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

June 9, 2025 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 32-week 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

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a randomized, double-blind, placebo-controlled, dose-escalating study with a placebo cross-over 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 within each dose level and over 24 weeks with all 3 dose levels combined will be descriptively summarized. For secondary objectives, changes from the 8-week pre-treatment Baseline Period will be made: 1) within each participant between crofelemer and placebo within each dose level, 2) within each participant between crofelemer and placebo through 24 weeks, 3) between the crofelemer and placebo groups (if multiple participants per group are enrolled) within each dose level, and 4) between the crofelemer and placebo groups (if multiple participants per group are enrolled) through 24 weeks.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2

Contacts and Locations

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

Study Contact

Study Contact Backup

Study Locations

  • Italy
      • Rome, Italy
        • Not yet recruiting
        • UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4
        • Contact:
        • Principal Investigator:
          • Antonella Diamanti, MD
  • United Arab Emirates
      • Dubai, United Arab Emirates
        • Recruiting
        • Al Jalila Children's Hospital
        • Contact:
        • Principal Investigator:
          • Christos Tzivinikos, MD, DCH
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital
        • Principal Investigator:
          • Lissette Jimenez, MD, MPH

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 between the ages of 3 months to 17 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, a 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:

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
  7. previously received an organ transplant
  8. any currently-diagnosed malignancy
  9. is pregnant or breastfeeding
  10. any investigator determined criteria for inability to participate in this study

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 Daily Loose/Watery Stool Volume
Time Frame: Average Weekly for 32 weeks
Measure and record the volume of loose/watery stools each day using a toilet hat for stool collection
Average Weekly for 32 weeks
Average Daily Stool Frequency
Time Frame: Average Weekly for 32 weeks
Record the number of stools/day using the Daily Stool Diary
Average Weekly for 32 weeks
Average Daily Stool Consistency
Time Frame: Average Weekly for 32 weeks
Record stool consistency of each stool using the 7-point pediatric version of the Bristol Stool Form Scale in the Daily Stool Diary
Average Weekly for 32 weeks
Stool Electrolyte
Time Frame: Average Weekly for 32 weeks
Stool electrolyte (Na+, K+, Cl-) concentration measured in mEq/L
Average Weekly for 32 weeks
PS Volume Requirements
Time Frame: Average Weekly for 32 weeks
Record daily weekly TPN and IV fluid volume requirements in the Daily TPN and IV Fluid Diary
Average Weekly for 32 weeks
Average Weekly Supplemental Electrolytes
Time Frame: Average Weekly for 32 Weeks
Record daily any IV supplements of Na+, K+, Cl-, or acetate on the Daily PS Diary
Average Weekly for 32 Weeks

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)

February 1, 2026

Study Completion (Estimated)

March 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 12, 2025

Last Update Submitted That Met QC Criteria

June 9, 2025

Last Verified

June 1, 2025

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.

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