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Crofelemer for

Phase 2

Microvillus Inclusion Disease | Small molecule | Rare Disease |Jaguar Health, Inc.|Last Updated: Jun 9, 2026

Target and mechanism

ModalitySmall molecule

Also known as Crofelemer Powder for Oral Solution

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment6

FDA Designations

No designations recorded

Clinical trial landscape

Crofelemer for · 2 trials · 8 indications

Phase 2 2
NCT06904872Safety and Efficacy of Crofelemer in Adult Patients With Short Bowel Syndrome and Intestinal Failure (SBS-IF) Without Colon-in-continuity (CIC)Short Bowel Syndrome
RECRUITING18 Analytics
NCT06721871Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)Microvillus Inclusion Disease
ACTIVE NOT_RECRUITING6 Analytics
PHASE2RECRUITING
Safety and Efficacy of Crofelemer in Adult Patients With Short Bowel Syndrome and Intestinal Failure (SBS-IF) Without Colon-in-continuity (CIC)
Short Bowel SyndromeUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Microvillus Inclusion DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety and Tolerability
24 weeks

Frequency of Treatment-Emergent-Adverse Events

Preliminary Efficacy
24 weeks

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

Changes in Physical Examination
32 Weeks

Changes from baseline in physical exam and signs of dehydration, such as decreased urine output, sunken eyes, lethargy and abnormal skin turgor.

Changes in Laboratory Values
32 Weeks

Incidence in changes from baseline of individual lab values within a chemistry, hematology and metabolic panel analysis.

Secondary Endpoints

Change in parenteral support volume
24 weeks
Change in parenteral support calories intake
24 weeks
Change in parenteral support electrolytes intake
24 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Crofelemer 3 mg/kg/dose three times daily (TID)EXPERIMENTALParticipants 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)EXPERIMENTALParticipants 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.
Matched Placebo three times per day (TID)PLACEBO_COMPARATORParticipants 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.
Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/dayEXPERIMENTALParticipants may be randomized to crofelemer powder for oral solution during Treatment Period 1 (1 month duration)
Dose Level 1/Treatment Period 1: Placebo 3x/dayPLACEBO_COMPARATORParticipants may be randomized to the placebo comparator during Treatment Period 1 (1 month duration)
Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/dayEXPERIMENTALParticipants will crossover to either crofelemer powder for oral solution or placebo comparator
Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/dayEXPERIMENTALParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration)
Dose Level 2/Treatment Period 1: Placebo 3x/dayPLACEBO_COMPARATORParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration)
Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/dayEXPERIMENTALParticipants will crossover to either crofelemer powder for oral solution or placebo comparator
Dose Level 2/Treatment Period 2: Placebo 3x/dayPLACEBO_COMPARATORParticipants will crossover to either crofelemer powder for oral solution or placebo comparator
Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/dayEXPERIMENTALParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 2 (1 month duration)
Dose Level 3/Treatment Period 1: Placebo 3x/dayPLACEBO_COMPARATORParticipants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solutionor the placebo comparator that they were previously on during Dose Level 2 (1 month duration)
Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/dayEXPERIMENTALParticipants will crossover to either crofelemer powder for oral solution or placebo comparator
Dose Level 3/Treatment Period 2: Placebo 3x/dayPLACEBO_COMPARATORParticipants will crossover to either crofelemer powder for oral solution or placebo comparator
Dose Level 1/Treatment Period 2: Placebo 3x/dayPLACEBO_COMPARATORParticipants will crossover to either crofelemer powder for oral solution or placebo comparator

Interventions

NameTypeDescription
Crofelemer Powder for Oral SolutionDRUGCrofelemer Powder for Oral Solution
Matched Placebo Powder for Oral SolutionDRUGMatched Placebo Powder for Oral Solution
Placebo Powder for Oral SolutionDRUGMatching Placebo Powder for Oral Solution
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria Patients will be enrolled in the study if they meet all the following criteria: 1. 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 assessment...

Countries:GermanyItalyUnited StatesUnited Arab Emirates
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Recent Changes (Last 90 Days)

MEDIUMAug 1, 2026NCT06904872primaryCompletionDate: changed
HIGHAug 1, 2026NCT06721871Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 1, 2026NCT06904872primaryCompletionDate: changed
HIGHAug 1, 2026NCT06721871Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 1, 2026NCT06904872primaryCompletionDate: changed
HIGHAug 1, 2026NCT06721871Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 26, 2026NCT06721871primaryCompletionDate: changed
LOWMay 24, 2026NCT06904872studyFirstPostDate: changed
LOWMay 24, 2026NCT06721871studyFirstPostDate: changed

Frequently asked questions about Crofelemer for

What is Crofelemer used for?

Crofelemer is an investigational small molecule being studied for cancer therapy-related diarrhea, breast cancer, short bowel syndrome, and microvillus inclusion disease. It is being developed by Jaguar Health, Inc. (JAGX) and is currently in Phase 2 clinical development for these indications.

How does Crofelemer work?

Crofelemer is a small molecule that targets the gastrointestinal tract to reduce diarrhea. It is being studied for its ability to prevent and treat diarrhea associated with cancer therapies, as well as diarrhea related to short bowel syndrome and microvillus inclusion disease.

Who makes Crofelemer?

Crofelemer is developed by Jaguar Health, Inc., a biopharmaceutical company traded under the ticker JAGX. The company is conducting clinical trials to evaluate the drug's safety and efficacy across multiple gastrointestinal and oncology-related conditions.

What phase is Crofelemer in?

Crofelemer is in Phase 2 clinical development. It is not FDA approved and remains investigational. The drug is being studied in multiple trials, including a Phase 2 trial for breast cancer and a Phase 2 trial for microvillus inclusion disease, with additional trials in short bowel syndrome.

What clinical trials is Crofelemer in?

Crofelemer has been studied in several clinical trials, including NCT02910219 for HER2 positive breast cancer patients, NCT04538625 for cancer therapy-related diarrhea, NCT06721871 for pediatric microvillus inclusion disease, and NCT06904872 for short bowel syndrome. These trials are at various stages of completion.

Is Crofelemer the same as Crofelemer Powder for Oral Solution?

Yes, Crofelemer is also known as Crofelemer Powder for Oral Solution. This formulation is being studied in clinical trials, including a Phase 2 trial for pediatric microvillus inclusion disease, to evaluate its safety and efficacy in treating diarrhea-related conditions.