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

Official title

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

Overview

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).

Interventions

• Drug High dose budesonide rectal foam
  Twice a day for 2 weeks, then once a day for 4 weeks
• Drug Low dose budesonide rectal foam
  Twice a day for 2 weeks, then once a day for 4 weeks
• Drug Matching placebo rectal foam
  Twice a day for 2 weeks, then once a day for 4 weeks

Primary outcome measures

• Proportion of participants with clinical remission [Time frame: Day 42]

Secondary outcome measures (3)

• Proportion of participants who achieve a rectal bleeding subscale score of 0 at the end of six weeks of treatment [Time frame: Day 42]
• Proportion of participants who achieve a stool frequency subscale score of 0 at the end of six weeks of treatment. [Time frame: Day 42]
• Proportion of subjects who achieve an endoscopy subscale score of 0 or 1 at the end of six weeks of treatment. [Time frame: Day 42]

Eligibility criteria

Inclusion criteria

• Male and Female (non-pregnant and non-lactating) 5 to ≤17 years of age at Screening.
• Established diagnosis of UC, based on clinical history, characteristic endoscopic findings, and histopathology results from biopsies.
• Disease limited to the distal colon and rectum, (disease involving only the first 15 centimeters or less proximal to the anal verge).
• 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.
• 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

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

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

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT05976802 · BFUC4991

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