DiarrheaPhase 4NCT04486326
Crofelemer for Functional Diarrhea
A Phase 4 study testing Crofelemer in people with Diarrhea, run by Beth Israel Deaconess Medical Center at 1 site in United States.
Trial summary
The primary objectives of this study are to evaluate the clinical response of patients with diarrhea to crofelemer relative to placebo and evaluate the overall safety and tolerability of crofelemer in the treatment of diarrhea. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Diarrhea
- What is being tested?
- Crofelemer
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Aug 2020to 23 Sep 2022
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient is a man or woman aged 18 to 65 years, inclusive, at Screening.
- Patient has functional diarrhea defined by the Rome IV criteria as loose (mushy) or watery stools ≥25% and hard or lumpy stools <25% of bowel movements
- Patient has had a colonoscopy performed:
- Within 10 years prior to Prescreening if patient is at least 50 years of age (alternatively, a flexible sigmoidoscopy, double contrast barium enema, or CT colonography within the past 5 years is acceptable [see recommendations of the American Cancer Society])
- Since the onset (if applicable) of any of the following alarm features for patients of any age (see Spiller and Thompson, 2010 - i.e. Patient has documented weight loss within the past 6 months; Patient has nocturnal symptoms; Patient has a familial history of colon cancer; or patient has blood mixed with their stool (excluding any blood from hemorrhoids).
- Patient has an average daily stool consistency score (BSS) of ≥5.5 on days without the use of an anti-diarrheal and at least 3 days with a BSS score ≥5 on a 1 to 7 scale over the week prior to randomization.
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- Patient has completed the daily diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
- Patient has not used loperamide rescue medication more than 4 days during the screening period.
- Patient is not planning to change his/her usual diet and lifestyle during the course of the study.
- Patient is willing to be compliant with study procedures including completing the daily diary during the screening period and throughout the study.
- Patient must sign an informed consent document before the initiation of any study-related procedures indicating that he or she understands the purpose of and procedures required for the study and is willing to participate in the study.
Who cannot join
- Patient has a history of inflammatory or immune-mediated GI disorders including inflammatory bowel disease (ie, Crohn's disease, ulcerative colitis, microscopic colitis) and celiac disease.
- Patient has a predominant symptom of abdominal pain.
- Patient has a history of diverticulitis within 3 months prior to screening. Patients with a history of diverticulosis are candidates for the study.
- Patient has a history of intestinal obstruction, stricture, toxic megacolon, GI perforation, fecal impaction, gastric banding, bariatric surgery, adhesions, ischemic colitis, or impaired intestinal circulation (eg, aortoiliac disease).
- Patient has any of the following surgical history:
- Any abdominal surgery within the 3 months prior to screening; or
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- Patient has a history of major gastric, hepatic, pancreatic, or intestinal surgery (appendectomy, cholecystectomy, hemorrhoidectomy, or polypectomy greater than 3 months are allowed). For the purposes of this study, laparoscopic surgeries without complication are considered minor and non-exclusionary, provided the condition for which the surgery was performed was not exclusionary.
- Patient has an unstable renal, hepatic, metabolic, or hematologic condition.
- Patient has a history of human immunodeficiency virus infection.
- Patient has a history of DSM(Diagnostic and Statistical Manual)-IV-TR-defined substance dependency, excluding nicotine and caffeine, within 2 years prior to Prescreening.
- Patient has a history of alcohol abuse as defined by DSM-IV-TR, binge drinking as defined by the National Institutes on Alcohol Abuse and Alcoholism, or any medical treatment for alcohol-related co-morbidities, within 5 years prior to Prescreening. Recovered alcoholics who have not consumed alcohol over the 5 years prior to Prescreening are candidates for the study.
- Patient has current (within 14 days of randomization) or expected use of any narcotic or opioid containing agents, tramadol
- Patient is unable to swallow solid, oral dosage forms whole with the aid of liquid (patients may not chew, divide, dissolve, or crush the study drug).
- Patient has received an investigational drug or used an investigational medical device within 30 days prior to randomization.
- Patient has a known pregnancy or is breastfeeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
The primary objectives of this study are to evaluate the clinical response of patients with diarrhea to crofelemer relative to placebo and evaluate the overall safety and tolerability of crofelemer in the treatment of diarrhea.
Read the detailed description
Crofelemer is a potent antisecretory agent and has promising therapeutic properties for functional diarrhea patients. Crofelemer is an active compound extracted from the latex of the Western South American plant Croton lechleri. The latex has been used for medicinal purposes for centuries by indigenous peoples, with its active properties attributable to the crofelemer compound (11). Crofelemer has an inhibitory effect on cyclic AMP-mediated secretion of chloride ions in T84 and Caco-2 epithelial cells, resulting in antisecretory effects in the colon that may alleviate diarrhea and related symptoms (12). Crofelemer has been shown to improve traveler's diarrhea and HIV-associated diarrhea (13,14). It's applicability to symptoms of functional diarrhea is still unclear.
In a Phase II randomized trial, Mangel \& Chaturvedi observed the effects of crofelemer in the setting of Inflammatory Bowel Syndrome-D (15). While they did not meet the primary endpoint of altering stool consistency in IBS-D patients, they did find upon further analysis that crofelemer significantly reduced the number of pain- and discomfort-free days in female patients with IBS-D. This study warrants further investigation of the effects of crofelemer in treating symptoms of functional diarrhea.
