Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

June 5, 2026 updated by: Napo Pharmaceuticals, Inc.

Evaluation of Safety, Tolerability and Efficacy of Crofelemer Following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants With Microvillus Inclusion Disease (MVID)

A study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each dose level, when administered to participants with MVID receiving parenteral support (PS, defined as TPN with or without supplementary IV fluid requirements). Blinded study drug will be administered as a novel crofelemer formulation, Crofelemer Powder for Oral Solution, or a matching placebo powder formulation for oral solution. Assigned study drug will be reconstituted and administered orally (or enterally) three times daily (TID) as a concentrated liquid formulation in each of the three dose levels.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The study consists of an initial randomized double-blind placebo-controlled study, followed by an up to 48-week partially blinded extension phase.

The double-blind study phase is a randomized, double-blind, placebo-controlled, dose-escalating study with a placebo crossover design within each dose level in this ultra-rare MVID participant population. For the primary objective, safety and tolerability, comparisons between the crofelemer and placebo, across treatment periods within each dose level and through the end of the double-blind treatment period will be descriptively summarized. For secondary objectives, changes from the Baseline Period will be assessed.

After completion of the study and the No Treatment Period, if the Investigator and the DMC consider it appropriate for the participant's best interest based on safety and tolerability, the participant will be eligible to enter a Partially Blinded Extension Phase. Following approval by the DMC, the participant will enter the extension phase for up to 48 additional weeks of treatment with crofelemer at the dose selected by the DMC. The Investigator and the Sponsor will remain blinded to the selected dose.

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Rome, Italy
        • UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4
  • United Arab Emirates
      • Dubai, United Arab Emirates
        • Al Jalila Children's Hospital
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Participants (assent for participants older than 7 years of age) and/or their legal parent/guardian sign an Informed Consent Form (ICF) indicating that they understand the purpose of the procedures required for the study and are willing to participate
  2. When appropriate, pediatric participants, whose age, cognitive skills, reading abilities and maturity allow the understanding of the study protocol should provide written assent to participate.
  3. Male or female participants from birth to < 18 years at the time of signing the informed consent or providing assent
  4. Have a confirmed diagnosis (genetic and/or histologic) of MVID
  5. Are able to ingest reconstituted Crofelemer Powder for Oral Solution either orally (PO) or through a previously-placed G-tube or GJ-Tube (not via J-Tube)
  6. Have, during the 8 weeks prior to baseline, an average weekly volume of PS that represents at least 50% (≥ 50%) of the participant's weekly hydration volume requirements
  7. If female participants have reached menarche, the participant (and caregiver) agree that the participant will remain abstinent or use two accepted methods of birth control during the course of the treatment period and for an additional 30 days following the last dose of study drug.
  8. Male participants (and caregiver) agree that the participant will remain abstinent or use contraception during the course of the treatment period and continue on for an additional 90 days following the last dose of study drug.

Exclusion Criteria:

  1. Within the last 4 weeks before study initiation, participants have:

    1. had significant changes to PS requirements (i.e., ± > 20%)
    2. had a new requirement for diuretics
    3. had any infection requiring IV antibiotic administration
    4. had a documented active gastrointestinal infection
    5. initiated any new anti-diarrheal drug
    6. had an increase in ALT, AST, or total bilirubin that is ≥2 times the participant's usual laboratory values
  2. previously received an organ transplant
  3. any currently-diagnosed malignancy
  4. is pregnant or breastfeeding
  5. any investigator determined criteria for inability to participate in this study
  6. Known hypersensitivity to any of the components of study drug

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/day
Participants may be randomized to crofelemer powder for oral solution during Treatment Period 1 (1 month duration)
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Placebo Comparator: Dose Level 1/Treatment Period 1: Placebo 3x/day
Participants may be randomized to the placebo comparator during Treatment Period 1 (1 month duration)
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Experimental: Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Experimental: Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/day
Participants 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)
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Placebo Comparator: Dose Level 2/Treatment Period 1: Placebo 3x/day
Participants 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)
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Experimental: Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Placebo Comparator: Dose Level 2/Treatment Period 2: Placebo 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Experimental: Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/day
Participants 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)
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Placebo Comparator: Dose Level 3/Treatment Period 1: Placebo 3x/day
Participants 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)
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Experimental: Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Crofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
Placebo Comparator: Dose Level 3/Treatment Period 2: Placebo 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Placebo Comparator: Dose Level 1/Treatment Period 2: Placebo 3x/day
Participants will crossover to either crofelemer powder for oral solution or placebo comparator
Drug: Placebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and Tolerability
Time Frame: 32 Weeks
Incidence of Adverse Events and Serious Adverse Events
32 Weeks
Changes in Physical Examination
Time Frame: 32 Weeks
Changes from baseline in physical exam and signs of dehydration, such as decreased urine output, sunken eyes, lethargy and abnormal skin turgor.
32 Weeks
Changes in Laboratory Values
Time Frame: 32 Weeks
Incidence in changes from baseline of individual lab values within a chemistry, hematology and metabolic panel analysis.
32 Weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Average PS Volume Requirements Normalized to Body Weight
Time Frame: Average Weekly for 32 weeks
Record daily weekly TPN and IV fluid volume requirements in the Daily TPN and IV Fluid Diary, and divide the value per body weight for the corresponding study visit (mL/kg)
Average Weekly for 32 weeks
Average Loose/Watery Stool Volume
Time Frame: Average every 2 weeks for 32 weeks
Measure and record the volume of loose/watery stools using a toilet hat for stool collection during 24 hours before each study visit
Average every 2 weeks for 32 weeks
Average TPN (including lipids) Volume Requirements
Time Frame: Average weekly for 32 weeks
Record daily weekly TPN volume requirements in the Daily TPN and IV Fluid Diary
Average weekly for 32 weeks
Average Supplemental IV Fluids Volume Requirements
Time Frame: Average weekly for 32 weeks
Record daily weekly supplemental IV fluid volume requirements in the Daily TPN and IV Fluid Diary
Average weekly for 32 weeks
Average Supplemental Electrolytes
Time Frame: Average weekly for 32 weeks
Record daily any supplements of Na+, K+, Cl- in PS and, separately in TPN and in IV fluids, in the Daily PS Diary
Average weekly for 32 weeks
Average Acetate or Lactate Supplementation
Time Frame: Average weekly for 32 weeks
Record daily any supplements of acetate or lactate in PS and, separately in TPN and in IV fluids, in the Daily PS Diary
Average weekly for 32 weeks
Stool Electrolytes
Time Frame: Measurement at baseline, week 20 and week 24
Stool electrolytes (Na+, K+, Cl-) concentration measured in mEq/L
Measurement at baseline, week 20 and week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Napo Pharmaceuticals, Inc.

Investigators

  • Principal Investigator: Lissette Jimenez, MD, MPH, Boston Children's Hospital
  • Study Chair: Pravin Chaturvedi, PhD, Napo Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 1, 2025

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2026

Study Registration Dates

First Submitted

November 20, 2024

First Submitted That Met QC Criteria

December 3, 2024

First Posted (Actual)

December 6, 2024

Study Record Updates

Last Update Posted (Actual)

June 9, 2026

Last Update Submitted That Met QC Criteria

June 5, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NP303-104

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Rare Diseases

Clinical Trials on Crofelemer Powder for Oral Solution

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ghana

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe