Short Bowel SyndromeEarly Phase 1NCT06326645
Open-Label Pilot Study With Crofelemer in Patients With Short Bowel Syndrome
A Early Phase 1 study testing Crofelemer Oral Product in people with Short Bowel Syndrome, run by Jessica Sosio at 1 site in United States.
Trial 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. . Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- Crofelemer Oral Product
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Mar 2025to 1 Oct 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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
Who cannot join
- 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;
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- 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;
- Use of infliximab, growth hormone or growth factors such as native glucagon-like peptide-2 (GLP-2) or other biological therapy within the last 12 weeks;
- Current or past use of teduglutide within the last 3 months;
- Use of systemic corticosteroids, methotrexate, cyclosporine, tacrolimus, sirolimus, octreotide, intravenous glutamine within the last 30 days;
- If taken at screening, use of antimotility and antidiarrheal agents (loperamide, difenoxylate, codeine and other opiates), H2-receptor antagonists, proton pump inhibitors, bile sequestering agents, oral glutamine, diuretics and oral rehydration solutions is required to be at stable average weekly doses for at least 4 weeks prior to screening evaluations and to remain stable for the entire study duration;
- Use of antibiotics within the last 30 days unless they were administered while ruling out line sepsis or for a dental procedure;
- Alcohol or drug abuse within the last year;
- Pregnant or lactating women;
- History of any malignancy in the past 1 year;
- History of psychiatric illnesses;
- History of any other uncontrolled chronic or acute concomitant disease which, in the Investigator's opinion, would contraindicate study participation or confound interpretation of the results;
- Patient not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements;
- Participation in any other interventional clinical study within 30 days prior to the screening visit.
Trial locations and contacts
1 site in 1 country.
About this study
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. .
Read the detailed description
Short bowel syndrome (SBS) is defined as less than 200 cm in adults of remaining small bowel (i.e. excluding colon) in continuity leading to the need for nutritional and fluid supplements due to impaired absorption of nutrients, electrolytes and fluids.
Most patients with SBS experience debilitating diarrhea that severely hinders their health outcomes and quality of life. SBS-associated diarrhea may have several etiologies including excessive secretion and/or impaired absorption of fluid and electrolytes across the intestinal epithelium. Diarrhea can lead to dehydration, electrolyte imbalance, protein-calorie malnutrition, and loss of critical vitamins and minerals. Consequently, diarrhea in SBS can be severe and life-threatening without proper treatment.
The aggressive use of anti-diarrheal medications in SBS are often clinically required to help manage symptoms. Anti- Secretory agents, including proton pump inhibitors, histamin-2 receptor agonists and somatostatin analogue (Octreotide) as well as various anti-motility agents, including loperamide, Diphenoxylate/atropine, and opioids (Codeine, Tincture of Opium), are often used in higher doses and various formulations with varying effects in patients to help control the diarrhea. Overall, the management of diarrhea is challenging requiring multidisciplinary teams and improved therapies are needed.
Parenteral Nutrition (PN) is a life-saving therapy for patients unable to meet nutritional needs by mouth and a recent study noted that SBS is the most common indication for home PN in the US. However, long-term PN is associated with experience serious metabolic complications, including hepatic and biliary disorders manifested by steatosis, fibrosis and cholestasis. Other complications include central line infections and decrease in quality of life. This has garnered orphan drug designation for intestinotrophic hormones like the glucagon-like peptide-2 (GLP-2) analogue, teduglutide (Gattex), which increases intestinal and portal blood flow, inhibits gastric acid secretion, and decreases intestinal motility, leading up to 20% decrease in PN provisions with treatment in patients with SBS. However, these agents can take weeks to months to take effect and is associated with known risk of developing intestinal growths and cancer that require ongoing surveillance with screening endoscopy. There is a need for other medications to help in the treatment of SBS.
