Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06721871 | Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID) | PHASE2 | RECRUITING | 12 | — | — | May 1, 2025 | Mar 1, 2026 | Jun 12, 2025 | 3 | United States, Italy +1 |
Incidence of Adverse Events and Serious Adverse Events
Changes from baseline in physical exam and signs of dehydration, such as decreased urine output, sunken eyes, lethargy and abnormal skin turgor.
Incidence in changes from baseline of individual lab values within a chemistry, hematology and metabolic panel analysis.
| Arm | Type | Description |
|---|---|---|
| Dose Level 1/Treatment Period 1: 1mg/kg/dose 3x/day | EXPERIMENTAL | Participants may be randomized to crofelemer powder for oral solution during Treatment Period 1 (1 month duration) |
| Dose Level 1/Treatment Period 1: Placebo 3x/day | PLACEBO_COMPARATOR | Participants may be randomized to the placebo comparator during Treatment Period 1 (1 month duration) |
| Dose Level 1/Treatment Period 2: 1mg/kg/dose 3x/day | EXPERIMENTAL | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 2/Treatment Period 1: 3mg/kg/dose 3x/day | EXPERIMENTAL | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) |
| Dose Level 2/Treatment Period 1: Placebo 3x/day | PLACEBO_COMPARATOR | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 1 (1 month duration) |
| Dose Level 2/Treatment Period 2: 3mg/kg/dose 3x/day | EXPERIMENTAL | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 2/Treatment Period 2: Placebo 3x/day | PLACEBO_COMPARATOR | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 3/Treatment Period 1: 10mg/kg/dose 3x/day | EXPERIMENTAL | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solution or the placebo comparator that they were previously on during Dose Level 2 (1 month duration) |
| Dose Level 3/Treatment Period 1: Placebo 3x/day | PLACEBO_COMPARATOR | Participants will crossover to either crofelemer powder for oral solution or placebo comparator or continue on crofelemer powder for oral solutionor the placebo comparator that they were previously on during Dose Level 2 (1 month duration) |
| Dose Level 3/Treatment Period 2: 10mg/kg/dose 3x/day | EXPERIMENTAL | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 3/Treatment Period 2: Placebo 3x/day | PLACEBO_COMPARATOR | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Dose Level 1/Treatment Period 2: Placebo 3x/day | PLACEBO_COMPARATOR | Participants will crossover to either crofelemer powder for oral solution or placebo comparator |
| Name | Type | Description |
|---|---|---|
| Crofelemer Powder for Oral Solution | DRUG | Crofelemer Powder for Oral Solution |
| Placebo Powder for Oral Solution | DRUG | Matching Placebo Powder for Oral Solution |
Inclusion Criteria: 1. Participants (assent for participants older than 7 years of age) and/or their legal parent/guardian sign an Informed Consent Form (ICF) indicating that they understand the purpose of the procedures required for the study and are willing to participate 2. When appropriate, ped...