A Double-blind Placebo-controlled Study to Evaluate Larazotide Acetate for the Treatment of Celiac Disease

Assess the efficacy of larazotide acetate versus placebo for the treatment of celiac disease in adults as an adjunct to a gluten-free diet

Time frame · CeD GSRS was completed weekly Baseline through the end of 12-week double-blind treatment period.

The primary efficacy endpoint was average on-treatment (Baseline to Week 12) score of theCeliac Disease Gastrointestinal Symptom Rating Scale (CeD GSRS).

Assess the safety and tolerability of larazotide acetate in subjects with active celiac disease

Time frame · Up to 12 weeks

Safety endpoints assessed in this study were adverse events, vital signs, physical examination results, clinical laboratory test results, concomitant medication usage and ECG results

Validate a CeD PRO diary instrument in subjects with celiac disease

Time frame · The CeD PRO was administered daily throughout the study.

The CeD PRO is a 12-item questionnaire that was developed to assess symptom severity in clinical trials in subjects with celiac disease. Items in the questionnaire were formulated based on one-on-one interviews with subjects with celiac disease and thus reflect the symptoms that subjects consider part of their celiac disease experience. Subjects rated their symptom severity on an 11-point (0-10) scale. Symptoms included abdominal cramping, abdominal pain, bloating, constipation, diarrhea, gas, loose stools, nausea, vomiting, headache, and tiredness. The questionnaire was designed as a self-administered daily diary, to be completed at the same time each day, and required \< 10 minutes to complete.

Compare various efficacy endpoints during 12 weeks of double-blind treatment

Time frame · GSRS - weekly; BSFS - daily; CGA - screening, baseline, Week 12 and follow-up visits; CeD-QoL and SF12v2 - start of placebo run-in, Week 12 and follow-up visits; CD and CFDCQ - follow-up visit.

Additional efficacy assessments comprised a variety of known scales used for evaluating subjects with celiac disease, including the Gastrointestinal Symptoms Rating Scale (GSRS), Bristol Stool Form Scale (BSFS), Short Form 12 health survey Version 2 (SF12v2), Celiac Disease Quality of Life (CeD-QoL) scale, and the Clinical Global Assessment (CGA). A Gluten-Free Diet Compliance Questionnaire (GFDCQ) and Celiac Dietary Adherence Test (CDAT) were also included to assess compliance with the gluten-free diet.