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Active, not recruiting Phase 2/3

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

NCT06721871 · tracked via the Priya Life Science USA tracker
Sponsor
Napo Pharmaceuticals, Inc
Phase
Phase 2/3
Started
2025-05-01
Last updated
2026-06-09

Condition(s) studied

Microvillus Inclusion DiseaseCongenital DisordersRare Diseases

Investigational drug(s) / intervention(s)

Crofelemer Powder for Oral Solution →Placebo Powder for Oral Solution

Crofelemer Powder for Oral Solution: Crofelemer Powder for Oral Solution

Placebo Powder for Oral Solution: Matching Placebo Powder for Oral Solution

Study summary

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.

Eligibility

Sex
ALL
Min age
—
Max age
17 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Safety and Tolerability — 32 Weeks
    Incidence of Adverse Events and Serious Adverse Events
  • 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.

Trial sites (3)

FacilityCityRegionStatus
Boston Children's Hospital Boston Massachusetts
UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4 Rome Italy
Al Jalila Children's Hospital Dubai United Arab Emirates
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06721871 on ClinicalTrials.gov ↗ ← All trials in the USA