Crofelemer is a novel anti-diarrheal drug that reduces intestinal chloride ion and fluid secretion. It is an FDA approved treatment for HIV-diarrhea (MytesiTM). Its anti-diarrheal properties are due to modulation of chloride ion channel secretion by cystic fibrosis transmembrane conductance regulator (CFTR) and/or Ca2+ activated Cl- channel (CaCC).
Crofelemer has been shown to be a dose-dependent, partial antagonism of CFTR and complete inhibition of CaCC without any changes in the intracellular cyclic Adenosin Monophosphate (cAMP) or calcium levels. Crofelemer also does not have any effects on gut motility or peristalsis. It also has been shown to have minimal absorptive capacity, therefore will not interact with other medications and limit serious adverse events.
It is unknown if Crofelemer can be effective in reducing diarrhea in patients with SBS. There have been case reports that suggest beneficial effects on nutritional status and improvement of diarrhea with tablets of Crofelemer in SBS. This study aims to assess the efficacy of Crofelemer in patients with SBS and ileostomy on Parenteral support.
- Conditions
- Short Bowel Syndrome
- Being tested
- Crofelemer Oral Product
- Keywords
- SBS
- Medical subject headings
- Short Bowel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Crofelemer Oral ProductDrug
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)
| Study group | Role | What they receive |
|---|---|---|
| Crofelemer Group | Experimental | Not described Receives: Drug: Crofelemer Oral Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ostomy output reduction
Change in ostomy output from baseline over the entire 12-week treatment period as average weekly ostomy output at week 12.
Measured: 12 weeks
Secondary outcomes (5)
Change in average weekly Parenteral Support (PS) volume
Measured: 16 weeks from treatment start
Change in SBS-QoL (Short bowel syndrome quality of life)
Patient reported questionnaire with
Measured: 16 weeks from treatment start
Change in GSRS (Gastrointestinal Symptom Rating Scale)
Measured: 16 weeks from treatment start
Change in intestinal absorption of fluids and macronutrients with consequent reduction of macronutrient requirements in patients with SBS
Measured: 12 weeks after start or End of Trial
Change in weekly need for parenteral electrolytes
Measured: 16 weeks from treatment start
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start3 Mar 2025
- Primary completion1 Oct 2026 (estimated)
- Study completion1 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Small exploratory study
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jessica Sosio
- Sponsor type
- University, hospital or other
- Collaborators
- Napo Pharmaceuticals, Inc.
- Responsible party
- Sponsor-investigator, Jessica Sosio, The Cleveland Clinic
- Investigators
- Lindsey Russell, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06326645
- Sponsor's protocol ID
- IRB23-891
- Status last verified
- Aug 2026
- First submitted
- 25 Nov 2023
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Plan to share unidentified data to Napo Pharmaceuticals Inc for future study planning
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Pilot Study With Crofelemer in Patients With Short Bowel Syndrome
- ClinicalTrials.gov ID
- NCT06326645
- Acronym / sponsor ID
- IRB23-891
- Conditions
- Short Bowel Syndrome
- Keywords
- SBS
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Jessica SosioUniversity, hospital or other
- Collaborators
- Napo Pharmaceuticals, Inc.
- Study start
- 3 Mar 2025 (actual)
- Primary completion
- 1 Oct 2026 (estimated)
- Study completion
- 1 Oct 2026 (estimated)
- First posted
- 22 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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. .
Detailed description
Short bowel syndrome (SBS) is defined as less than 200 cm in adults of remaining small bowel (i.e. excluding colon) in continuity leading to the need for nutritional and fluid supplements due to impaired absorption of nutrients, electrolytes and fluids.
Most patients with SBS experience debilitating diarrhea that severely hinders their health outcomes and quality of life. SBS-associated diarrhea may have several etiologies including excessive secretion and/or impaired absorption of fluid and electrolytes across the intestinal epithelium. Diarrhea can lead to dehydration, electrolyte imbalance, protein-calorie malnutrition, and loss of critical vitamins and minerals. Consequently, diarrhea in SBS can be severe and life-threatening without proper treatment.
