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

Phase 4

Recruiting

• Conditions: Ulcerative Colitis
• Interventions: Drug: Placebo; Drug: Low Dose Budesonide; Drug: High Dose Budesonide

• Registration Number: NCT04314375
• Lead Sponsor: Bausch Health Americas, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-03-17
• Study First Submitted QC: 2020-03-17
• Study First Posted: 2020-03-19
• Study Start: 2023-09-29
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-05
• Last Update Posted: 2023-10-10
• Primary Completion: 2024-12
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 70

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

1. Current or prior diagnosis of Crohn's disease or indeterminate colitis.

2. Limited distal proctitis (disease involving only the first 15 centimeters or less proximal to the anal verge.

3. Severe UC, defined as total Mayo score >10.

4. 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.

5. Infectious colitis (based on positive microbiologic tests at Screening) or any recent history of infectious colitis (within 30 days prior to Screening).

6. 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.

7. Evidence or history of toxic megacolon or bowel resection.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
Low Dose Budesonide	Low Dose Budesonide	-
High Dose Budesonide	High Dose Budesonide	-

Primary Outcome Measures

Name	Time	Method
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.	56 days	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. The Mayo score is a composite index score based on 4 components: stool frequency (subject reported), rectal bleeding (subject reported), findings of endoscopy, and the physician's rating of disease activity. Each component is scored from 0 to 3, with total scores range from 0 to 12 with higher scores indicating more severe disease.

Secondary Outcome Measures

Name	Time	Method
Proportion of subjects who achieve a Mayo rectal bleeding subscale score of 0 at Day 56.	56 days	Proportion of subjects who achieve a Mayo rectal bleeding subscale score of 0 at Day 56. The Mayo score is a composite index score based on 4 components: stool frequency (subject reported), rectal bleeding (subject reported), findings of endoscopy, and the physician's rating of disease activity. Each component is scored from 0 to 3, with total scores range from 0 to 12 with higher scores indicating more severe disease.
Proportion of subjects who achieve a Mayo endoscopy subscale score of 0 or 1 at Day 56.	56 days	Proportion of subjects who achieve a Mayo endoscopy subscale score of 0 or 1 at Day 56. The Mayo score is a composite index score based on 4 components: stool frequency (subject reported), rectal bleeding (subject reported), findings of endoscopy, and the physician's rating of disease activity. Each component is scored from 0 to 3, with total scores range from 0 to 12 with higher scores indicating more severe disease.
Proportion of subjects who achieve a Mayo stool frequency subscale score of 0 at Day 56.	56 days	Proportion of subjects who achieve a Mayo stool frequency subscale score of 0 at Day 56. The Mayo score is a composite index score based on 4 components: stool frequency (subject reported), rectal bleeding (subject reported), findings of endoscopy, and the physician's rating of disease activity. Each component is scored from 0 to 3, with total scores range from 0 to 12 with higher scores indicating more severe disease.

Trial Locations

Locations (4)

Bausch Health Site 008; 🇺🇸 Garden Grove, California, United States

Bausch Health Site 003; 🇺🇸 Indianapolis, Indiana, United States

Bausch Health Site 006; 🇺🇸 Bronx, New York, United States

Bausch Health Site 010; 🇺🇸 Greenville, North Carolina, United States