Evaluation of Safety, Tolerability and Efficacy of Crofelemer following Multiple Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Participants with Microvillus Inclusion Disease (MVID)

Conditions

Microvillous Inclusion Disease (MVID)

Brief summary

Assessment of AEs, SAEs, laboratory evaluations, physical exam (including height, weight, BMI, and assessment of hydration status), and urine output

Detailed description

Assessment of change from baseline, compared to placebo, within each dose level and over the 24 weeks of treatment in the following endpoints: • Average daily loose/watery stool volume during 24-hour stool collections every 2 weeks., • Average daily loose/watery stool frequency during 24-hour stool collections every 2 weeks, • Average daily stool consistency using the 7point pediatric version of the Bristol Stool Form Scale during 24-hour stool collections every 2 weeks, • Daily stool electrolytes changes (Na+, K+, Cl-) at the end of dose level 3 (at 20 and 24 weeks), • Average weekly acetate supplementation, • Average weekly PS volume requirements, • Average weekly TPN volume requirements, • Average weekly supplemental IV fluid volume requirements, • Average weekly supplemental electrolytes (Na+, K+, Cl-) in PS and, separately, in TPN and IV fluids

Related papers

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Interventions

DRUGCrofelemer Placebo Powder for Oral Solution

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Assessment of AEs, SAEs, laboratory evaluations, physical exam (including height, weight, BMI, and assessment of hydration status), and urine output	—

Secondary

Measure	Time frame
Assessment of change from baseline, compared to placebo, within each dose level and over the 24 weeks of treatment in the following endpoints: • Average daily loose/watery stool volume during 24-hour stool collections every 2 weeks., • Average daily loose/watery stool frequency during 24-hour stool collections every 2 weeks, • Average daily stool consistency using the 7point pediatric version of the Bristol Stool Form Scale during 24-hour stool collections every 2 weeks, • Daily stool electrolytes changes (Na+, K+, Cl-) at the end of dose level 3 (at 20 and 24 weeks), • Average weekly acetate supplementation, • Average weekly PS volume requirements, • Average weekly TPN volume requirements, • Average weekly supplemental IV fluid volume requirements, • Average weekly supplemental electrolytes (Na+, K+, Cl-) in PS and, separately, in TPN and IV fluids	—

Measure

Time frame

Assessment of change from baseline, compared to placebo, within each dose level and over the 24 weeks of treatment in the following endpoints: • Average daily loose/watery stool volume during 24-hour stool collections every 2 weeks., • Average daily loose/watery stool frequency during 24-hour stool collections every 2 weeks, • Average daily stool consistency using the 7point pediatric version of the Bristol Stool Form Scale during 24-hour stool collections every 2 weeks, • Daily stool electrolytes changes (Na+, K+, Cl-) at the end of dose level 3 (at 20 and 24 weeks), • Average weekly acetate supplementation, • Average weekly PS volume requirements, • Average weekly TPN volume requirements, • Average weekly supplemental IV fluid volume requirements, • Average weekly supplemental electrolytes (Na+, K+, Cl-) in PS and, separately, in TPN and IV fluids

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Countries

Italy

Outcome results

None listed