Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric 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

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.