- Conditions
- Diarrhea
- Being tested
- Crofelemer, Placebo
- Medical subject headings
- Diarrhea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrofelemerDrug
Also called mytesi
Crofelemer
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| crofelemer | Experimental | 125mg bid Receives: Drug: Crofelemer |
| placebo | Placebo comparator | bid Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Consistency of Stool at Week 4
Average stool consistency score on the scale from 1 to 7 (from Bristol stool form scale (BSFS)). This rates the stool consistency by type from 1-7. BSFS 1 indicates hard stool consistent with constipation, whereas BSFS of 7 indicates loose, watery stool consistent with diarrhea. Therefore, in this study, a higher score indicates more diarrhea/worse outcome
Measured: week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2020
- Study start20 Aug 2020
- Primary completion23 Sep 2022
- Study completion23 Sep 2022
Why it stopped: low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for DiarrheaStudies of the same condition, open and under way.
- More trials run by Beth Israel Deaconess Medical CenterThe sponsor's other registered studies.
- 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
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beth Israel Deaconess Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Judy Nee, Beth Israel Deaconess Medical Center
- Investigators
- Anthony Lembo, MD, Principal investigator, Beth Israel Deaconess Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04486326
- Sponsor's protocol ID
- 2020P-000464
- Status last verified
- Mar 2026
- First submitted
- 20 Jul 2020
- Last updated
- 10 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Crofelemer for Functional Diarrhea
- ClinicalTrials.gov ID
- NCT04486326
- Acronym / sponsor ID
- 2020P-000464
- Conditions
- Diarrhea
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low enrollment
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Beth Israel Deaconess Medical CenterUniversity, hospital or other
- Study start
- 20 Aug 2020 (actual)
- Primary completion
- 23 Sep 2022 (actual)
- Study completion
- 23 Sep 2022 (actual)
- First posted
- 24 Jul 2020 (actual)
- Results
- Posted 10 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objectives of this study are to evaluate the clinical response of patients with diarrhea to crofelemer relative to placebo and evaluate the overall safety and tolerability of crofelemer in the treatment of diarrhea.
Detailed description
Crofelemer is a potent antisecretory agent and has promising therapeutic properties for functional diarrhea patients. Crofelemer is an active compound extracted from the latex of the Western South American plant Croton lechleri. The latex has been used for medicinal purposes for centuries by indigenous peoples, with its active properties attributable to the crofelemer compound (11). Crofelemer has an inhibitory effect on cyclic AMP-mediated secretion of chloride ions in T84 and Caco-2 epithelial cells, resulting in antisecretory effects in the colon that may alleviate diarrhea and related symptoms (12). Crofelemer has been shown to improve traveler's diarrhea and HIV-associated diarrhea (13,14). It's applicability to symptoms of functional diarrhea is still unclear.
In a Phase II randomized trial, Mangel \& Chaturvedi observed the effects of crofelemer in the setting of Inflammatory Bowel Syndrome-D (15). While they did not meet the primary endpoint of altering stool consistency in IBS-D patients, they did find upon further analysis that crofelemer significantly reduced the number of pain- and discomfort-free days in female patients with IBS-D. This study warrants further investigation of the effects of crofelemer in treating symptoms of functional diarrhea.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| crofelemer | Experimental | Drug: Crofelemer125mg bid |
| placebo | Placebo comparator | Drug: Placebobid |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patient is a man or woman aged 18 to 65 years, inclusive, at Screening.
- Patient has functional diarrhea defined by the Rome IV criteria as loose (mushy) or watery stools ≥25% and hard or lumpy stools <25% of bowel movements
- Patient has had a colonoscopy performed:
- Patient has an average daily stool consistency score (BSS) of ≥5.5 on days without the use of an anti-diarrheal and at least 3 days with a BSS score ≥5 on a 1 to 7 scale over the week prior to randomization.
- Patient has completed the daily diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
Key exclusion criteria (15 listed)
- Patient has a history of inflammatory or immune-mediated GI disorders including inflammatory bowel disease (ie, Crohn's disease, ulcerative colitis, microscopic colitis) and celiac disease.
- Patient has a predominant symptom of abdominal pain.
- Patient has a history of diverticulitis within 3 months prior to screening. Patients with a history of diverticulosis are candidates for the study.
- Patient has a history of intestinal obstruction, stricture, toxic megacolon, GI perforation, fecal impaction, gastric banding, bariatric surgery, adhesions, ischemic colitis, or impaired intestinal circulation (eg, aortoiliac disease).
- Patient has any of the following surgical history:
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 |
|---|---|---|
| CrofelemerAlso: mytesiCrofelemer | Drug | crofelemer |
| Placeboplacebo | Drug | placebo |
Primary outcome
Consistency of Stool at Week 4
Time frame: week 4
Average stool consistency score on the scale from 1 to 7 (from Bristol stool form scale (BSFS)). This rates the stool consistency by type from 1-7. BSFS 1 indicates hard stool consistent with constipation, whereas BSFS of 7 indicates loose, watery stool consistent with diarrhea. Therefore, in this study, a higher score indicates more diarrhea/worse outcome
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Anthony Lembo, MD principal investigator
Beth Israel Deaconess Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Crofelemer in people with Diarrhea, run by Beth Israel Deaconess Medical Center 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 11 inclusion and 15 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: stopped early, as of the sponsor's last update on 10 Mar 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. Some participants in this study may receive a placebo. 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?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Consistency of Stool at Week 4 (time frame: week 4). 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 10 Mar 2026.