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Assessing the safety, tolerability, and effectiveness of crofelemer in people with the intestinal disease Microvillus Inclusion Disease (MVID) through multiple doses of this new drug
EU CTIS ID: 2024-512845-18-00
What this study is testing
To evaluate safety and tolerability of 3 ascending doses of Crofelemer Powder for Oral Solution, compared to placebo, when administered to participants with MVID receiving PS
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participants (and/or their legal caregiver and/or participant assent for participants older than 7 years of age) sign an Informed Consent Form (ICF) indicating they understand the purpose of the procedures required for the study and are willing to participate.
- 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.
- Male or female participants between the ages of 3 months to 17 years at the time of signing the informed consent by their legal caregiver and/or participant assent for eligible participants
- Have a confirmed diagnosis (genetic and/or histologic) of MVID
- Are able to ingest reconstituted Crofelemer Powder for Oral Solution either orally (PO) or through a previously placed G-tube or GJ-Tube (not via J-Tube)
- Have required, during the 8 weeks prior to baseline, an average weekly volume of PS that represents ≥ 50% of the participant’s average weekly hydration volume requirements
You likely can't join if
- 1. Within the last 4 weeks before study day -2 (day of study initiation), participants have: a. had significant changes to PS requirements (i.e., ± > 20%)
- 4. is pregnant or breastfeeding
- 6. Known hypersensitivity to any of the components of study drug
- 5. any investigator determined criteria for inability to participate in this study
- b. had a new requirement for diuretics
- c. had any infection requiring IV antibiotic administration
See the full eligibility criteria
- Participants (and/or their legal caregiver and/or participant assent for participants older than 7 years of age) sign an Informed Consent Form (ICF) indicating they understand the purpose of the procedures required for the study and are willing to participate.
- 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.
- Male or female participants between the ages of 3 months to 17 years at the time of signing the informed consent by their legal caregiver and/or participant assent for eligible participants
- Have a confirmed diagnosis (genetic and/or histologic) of MVID
- Are able to ingest reconstituted Crofelemer Powder for Oral Solution either orally (PO) or through a previously placed G-tube or GJ-Tube (not via J-Tube)
- Have required, during the 8 weeks prior to baseline, an average weekly volume of PS that represents ≥ 50% of the participant’s average weekly hydration volume requirements
- If female participants have reached menarche, the participant (and caregiver) agree that the participant will remain abstinent or use two accepted methods of birth control during the course of the treatment period and for an additional 30 days following the last dose of study drug.
- Male participants (and caregiver) agree that the participant will remain abstinent or use contraception during the course of the treatment period and continue on for an additional 90 days following the last dose of study drug.
- 1. Within the last 4 weeks before study day -2 (day of study initiation), participants have: a. had significant changes to PS requirements (i.e., ± > 20%)
- 4. is pregnant or breastfeeding
- 6. Known hypersensitivity to any of the components of study drug
- 5. any investigator determined criteria for inability to participate in this study
- b. had a new requirement for diuretics
- c. had any infection requiring IV antibiotic administration
- d. had a documented active gastrointestinal infection
- e. initiated any new anti-diarrheal drug
- f. had an increase in ALT, AST, or total bilirubin that is ≥2 times the participant’s usual laboratory values within the past 3 months04. had a documented active gastrointestinal infection
- 2. previously received an organ transplant
- 3. any currently diagnosed malignancy
The study team makes the final eligibility decision.
Where it's taking place
- United Arab Emirates
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Arab Emirates; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.