Actively Recruiting
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Led by Napo Pharmaceuticals, Inc. · Updated on 2025-06-12
12
Participants Needed
3
Research Sites
43 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants or their legal guardians sign informed consent
- Participants older than 7 years provide written assent if able
- Male or female participants aged 3 months to 17 years
- Confirmed genetic and/or histologic diagnosis of Microvillus Inclusion Disease (MVID)
- Ability to take crofelemer orally or via G-tube or GJ-tube (not J-tube)
- Receiving parenteral support providing at least 50% of weekly hydration volume for 8 weeks before baseline
- If female and post-menarche, agree to abstinence or use two birth control methods during treatment and 30 days after
- Male participants agree to abstinence or contraception during treatment and 90 days after
You will not qualify if you...
- Significant changes (> ±20%) in parenteral support needs within 4 weeks before study
- New requirement for diuretics within 4 weeks before study
- Any infection needing IV antibiotics within 4 weeks before study
- Active gastrointestinal infection documented within 4 weeks before study
- Started new anti-diarrheal drug within 4 weeks before study
- Increased ALT, AST, or bilirubin ≥2 times usual lab values within 4 weeks before study
- Previous organ transplant
- Current malignancy
- Pregnancy or breastfeeding
- Any other investigator-determined reason preventing study participation
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
UOS Gastroenterolgia e Riabilitazione nutrizionale Piazza Sant' Onofrio 4
Rome, Italy
Not Yet Recruiting
3
Al Jalila Children's Hospital
Dubai, United Arab Emirates
Actively Recruiting
Research Team
M
Maha Dakhloul, BSc.Pharmacy
CONTACT
S
Sara Papetti, MA
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
12
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