They do not set a clinical schedule.
Six-week gluten challenge
The six-week trial randomized 184 people on a gluten-free diet. They received larazotide 1, 4, or 8 mg three times daily, or placebo. They were also given 2.7 g/day gluten.
LAMA did not differ significantly from placebo.
The 1 mg arm limited symptoms on GSRS (p=0.002). Anti-tissue-transglutaminase IgA ratios were lower at all three doses. Adverse-event rates were similar.
These were exploratory results. The 1 mg symptom finding does not establish the same effect at 4 or 8 mg.
Persistent symptoms despite a gluten-free diet
The 12-week paper and NCT01396213 registry describe 342 adults. They had kept a gluten-free diet for at least 12 months. First came a four-week placebo run-in.
Then came 12 randomized weeks of treatment. People took 0.5, 1, or 2 mg three times daily, or placebo. A four-week placebo run-out followed.
The main endpoint was average on-treatment CeD-GSRS score.
The 0.5 mg arm met the main endpoint. The analysis used the modified intention-to-treat group of 340. ANCOVA gave p=0.022; the repeated-measures model gave p=0.005.
The 1 mg and 2 mg arms did not differ from placebo on any endpoint. Reported safety was comparable with placebo.
The findings were mixed. They did not follow a steady dose-response pattern. They do not establish a 0.5–2 mg “range.” They do not support raising the dose if a lower amount fails.
The 0.5 mg result cannot be applied to the higher-dose groups.
Phase 3 CeDLara program stopped
The CeDLara registry describes a randomized Phase 3 trial. It compared 0.25 mg three times daily, 0.50 mg three times daily, and matching placebo. The record is TERMINATED.
It says “Trial terminated by Sponsor.” Actual enrollment was 307. The registry posts no results.
The registry names the proportion of binary responders as the main endpoint. It uses reduced CeD PRO abdominal-domain scores over 12 weeks. The sponsor’s June 2022 SEC-filed interim release instead names mean change from baseline.
That is change in CeD PRO abdominal-domain symptom severity over 12 weeks. The checked sources do not resolve the difference. Both descriptions must stay labeled by source.
A protocol history or final analysis would be needed to resolve it.
The sponsor first planned 525 people. Each of two active arms would have 175. Placebo would also have 175.
An independent statistician checked the needed sample size partway through. This interim reassessment was specified in advance. About the first half of the original target had finished the 12-week double-blind benefit portion.
The sponsor said too many more people would be needed to detect a statistically significant clinical effect. It did not support continuing the trial.
The later annual report says the sponsor stopped further development for celiac disease. This does not establish a safety-driven stop. It does not supply a posted Phase 3 benefit estimate.
Nor does it show failure in every possible condition. The initial plan was 525, and actual enrollment was 307. Neither counts those who completed treatment or follow-up.
Small pediatric MIS-C study
The NCT05022303 registry reports 12 children in the hospital. It is TERMINATED because MIS-C cases declined. CeDLara stopped for a different reason: its sample-size reassessment.
The children were randomized to added larazotide or matching placebo with standard care. The sources do not give the arm split. The active oral schedule was four times daily.
Each dose was 10 micrograms/kg, capped at 500 micrograms per dose. It was rounded to the nearest 50 micrograms.
The Phase 2a paper says treatment lasted three weeks. Safety follow-up lasted 24 weeks. The children had acute MIS-C.
Their median age was 5.7 years. The paper reported no related adverse events. With larazotide, stomach and gut symptoms resolved faster.
Spike-antigen clearance was faster too. Children also returned to usual activities faster.
This study enrolled 12 children with acute disease. It cannot set a routine schedule for children. It cannot establish long-COVID use, general stomach and gut treatment, or prevention.
The registry posts no tabular results. The observed outcomes come from the paper, not the study-status field.
Current US regulatory status
FDA describes Drugs@FDA as its approved-product database. We checked its September 4, 2026 data page and 12-table archive. No match appeared, regardless of letter case, for larazotide, larazotide acetate, CeDLara, or AT-1001.
The exact openFDA ingredient query returned HTTP 404 “No matches found.” The exact DailyMed search returned zero records.
Current US sources showed no approved larazotide product. The FDA substance record establishes identity, not approval. These search results make no claim about another country.
Reader questions
Did higher larazotide doses work better?
There was no consistent dose-response pattern. In the persistent-symptoms trial, 0.5 mg three times daily met the main endpoint. The 1 mg and 2 mg arms did not differ from placebo on any endpoint.
The six-week gluten-challenge trial found a symptom result at 1 mg three times daily. It did not establish that result generally for 4 or 8 mg. Do not pool these findings into one dose range.
Did larazotide improve intestinal permeability?
Not on the LAMA comparisons reviewed here. Neither gluten-challenge trial found a significant difference from placebo. In the 14-day study, gluten challenge did not significantly raise LAMA above the gluten-free control.
This makes the selected symptom findings harder to interpret.
Why did the Phase 3 celiac trial stop?
The sponsor checked the needed sample size during the trial. It said too many more people were needed to detect a statistically significant clinical effect. The registry says the sponsor stopped the trial.
It posts no results. The checked sources do not name a specific safety event as the cause.
How many people completed CeDLara?
The sources do not give that count. The first plan was 525 people. The registry reports actual enrollment of 307.
The sponsor says about the first half of the original target finished the 12-week benefit portion before the reassessment. These statements cannot yield one exact completion count.
What was the CeDLara primary endpoint?
The current registry names the proportion of binary responders. It bases this on reduced CeD PRO abdominal-domain scores. The SEC-filed interim release instead names mean change from baseline in that score.
The difference remains unresolved. Neither should be chosen without a fuller protocol history.
Does the MIS-C study establish pediatric or long-COVID treatment?
No. The trial enrolled 12 children with acute MIS-C while in the hospital. They were randomized to added larazotide or matching placebo with standard care.
The arm split was not given. It stopped because MIS-C cases declined. It does not establish treatment for children more broadly.
It does not establish long-COVID use, general GI treatment, or prevention.
Is larazotide approved in the United States?
Current US checks found no approved larazotide product. These checked the Drugs@FDA archive and exact openFDA ingredient query. They also checked an exact DailyMed search.
An FDA substance or UNII record does not prove product approval.
Can the trial capsule be recreated from these reports?
No. The sponsor described enteric-coated multiparticulate beads that released drug in stages. The sources lack the formulation details needed to recreate it.
The studied amount per use is not a bead concentration. It is not a compounding specification.