NCT06721871

Who might be able to join this trial:

• The participant (and/or their parent or guardian) must be willing to sign a consent form agreeing to take part in the study; children older than 7 years old may also need to sign their own agreement form.
• Children who are old enough and able to understand the study should also give their written agreement to participate.
• Participants must be male or female, from newborn up to (but not including) 18 years old at the time of signing the consent forms.
• Participants must have a confirmed diagnosis of MVID (Microvillus Inclusion Disease), proven through genetic testing and/or a tissue sample.
• Participants must be able to take the study medication (a powder mixed into a liquid) either by mouth or through a feeding tube already placed in the stomach (G-tube or GJ-tube — not a J-tube alone).
• In the 8 weeks before the study starts, at least half of the participant's weekly fluid needs must have been met through parenteral support (fluids given through a vein or similar method).
• Female participants who have started their periods must agree to either abstain from sex or use two approved forms of birth control during the study and for 30 days after the last dose of the study drug.
• Male participants must agree to either abstain from sex or use contraception during the study and for 90 days after the last dose of the study drug.

Who may not be able to join:

• Participants who, in the last 4 weeks before the study starts, have had a significant change (more than 20%) in their fluid support needs.
• Participants who have newly started needing water-removing medications (diuretics) in the last 4 weeks.
• Participants who have had an infection requiring antibiotics given through a vein (IV) in the last 4 weeks.
• Participants who have had an active confirmed gut/stomach infection in the last 4 weeks.
• Participants who have started any new anti-diarrhea medication in the last 4 weeks.
• Participants who have had a significant rise in certain liver-related blood test results (ALT, AST, or bilirubin — confirm with trial site) in the last 4 weeks.
• Participants who have previously received an organ transplant.
• Participants who currently have any form of cancer.
• Participants who are pregnant or breastfeeding.
• Participants who the study doctor believes are unable to safely participate for any reason (confirm with trial site).
• Participants who are known to be allergic to any ingredient in the study medication.

Important: Always verify eligibility with the trial site directly before applying.