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)
Napo Pharmaceuticals, Inc.
Summary
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
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 c…
Eligibility
- Age range
- 0–17 years
- Sex
- All
- 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 between the ages of 3 months to 17 years at the time of signing the informed consent or providin…
Interventions
- DrugCrofelemer Powder for Oral Solution
Crofelemer Powder for Oral Solution
- DrugPlacebo Powder for Oral Solution
Matching Placebo Powder for Oral Solution
Locations (3)
- Boston Children's HospitalBoston, Massachusetts
- UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4Rome
- Al Jalila Children's HospitalDubai