Table 4.

Aims and efficacy results of trial CLIN 1001-012.

Aims	Efficacy results
Primary endpoint:
Weekly changes at CeD-GSRS score	• Improvement in CeD-GSRS reported in the 0.5 mg larazotide acetate treatment versus placebo (p = 0.005). However, there was no improvement with 1 and 2 mg doses versus placebo.
Secondary endpoints:
Weekly changes from baseline in CeD-GSRS score	• No significant improvement observed for 1 and 2 mg doses versus placebo. • The mean total GSRS decreased in the 0.5 mg larazotide acetate treatment group compared with placebo group (p = 0.004). • The average on GSRS in indigestion, constipation and abdominal pain domains significantly decreased in the 0.5 mg treatment arm versus the placebo group (p = 0.29, 0.05 and 0.29, respectively)
Average weekly on treatment score and changes from baseline in the CeD PRO gastrointestinal and abdominal domain scores	• No significant improvement observed for 1 and 2 mg doses versus placebo. • The 0.5 mg larazotide acetate decreased the CeD PRO abdominal domain score compared with placebo (p = 0.041). • The 0.5 mg larazotide acetate reduced more than 50% of weekly symptoms in the CeD PRO abdominal and GI domain compared with placebo (p = 0.022 and 0.002 respectively) • Abdominal pain and extra-gastrointestinal symptoms significantly decreased with treatment of 0.5 mg larazotide acetate compared with placebo. In addition, symptomatic days decreased in 0.5 mg treatment group versus placebo group (p = 0.017)

GSRS: gastrointestinal rating symptoms scale; CeD-GSRS: celiac disease gastrointestinal rating symptoms scale; PGWBI: Psychological General Well-Being Index; tTG: tissue transglutaminase; CeD PRO: celiac disease patient-reported outcome.