Clinical trial NCT04314375: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Ulcerative Colitis
Lead sponsor: Bausch Health Americas, Inc.
Sponsor class: INDUSTRY
First posted: 2020-03-19
Enrollment: 70
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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:

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.

Primary outcomes:
measure: 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.
timeFrame: 56 days
Secondary outcomes:
measure: Proportion of subjects who achieve a Mayo rectal bleeding subscale score of 0 at Day 56.
timeFrame: 56 days
measure: Proportion of subjects who achieve a Mayo stool frequency subscale score of 0 at Day 56.
timeFrame: 56 days
measure: Proportion of subjects who achieve a Mayo endoscopy subscale score of 0 or 1 at Day 56.
timeFrame: 56 days
Other outcomes:
Locations:
city: Garden Grove
country: United States
facility: Bausch Health Site 008
state: California
city: Indianapolis
country: United States
facility: Bausch Health Site 003
state: Indiana
city: Bronx
country: United States
facility: Bausch Health Site 006
state: New York
city: Greenville
country: United States
facility: Bausch Health Site 010
state: North Carolina
Study officials:
affiliation: Bausch Health
name: Varsha Bhatt
role: STUDY_DIRECTOR
References: