Table 3.

Aims and efficacy results of trial CLIN 1001-006.

Aims	Efficacy results
Primary efficacy outcome:
LAMA ratio	• There was no significant difference between larazotide acetate treated groups and placebo group. • The 1 mg larazotide acetate treatment group had the changes in LAMA ratio. However, this was not statistically significant.
Secondary efficacy outcomes:
Gastrointestinal severity symptoms	According to changes in total GSRS and CeD-GSRS score from baseline: • The 1 mg larazotide acetate treatment group had significantly limited gluten induced symptoms versus placebo. • The mean total GSRS and CeD-GSRS scores increased by 0.3–0.4 unit during gluten challenge in the placebo group. However, the 1 mg treatment group remained close to baseline. • The changes in total GSRS score in the 1 mg treatment group were significantly lower than in the placebo group. The biggest difference (0.5 units) between these two was on the diarrhea subdomain of the GSRS. • The scores in the abdominal pain and indigestion subdomains of GSRS were significantly lower in the 1 mg larazotide acetate treatment group versus the placebo group. • The reduction of total GSRS scores in other treatment groups was not significant versus the placebo group.
Anti-tTG antibodies	• The mean of anti-tTG antibodies levels in the placebo group became 19 U/ml after 6 weeks of gluten challenges. However, the mean levels of antibodies in all treatment groups remained less than 10 U/ml (the cutoff for positive). This was significantly lower than the placebo group.
Quality of life	• The placebo group had lower PGWBI scores compared with treatment groups (the difference was not significant).

CeD-GSRS, Celiac Disease Gastrointestinal Rating Symptoms Scale; GSRS, Gastrointestinal Rating Symptoms Scale; LAMA, lactulose to mannitol; PGWBI, Psychological General Well-Being Index; tTG, tissue transglutaminase.