Open-Label Pilot Study With Crofelemer in Patients With Short Bowel Syndrome

NCT06326645 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: IRB23-891
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The objectives of this study are to evaluate the efficacy and safety of crofelemer treatment in adults affected by Short Bowel Syndrome (SBS) with an ileostomy on parenteral support (PS) in reducing output or PS needs. Crofelemer will be provided as a powder three times daily for 12 weeks and a 4 week follow up. .

Conditions

Short Bowel Syndrome

Keywords

SBS

Outcome Measures

Primary Outcomes (1)

• Ostomy output reduction

  Change in ostomy output from baseline over the entire 12-week treatment period as average weekly ostomy output at week 12.

  12 weeks

Secondary Outcomes (5)

• Change in average weekly Parenteral Support (PS) volume

  16 weeks from treatment start

• Change in SBS-QoL (Short bowel syndrome quality of life)

  16 weeks from treatment start

• Change in GSRS (Gastrointestinal Symptom Rating Scale)

  16 weeks from treatment start

• Change in intestinal absorption of fluids and macronutrients with consequent reduction of macronutrient requirements in patients with SBS

  12 weeks after start or End of Trial

• Change in weekly need for parenteral electrolytes

  16 weeks from treatment start

Study Arms (1)

Crofelemer Group

EXPERIMENTAL

Drug: Crofelemer Oral Product

Interventions

Crofelemer Oral Product DRUG

Crofelemer powder for solution (50g and 100g) or Crofelemer Powder for Oral Solution (100 mg/mL or 30 mg/mL vials for reconstitution and oral dosing)

Crofelemer Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must understand and provide written informed consent before they can participate in the study. They must understand the study procedures of the trial and be willing to complete the required assessments;
• Male and female patients aged ≥ 18 years;
• SBS patients without colon in continuity:
• 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, intussusceptions or other causes). Diagnosis of SBS will be defined as remaining small bowel excluding colon in continuity and considered stable with regard to parenteral support (PS) need. Intestinal failure will be defined according to the recommendations of the American Society of Parenteral and Enteral Nutrition (ASPEN), 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;
• At least 6 months elapsed since last surgical bowel resection;
• No restorative surgery planned in the overall study period;
• Patients with at least 6 continuous months of PS dependency (parenteral nutrition and/or intravenous fluids) before entry in the study;
• Patients receiving stable PS (fluids, electrolytes or nutrients) at least three times per week to meet caloric, fluid or electrolytes needs with no major changes in provisions for at least 12 weeks;
• Patients with Crohn's disease will have to be in clinical remission for ≥ 12 weeks before entry in the study;
• Patients must be able to ingest solid or semi-solid foods and drink fluids;
• If female and of child-bearing potential, the patient use a highly effective method of birth control for the entire study duration. Highly effective birth control methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomized partner; sexual abstinence;
• If female and of child-bearing potential, the patient must have a negative urine pregnancy test prior the first administration of the study drugs;
• Male patients must agree to use an acceptable form of birth control during the study and for 30 days after the last dose;
• Satisfactory general health status as determined by the investigator based on medical history and physical examination

You may not qualify if:

• Body mass index (BMI) \<17.5 or \>30 kg/m2;
• Presence of clinically significant intestinal adhesions and/or chronic abdominal pain that can interfere with the conduct of the study;
• Presence of confirmed active infection, temperatures \>100°Farenheit, or symptoms of an upper respiratory infection
• Patients with Radiological (Radiography and/or CT) signs of significant bowel dilatation or pseudo-obstruction;
• Active Crohn's disease as evaluated by standard procedures employed by the investigator;
• Inflammatory bowel disease (IBD) that required immunosuppressant therapy that has been introduced or changed within last 3 months or treatment with biologics within the last 6 months;
• Intestinal or other major surgery scheduled within the time frame of the study;
• Visible blood in the stool within the last 3 months;
• Ongoing radiation enteritis or the presence of damaged enteral tissue due to radiation enteritis, scleroderma, celiac disease, refractory or tropical sprue;
• Compromised immune system (e.g., acquired immune deficiency syndrome \[AIDS\], severe combined immunodeficiency);
• Inadequate hepatic function: alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>2 upper limit of normal (ULN), total bilirubin \>1.25 ULN or alkaline phosphatases \>2.5 ULN;
• Inadequate renal function: serum creatinine or blood urea nitrogen \>1.5 ULN;
• Urine sodium \<20 mmol/day;
• More than four SBS-related hospital admissions (unless one or more admissions were to rule out line sepsis) within the past 12 months or hospital admission within 1 month before screening;
• Previous use of Crofelemer or potential allergies to Crofelemer or its constituents;

Sponsors & Collaborators

• Lindsey Russell, MD: lead
• Napo Pharmaceuticals, Inc.: collaborator

Study Sites (1)

Cleveland Clinic Main Campus

Cleveland, Ohio, 44106, United States

RECRUITING

MeSH Terms

Conditions

Short Bowel Syndrome

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Lindsey Russell

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lindsey Reardon, AS, BA, MS

CONTACT

(216) 444-7493; reardol3@ccf.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Staff Gastroenterologist

Model Details: Small exploratory study

Study Record Dates

• First Submitted: November 25, 2023
• First Posted: March 22, 2024
• Study Start: March 3, 2025
• Primary Completion: February 1, 2026
• Study Completion: February 1, 2026
• Last Updated: February 12, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)