Safety and Efficacy of Crofelemer in Adult Patients With Short Bowel Syndrome and Intestinal Failure (SBS-IF) Without Colon-in-continuity (CIC)

Phase 2

Not yet recruiting

• Conditions: Short Bowel Syndrome; Malabsorption Syndromes; Short Gut Syndrome; Post-Op Complication; Functional Gastrointestinal Disorders
• Interventions: Drug: Matched Placebo Powder for Oral Solution; Drug: Crofelemer Powder for Oral Solution

• Registration Number: NCT06904872
• Lead Sponsor: Napo Therapeutics, S.p.A.

Brief Summary

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 and randomization will be stratified by baseline PS volume (≤4 or \>4 L/week).

Detailed Description

This is 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).

After an up to 4-week screening period and a PS stabilization period that will last from 2 to 12 weeks, eligible patients who have meet the stabilization requirements will be randomized 1:1:1 to the following treatment groups and entered into the 24-week double-blind treatment period:

* Crofelemer 3 mg/kg/dose TID, morning, midday and evening;

* Crofelemer 10 mg/kg/dose TID, morning, midday and evening;

* Matched placebo TID, morning, midday and evening.

Visits during the 24-week treatment period will be performed at baseline (Day 0) and after 1, 2, 4, 8, 12, 16, 20 and 24 weeks of treatments.

At the end of the 24-week treatment period, patients will be followed up for 4 weeks for safety.

For the primary and secondary objectives, changes between the two crofelemer and placebo arms will be assessed over the 24-week treatment period versus baseline.

Study Timeline

• Study First Submitted: 2025-03-10
• Study First Submitted QC: 2025-03-25
• Status Verified: 2025-04
• Study Start: 2025-04
• Study First Posted: 2025-04-01
• Last Update Submitted: 2025-04-08
• Last Update Posted: 2025-04-11
• Primary Completion: 2026-02
• Study Completion: 2026-02

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Matched Placebo three times per day (TID)	Matched Placebo Powder for Oral Solution	Participants randomized to the matched placebo arm will receive matched placebo by oral route three times per day (TID) morning, midday, and evening for 24 weeks.
Crofelemer 3 mg/kg/dose three times daily (TID)	Crofelemer Powder for Oral Solution	Participants randomized to the Crofelemer 3 mg/kg/dose arm will receive Crofelemer 3 mg/kg/dose by oral route three times per day (TID) morning, midday, and evening for 24 weeks.
Crofelemer 10 mg/kg/dose three times daily (TID)	Crofelemer Powder for Oral Solution	Participants randomized to the Crofelemer 10 mg/kg/dose arm will receive Crofelemer 10 mg/kg/dose by oral route three times per day (TID) morning, midday, and evening for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	24 weeks	Frequency of IP interruption and/or discontinuation considered related to the study drug
Preliminary Efficacy	24 weeks	Change in weekly stool volume from baseline, by measuring and recording daily stool volume in the patient daily diary

Secondary Outcome Measures

Name	Time	Method
Change in parenteral support electrolytes intake	24 weeks	Change in total number of electrolytes administered, as detailed on the PS prescription written by the study doctor
Change in parenteral support volume	24 weeks	Change from baseline in weekly PS volume at different study timepoints, as recorded in the patient daily diary
Change in parenteral support calories intake	24 weeks	Change in total number of calories administered, as detailed on the PS prescription written by the study doctor
Change in weekly oral fluid volume intake	24 weeks	Change from baseline in weekly oral fluid volume intake, as recorded in the patient daily diary
Proportion of patients with change in number of days/week of PS	24 weeks	Proportion of patients with at least one day reduction in weekly PS
Number of days/week of PS	24 weeks	Change in number of days/week of PS requirements from baseline
Change in volume of loose/watery stool	24 weeks	Change from baseline in weekly loose/watery stools as measured by the volume in the ostomy bag or other measuring devices and recorded in the patient daily diary
Changes from baseline in stool consistency	24 weeks	Recording stool consistency of each stool using the 7-point Bristol Stool Scale in the patient daily diary
Changes in laboratory parameters	24 weeks	Changes from baseline of individual lab values within a chemistry and metabolic panel analysis
Changes in physical examination	24 weeks	Changes from baseline in physical examination findings (such as head, ears, eyes, nose, mouth, skin, heart, lung, lymph nodes, gastrointestinal, skeletal, and neurological signs and symptoms)

Trial Locations

Locations (8)

Universitäatsklinik RWTH; 🇩🇪 Aachen, Germany

Charité Universitätsmedizin; 🇩🇪 Berlin, Germany

Universitätsklinikum; 🇩🇪 Essen, Germany

Asklepios Klinik St. Georg; 🇩🇪 Hamburg, Germany

Universitätsmedizin; 🇩🇪 Rostock, Germany

Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi; 🇮🇹 Bologna, Italy

Ospedale Università di Padova; 🇮🇹 Padova, Italy

Azienda Ospedaliera Universitaria Federico II; 🇮🇹 Napoli, Italy