This is a Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel-group Study of the Efficacy and Safety of 2 Weight-based Treatment Groups of Plecanatide Versus Placebo in Children and Adolescent Participants 6 to Less Than 18 Years of Age With FC
brief summary
The goal of this clinical trial is to learn if plecanatide can treat functional constipation in children and adolescents aged 6 to less than 18 years. It will also learn about the safety and pharmacokinetics of plecanatide in this population. The main questions it aims to answer are: * Does plecanatide increase the number of spontaneous bowel movements compared to placebo after 8 weeks of treatment? * What medical problems do participants experience when taking plecanatide? Researchers will compare low-dose plecanatide and high-dose plecanatide to placebo to see if plecanatide improves bowel movement frequency, stool consistency, and constipation-related symptoms. Participants will: * Complete a screening period with daily electronic diary entries to record bowel movements, symptoms, and rescue medication use * Take plecanatide or placebo by mouth once daily for 8 weeks * Continue daily electronic diary entries throughout the study * Attend clinic visits for physical exams, laboratory tests, and assessments of symptoms and safety * Provide blood samples for pharmacokinetic and safety evaluations * Complete questionnaires about constipation symptoms and quality of life
detailed description
This Phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group study is designed to evaluate the efficacy, safety, and pharmacokinetics (PK) of plecanatide administered orally once daily in pediatric participants with functional constipation (FC). Approximately 180 participants will be enrolled at up to 30 investigational sites in the United States and randomized in a 1:1:1 ratio to receive low-dose plecanatide, high-dose plecanatide, or matching placebo. Randomization will be stratified by age group (6 to 11 years and 12 to less than 18 years) and sex.
Background and Rationale Functional constipation is a common condition in children and adolescents and is characterized by infrequent bowel movements, hard stool consistency, and associated symptoms such as straining and abdominal discomfort. Available treatment options in the pediatric population are limited, and there remains a need for additional therapies.
Plecanatide is an orally administered guanylate cyclase-C (GC-C) agonist that acts locally in the gastrointestinal tract to increase intestinal fluid secretion and transit. Clinical studies in adults with chronic idiopathic constipation have demonstrated improvements in bowel movement frequency and stool consistency, as well as an acceptable safety profile. The current study is designed to evaluate plecanatide in a pediatric population using a weight-based dosing approach intended to achieve exposure levels comparable to those associated with efficacy in adults.
Study Design The study consists of three periods: a Screening/Baseline Period, an 8-week Treatment Period, and a Post-treatment Follow-up Period. The total duration of participation is approximately 14 weeks (98 days).
During the Screening/Baseline Period (up to 28 days), participants and/or caregivers complete daily assessments using an electronic diary (eDiary) to record bowel movements, stool characteristics, and constipation-related symptoms. Data from the eDiary are used to establish baseline values and confirm eligibility prior to randomization.
Participants who meet all eligibility criteria are randomized on Day 1 and enter the Treatment Period. Study drug is administered once daily for 8 weeks. Participants are required to continue daily eDiary entries throughout the Treatment Period to capture bowel movement frequency, stool consistency, symptom severity, and use of rescue medication.
Following completion of the Treatment Period, participants enter a 2-week Post-treatment Follow-up Period, during which eDiary assessments continue. A final study visit is conducted at the end of follow-up.
official title
A Phase 2b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study of the Efficacy and Safety of Plecanatide in Children and Adolescents Age 6 to <18 Years of Age With Functional Constipation (FC)