Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
Other trials for the same condition
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