Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets in Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis

Official title

A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets n Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis

Overview

This is a randomized, double-blind, placebo-controlled, multicenter, study to evaluate the efficacy, safety, and pharmacokinetics (PK) of budesonide extended-release tablets for the induction of remission in pediatric subjects, with active, mild to moderate ulcerative colitis (UC). Subjects will be permitted to continue taking background oral or rectal 5-aminosalicylate (5-ASA) products.

Interventions

• Drug Low Dose Budesonide
  3 milligrams once daily for ages 5 to 11 years 11 months. 6 milligrams once daily for ages 12 to 17 years.
• Drug High Dose Budesonide
  6 milligrams once daily for ages 5 to 11 years 11 months. 9 milligrams once daily for ages 12 to 17 years.
• Drug Placebo
  Matching placebo once daily.

Primary outcome measures

• The primary efficacy endpoint is the proportion of subjects who achieve clinical remission at Day 56, defined as a total Mayo score of ≤ 1 with subscores of 0 for both rectal bleeding and stool frequency, and a subscore of ≤ 1 for endoscopy. [Time frame: 56 days]

Secondary outcome measures (3)

• Proportion of subjects who achieve a Mayo rectal bleeding subscale score of 0 at Day 56. [Time frame: 56 days]
• Proportion of subjects who achieve a Mayo stool frequency subscale score of 0 at Day 56. [Time frame: 56 days]
• Proportion of subjects who achieve a Mayo endoscopy subscale score of 0 or 1 at Day 56. [Time frame: 56 days]

Eligibility criteria

Inclusion criteria

• Established diagnosis of UC is based on:

• Clinical history
• Characteristic endoscopic findings
• Histopathology results from biopsies
• Age 5 to 17 years, inclusive, at Screening. Subjects should weigh greater than 13.6 kg at Baseline.
• Active UC of mild or moderate severity, defined as a total Mayo score between 4 and 10, inclusive, with rectal bleeding subscore of ≥ 1, stool frequency subscore of ≥1 and an endoscopy subscore of ≥ 1.
• If on a background oral or rectal 5-ASA, the dose and formulation have remained unchanged for at least 6 weeks prior to Visit 2 (randomization), Baseline and the subject is willing to remain on the same formulation and dose for the duration of the study.

Exclusion criteria

• Current or prior diagnosis of Crohn's disease or indeterminate colitis.
• Limited distal proctitis (disease involving only the first 15 centimeters or less proximal to the anal verge.
• Severe UC, defined as total Mayo score >10.
• Not currently in an active phase or flare, defined as a total Mayo score <4, or Mayo score between 4 and 10, but Mayo subscore of 0 for rectal bleeding, stool frequency or endoscopy.
• Infectious colitis (based on positive microbiologic tests at Screening) or any recent history of infectious colitis (within 30 days prior to Screening).
• Prior gastrointestinal surgery, except appendectomy or hernia (e.g., inguinal, umbilical).

NOTE: Prior cholecystectomy is not exclusionary if more than 1 year prior to Screening.

• Evidence or history of toxic megacolon or bowel resection.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Double blind

Primary purpose

Treatment

Study locations

United States · 4 centers

• Bausch Health Site 008 — Garden Grove
• Bausch Health Site 003 — Indianapolis
• Bausch Health Site 006 — The Bronx
• Bausch Health Site 010 — Greenville

Identifiers

NCT: NCT04314375 · BUUC4991

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