The aggressive use of anti-diarrheal medications in SBS are often clinically required to help manage symptoms. Anti- Secretory agents, including proton pump inhibitors, histamin-2 receptor agonists and somatostatin analogue (Octreotide) as well as various anti-motility agents, including loperamide, Diphenoxylate/atropine, and opioids (Codeine, Tincture of Opium), are often used in higher doses and various formulations with varying effects in patients to help control the diarrhea. Overall, the management of diarrhea is challenging requiring multidisciplinary teams and improved therapies are needed.
Parenteral Nutrition (PN) is a life-saving therapy for patients unable to meet nutritional needs by mouth and a recent study noted that SBS is the most common indication for home PN in the US. However, long-term PN is associated with experience serious metabolic complications, including hepatic and biliary disorders manifested by steatosis, fibrosis and cholestasis. Other complications include central line infections and decrease in quality of life. This has garnered orphan drug designation for intestinotrophic hormones like the glucagon-like peptide-2 (GLP-2) analogue, teduglutide (Gattex), which increases intestinal and portal blood flow, inhibits gastric acid secretion, and decreases intestinal motility, leading up to 20% decrease in PN provisions with treatment in patients with SBS. However, these agents can take weeks to months to take effect and is associated with known risk of developing intestinal growths and cancer that require ongoing surveillance with screening endoscopy. There is a need for other medications to help in the treatment of SBS.
Crofelemer is a novel anti-diarrheal drug that reduces intestinal chloride ion and fluid secretion. It is an FDA approved treatment for HIV-diarrhea (MytesiTM). Its anti-diarrheal properties are due to modulation of chloride ion channel secretion by cystic fibrosis transmembrane conductance regulator (CFTR) and/or Ca2+ activated Cl- channel (CaCC).
Crofelemer has been shown to be a dose-dependent, partial antagonism of CFTR and complete inhibition of CaCC without any changes in the intracellular cyclic Adenosin Monophosphate (cAMP) or calcium levels. Crofelemer also does not have any effects on gut motility or peristalsis. It also has been shown to have minimal absorptive capacity, therefore will not interact with other medications and limit serious adverse events.
It is unknown if Crofelemer can be effective in reducing diarrhea in patients with SBS. There have been case reports that suggest beneficial effects on nutritional status and improvement of diarrhea with tablets of Crofelemer in SBS. This study aims to assess the efficacy of Crofelemer in patients with SBS and ileostomy on Parenteral support.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Small exploratory study
| Arm | Type | Receives |
|---|---|---|
| Crofelemer Group | Experimental | Drug: Crofelemer Oral Product |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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;
Key exclusion criteria (27 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Crofelemer Oral ProductCrofelemer 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) | Drug | Crofelemer Group |
Primary outcome
Ostomy output reduction
Time frame: 12 weeks
Change in ostomy output from baseline over the entire 12-week treatment period as average weekly ostomy output at week 12.
5 secondary outcomes
Change in average weekly Parenteral Support (PS) volume
Time frame: 16 weeks from treatment start
Change in SBS-QoL (Short bowel syndrome quality of life)
Time frame: 16 weeks from treatment start
Change in GSRS (Gastrointestinal Symptom Rating Scale)
Time frame: 16 weeks from treatment start
Change in intestinal absorption of fluids and macronutrients with consequent reduction of macronutrient requirements in patients with SBS
Time frame: 12 weeks after start or End of Trial
Change in weekly need for parenteral electrolytes
Time frame: 16 weeks from treatment start
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2026.
- Individual participant dataSharing planned: No. Plan to share unidentified data to Napo Pharmaceuticals Inc for future study planning
Related trials for Short Bowel Syndrome
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Registry for Participants With Short Bowel Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lindsey Russell principal investigator
The Cleveland Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Crofelemer Oral Product in people with Short Bowel Syndrome, run by Jessica Sosio at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ostomy output reduction (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2026.