A 24-week, randomized, placebo-controlled, double-blind study to evaluate the efficacy, safety and tolerability of crofelemer in patients with Short Bowel Syndrome and Intestinal Failure (SBS-IF) without colon-in-continuity (CIC) requiring parenteral support (PS).
Blinded study drug will be administered orally (or enterally) three times daily (TID) as a novel crofelemer formulation, Crofelemer Powder for Oral Solution, or a matching placebo powder formulation for oral solution.
Patients will be randomized in a 1:1:1 ratio to crofelemer 3 mg/kg/dose TID, crofelemer 10 mg/kg/dose TID or placebo.
Patients must understand and provide written informed consent before they can participate in the study. They must understand the study procedures and be willing to complete the required assessments;
Male and female patients aged ≥ 18 years;
SBS patients with intestinal failure and without colon-in-continuity who are not eligible or not willing to receive an approved marketed GLP-2;
Patients with history of SBS resulting in intestinal failure caused by a major intestinal resection (e.g., injury, cancer*, Crohn's disease, vascular disease, volvulus) without colon-in-continuity (patients with duodenostomy, Jejunostomy or Ileostomy). Intestinal failure will be defined according to the recommendations of the European Society for Clinical Nutrition and Metabolism (ESPEN), i.e., a reduction of gut function below the minimum necessary for the absorption of macronutrients and/or water and electrolytes, such that intravenous (IV) supplementation is required to maintain health and/or growth. *Patients with history of cancer, should be in remission for the last 6 months and with not ongoing anticancer therapy (long-term hormonal therapy is allowed).
Diagnosis of celiac disease or active or refractory tropical sprue;
Presence of clinically significant intestinal adhesions and/or chronic abdominal pain that can interfere with the conduct of the study;
Patients with current radiological (Radiography and/or CT) evidence of bowel dilatation or pseudo-obstruction;
Active Crohn's disease as evaluated by standard procedures employed by the investigator;
Parallel
Crofelemer Powder for Oral Solution
DrugCrofelemer Powder for Oral Solution
Matched Placebo Powder for Oral Solution
DrugMatched Placebo Powder for Oral Solution
Safety and Tolerability
Frequency of Treatment-Emergent-Adverse Events
Safety and Tolerability
Frequency of IP interruption and/or discontinuation considered related to the study drug
Preliminary Efficacy
Change in weekly volume of parenteral support (PS: parenteral nutrition (PN) with or without intravenous (IV) fluid volume) from baseline, by recording PS volume in the patient daily diary
Preliminary Efficacy
Change in weekly stool volume from baseline, by measuring and recording daily stool volume in the patient daily diary
Change in parenteral support volume
Change from baseline in weekly PS volume at different study timepoints, as recorded in the patient daily diary
Change in parenteral support calories intake
Change in total number of calories administered, as detailed on the PS prescription written by the study doctor
Change in parenteral support electrolytes intake
Change in total number of electrolytes administered, as detailed on the PS prescription written by the study doctor
Change in weekly oral fluid volume intake
Change from baseline in weekly oral fluid volume intake, as recorded in the patient daily diary
Changes in Quality of life (QoL)
Measuring Quality of Life through administration of SBS-QoL questionnaire (17 questions to be responded with a visual analog scale) together with additional daily diary questions
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