Dose Ranging Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Rectal Foam in Pediatric Patients Aged 5 to 17 Years With Active, Mild to Moderate Distal Ulcerative Colitis

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Dose Ranging Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Rectal Foam BID for 2 Weeks, Followed by QD for 4 Weeks, in Pediatric Patients Aged 5 to 17 Years With Active, Mild to Moderate Distal Ulcerative Colitis

The primary objective of this study is to evaluate the efficacy (as measured by induction of remission) of two dose levels (low and high) per age group (5 to <12 and 12 to ≤17 years) of budesonide rectal foam as compared to an equivalent volume of rectally administered placebo foam over the same dosing schedule, in pediatric subjects with active, mild to moderate distal ulcerative colitis (UC).

Study Overview

• Status: Not yet recruiting
• Conditions: Ulcerative Colitis
• Intervention / Treatment: Drug: High dose budesonide rectal foam; Drug: Low dose budesonide rectal foam; Drug: Matching placebo rectal foam

• Study Type: Interventional
• Enrollment (Estimated): 100
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Varsha Bhatt; Phone Number: 707-285-1528; Email: Varsha.Bhatt@bauschhealth.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male and Female (non-pregnant and non-lactating) 5 to ≤17 years of age at Screening.
2. Established diagnosis of UC, based on clinical history, characteristic endoscopic findings, and histopathology results from biopsies.
3. Disease limited to the distal colon and rectum, (disease involving only the first 15 centimeters or less proximal to the anal verge).
4. Active, distal UC of mild or moderate severity, defined as a Modified Mayo Clinic Score (mMCS) between 4 and 8, inclusive, that includes an endoscopy subscore ≥ 2 and a rectal bleeding subscore ≥ 1.
5. If on a background oral 5-ASA, the dose and formulation have remained unchanged for at least 6 weeks prior to Visit 2 (Run-In) and the subject is willing to remain on the same dosage form and regimen for the duration of the study.

Exclusion Criteria:

1. Current or prior diagnosis of Crohn's disease or indeterminate colitis.
2. Ulcerative colitis involving the proximal colon - i.e, the sigmoid, left, transverse, and/or ascending colon and cecum;
3. Severe UC, defined as an mMCS > 8.
4. Infectious colitis (based on positive microbiologic tests at Screening) or any recent history of infectious colitis (within 30 days of Screening).

5. 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.

6. Evidence or history of toxic megacolon or bowel resection.
7. Active proctologic pathology such as hemorrhoids, fistulas and fissures or other historical anatomic problems which would impair rectal administration (e.g., cloaca, imperforate anus history)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: High dose budesonide rectal foam	Drug: High dose budesonide rectal foam; Twice a day for 2 weeks, then once a day for 4 weeks
Experimental: Low dose budesonide rectal foam	Drug: Low dose budesonide rectal foam; Twice a day for 2 weeks, then once a day for 4 weeks
Placebo Comparator: Matching placebo rectal foam	Drug: Matching placebo rectal foam; Twice a day for 2 weeks, then once a day for 4 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with clinical remission	Clinical remission based on the modified Mayo Clinic Score, defined as an endoscopy score ≤ 1 (excluding friability), a rectal bleeding subscore of 0, and at least a 1-point decrease in stool frequency subscore from Baseline to achieve a stool frequency subscore ≤ 1. Each component is scored from 0 to 3, with total scores ranging from 0 to 9, with higher scores indicating more severe disease.	Day 42

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants who achieve a rectal bleeding subscale score of 0 at the end of six weeks of treatment	Scored from 0 to 3, with higher scores indicating more severe disease.	Day 42
Proportion of participants who achieve a stool frequency subscale score of 0 at the end of six weeks of treatment.	Scored from 0 to 3, with higher scores indicating more severe disease.	Day 42
Proportion of subjects who achieve an endoscopy subscale score of 0 or 1 at the end of six weeks of treatment.	Scored from 0 to 3, with higher scores indicating more severe disease.	Day 42

Collaborators and Investigators

• Sponsor: Bausch Health Americas, Inc.
• Investigators: Study Director: Varsha Bhatt, Bausch Health

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2024
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: July 28, 2023
• First Submitted That Met QC Criteria: July 28, 2023
• First Posted (Actual): August 4, 2023

Study Record Updates

• Last Update Posted (Actual): August 4, 2023
• Last Update Submitted That Met QC Criteria: July 28, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer; Colitis; Colitis, Ulcerative; Physiological Effects of Drugs; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Bronchodilator Agents; Anti-Asthmatic Agents; Respiratory System Agents; Budesonide
• Other Study ID Numbers: BFUC4991

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No