Not recruitingRare disease

Study on the Safety and Effectiveness of Crofelemer for Children with Microvillus Inclusion Disease (MVID)

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare intestinal disease called Microvillus Inclusion Disease (MVID). This condition affects the intestines and can cause severe diarrhea and nutrient absorption issues. The trial will test a new treatment called Crofelemer, which is provided as a powder for oral solution. Crofelemer is being tested in different doses to see how safe and tolerable it is for children with MVID. The study will also compare Crofelemer to a placebo to understand its effectiveness better.

The purpose of the study is to evaluate the safety and tolerability of Crofelemer when given in increasing doses to participants with MVID. Participants will receive the treatment over a period of time, and their health will be monitored closely. The study will involve regular check-ups, including physical exams and laboratory tests, to ensure the safety of the participants. The trial will also look at how the treatment affects stool volume and frequency, as well as other health indicators.

Participants in the study will be given Crofelemer in the form of a powder that is mixed with water and taken orally. The study will use different devices, such as syringes, to help administer the treatment. The trial aims to provide valuable information on how Crofelemer can help manage symptoms of MVID and improve the quality of life for those affected by this condition. The study is expected to continue until the end of 2026, with recruitment starting in late 2024.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the participant or their legal caregiver signs an informed consent form. This indicates understanding of the study's purpose and procedures.
Participants aged 7 years or older, who can understand the study, provide written assent to participate.
Step 2
Initial assessment
An initial assessment is conducted to confirm the diagnosis of Microvillus Inclusion Disease (MVID) through genetic or histologic tests.
Participants must be able to ingest the study medication, Crofelemer Powder for Oral Solution, either orally or through a G-tube or GJ-tube.
Step 3
Medication administration
Participants receive Crofelemer Powder for Oral Solution, which is administered orally.
The study involves three ascending doses of the medication, compared to a placebo, to evaluate safety and tolerability.
Step 4
Treatment period
The treatment period lasts for 24 weeks, during which participants receive the medication at different dose levels.
Every two weeks, stool volume and frequency are monitored through 24-hour stool collections.
Step 5
Monitoring and evaluation
Throughout the study, assessments include physical exams, laboratory evaluations, and monitoring of hydration status and urine output.
Changes in stool consistency and electrolytes are evaluated, along with weekly requirements for parenteral support and fluids.
Step 6
End of treatment
At the end of the treatment period, final assessments are conducted to evaluate the safety and effectiveness of the medication.
Participants who are female and have reached menarche, and male participants, must follow specific guidelines regarding birth control during and after the study.

Who can join the trial?

8 criteria

Participants, or their legal caregiver, must sign an Informed Consent Form (ICF), which means they understand the study and agree to take part.
Children who are old enough to understand the study should also give their written agreement to participate.
Participants must be between 3 months and 17 years old at the time of signing the consent form.
Participants must have a confirmed diagnosis of Microvillous Inclusion Disease (MVID), which is a specific medical condition.
Participants must be able to take the study medication, Crofelemer Powder for Oral Solution, either by mouth or through a feeding tube called a G-tube or GJ-tube (but not a J-tube).
Participants must have needed a certain amount of Parenteral Support (PS) for hydration in the 8 weeks before starting the study. PS is a way to provide nutrition and fluids through a vein.
If female participants have started their menstrual periods, they and their caregiver must agree to use two methods of birth control or remain abstinent during the study and for 30 days after the last dose of the study drug.
Male participants and their caregiver must agree to use contraception or remain abstinent during the study and for 90 days after the last dose of the study drug.

Who cannot join the trial?

8 criteria

Participants who are not diagnosed with Microvillous Inclusion Disease (MVID) cannot participate. MVID is a rare condition affecting the intestines.
Participants who are not receiving PS cannot participate. PS stands for parenteral support, which is a way of providing nutrition through the veins.
Participants who are not within the specified age range cannot participate. The study is for children aged 2 years and older.
Participants who are not able to follow the study procedures or take the study medication as directed cannot participate.
Participants who have any other medical condition that might interfere with the study cannot participate.
Participants who are pregnant or breastfeeding cannot participate.
Participants who are taking certain medications that might interfere with the study cannot participate.
Participants who have a history of allergic reactions to the study medication cannot participate.

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Verified sites

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Verified
Ospedale Pediatrico Bambino Gesu'
Rome, Italy

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Investigated drugs

Crofelemer is being tested in this clinical trial. It is a medication used to help manage diarrhea. In this study, it is given as a powder that is mixed with water to make an oral solution. The trial aims to see how safe and well-tolerated Crofelemer is when given in increasing amounts to children with a rare condition called Microvillus Inclusion Disease (MVID). This condition affects the intestines and causes severe diarrhea. The goal is to find out if Crofelemer can help reduce the symptoms of diarrhea in these patients.

Crofelemer

What is already known about the treatment

Crofelemer – Crofelemer is administered orally in the form of a powder for solution. It is currently being evaluated in clinical trials for its safety, tolerability, and efficacy in treating Microvillous Inclusion Disease (MVID) in pediatric patients. The main therapeutic indication for Crofelemer is to manage symptoms associated with MVID, a rare intestinal disorder. At the molecular level, Crofelemer works by inhibiting chloride channels in the intestines, which helps reduce fluid secretion and diarrhea. It is classified pharmacologically as an anti-diarrheal agent.

Investigated diseases

Microvillous Inclusion Disease – Microvillous Inclusion Disease (MVID) is a rare genetic disorder that affects the intestines, leading to severe, chronic diarrhea. It is characterized by the presence of abnormal microvilli in the cells lining the intestines, which are essential for nutrient absorption. This condition typically presents in infancy, causing significant fluid and electrolyte loss due to the inability to properly absorb nutrients. As the disease progresses, affected individuals may experience malnutrition and require specialized nutritional support. The condition is often identified by persistent diarrhea that does not respond to standard treatments. MVID is a lifelong condition that requires careful management to maintain hydration and nutritional status.