Ongoing Clinical Trials for Microvillous Inclusion Disease
There is currently 1 ongoing clinical trial investigating new treatments for Microvillous Inclusion Disease, a rare genetic disorder affecting the intestines that causes severe chronic diarrhea in infants and children. The trial is testing the safety and effectiveness of Crofelemer, an investigational medication designed to help manage diarrhea symptoms.
Clinical trial locations
Study on the Safety and Effectiveness of Crofelemer for Children with Microvillus Inclusion Disease (MVID)
This clinical trial is investigating Crofelemer as a potential treatment for children with Microvillus Inclusion Disease, a rare condition that affects the intestines and causes severe diarrhea and problems absorbing nutrients. The study is taking place in Italy and is expected to continue until the end of 2026.
Main inclusion criteria:
- Children between 3 months and 17 years old at the time of enrollment
- Confirmed diagnosis of Microvillous Inclusion Disease through genetic or tissue testing
- Ability to take the medication by mouth or through a feeding tube (G-tube or GJ-tube, but not J-tube)
- Requirement for parenteral support (nutrition and fluids given through a vein) for hydration in the 8 weeks before starting the study
- Signed informed consent from parents or legal caregivers, with written agreement from children aged 7 years or older who can understand the study
- Female participants who have started menstruation and male participants must agree to use appropriate birth control methods during the study and for a specified period afterward
Main exclusion criteria:
- Participants without a confirmed diagnosis of Microvillous Inclusion Disease
- Participants not receiving parenteral support
- Participants outside the specified age range
- Inability to follow study procedures or take the medication as directed
- Other medical conditions that might interfere with the study
- Pregnancy or breastfeeding
- Taking certain medications that might interfere with the study
- History of allergic reactions to the study medication
Focus and goals:
The primary goal of this trial is to evaluate the safety and tolerability of Crofelemer when given in increasing doses to children with Microvillous Inclusion Disease. The study involves a 24-week treatment period during which participants receive different dose levels of the medication compared to a placebo. Researchers will monitor how the treatment affects stool volume and frequency through regular 24-hour stool collections every two weeks.
Throughout the study, participants will undergo regular health monitoring including physical exams, laboratory tests, and assessments of hydration status and urine output. The trial will also evaluate changes in stool consistency, electrolyte levels, and the weekly requirements for parenteral support and fluids. This comprehensive approach aims to provide valuable information about how Crofelemer can help manage the symptoms of this condition and potentially improve quality of life for affected children.
Investigational drug:
The medication being tested is Crofelemer, which is provided as a powder that is mixed with water to make an oral solution. Crofelemer works at the molecular level by inhibiting chloride channels in the intestines, which helps reduce fluid secretion and diarrhea. It is classified as an anti-diarrheal agent and is administered orally or through a feeding tube. The study will test three ascending doses of Crofelemer to determine which dose is safest and most effective for children with Microvillous Inclusion Disease.
Summary
Currently, there is only one active clinical trial for Microvillous Inclusion Disease, which is being conducted in Italy. This reflects the rarity of the condition, which typically presents in infancy and requires lifelong specialized care. The trial focuses on Crofelemer, an investigational anti-diarrheal medication that may offer a new treatment option for managing the severe chronic diarrhea associated with this genetic disorder.
The study is particularly noteworthy because it specifically addresses the needs of pediatric patients, from infants as young as 3 months to adolescents up to 17 years old. Given that many children with this condition rely on parenteral support for nutrition and hydration, the potential for an oral medication to reduce diarrhea symptoms could represent a significant advancement in care. The trial’s comprehensive monitoring approach, including regular assessments of stool volume, hydration status, and nutritional support requirements, demonstrates a thorough evaluation of both safety and potential therapeutic benefits.
