A Study of Ustekinumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis (UC)

Janssen Research & Development, LLC112 enrolled

Overview

The purpose of this study is to evaluate: a) the efficacy of ustekinumab dosing in inducing clinical remission, b) safety profile of ustekinumab, and c) ustekinumab exposure (pharmacokinetics \[PK\]) in pediatric participants with moderately to severely active UC.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

112

Conditions

Colitis, Ulcerative

Interventions

Ustekinumab Dose Based on BSA and Body WeightDRUG

As per BSA and body weight Ustekinumab will be administered SC and IV.

Matching PlaceboDRUG

Placebo will be administered subcutaneously.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Medically stable on the basis of physical examination, medical history, and vital signs, performed at screening. Any abnormalities must be consistent with the underlying illness in the study population and this determination must be recorded in the participant's source documents and acknowledged by the investigator * Must have had UC diagnosed prior to screening * Have moderately to severely active UC, defined as a baseline Mayo score of 6 through 12, inclusive, with a screening Mayo endoscopy subscore greater than or equal to (\>=) 2 as determined by a central review of the video of the endoscopy * A participant who has had extensive colitis for \>= 8 years, or disease limited to the left side of the colon for \>= 10 years, must: a) have had a full colonoscopy to assess for the presence of dysplasia within 1 year before the first administration of study intervention or b) have a full colonoscopy with surveillance for dysplasia as the baseline endoscopy during the screening period. Results from these surveillance biopsies must be negative for dysplasia (low-grade, high-grade, or indeterminant) prior to the first administration of study intervention * Females of childbearing potential must have a negative highly sensitive urine pregnancy test at screening and at Week I-0 prior to study intervention administration Exclusion Criteria: * Have UC limited to the rectum only or to less than (\<) 20 centimeter (cm) of the colon * Presence or history of colonic or small bowel obstruction within 6 months prior to screening, confirmed by objective radiographic or endoscopic evidence of a stricture with resulting obstruction (dilation of the colon or small bowel proximal to the stricture on barium radiograph or an inability to traverse the stricture at endoscopy) * Have a history of latent or active granulomatous infection, histoplasmosis, or coccidioidomycosis, or have had a nontuberculous mycobacterial infection prior to screening * Presence or history of any malignancy including presence or history of lymphoproliferative disease including lymphoma, or signs and symptoms suggestive of possible lymphoproliferative disease, such as lymphadenopathy of unusual size or location (example, nodes in the posterior triangle of the neck, infraclavicular, epitrochlear, or periaortic areas) and monoclonal gammopathy of undetermined significance, or clinically significant hepatomegaly or splenomegaly * Has known allergies, hypersensitivity, or intolerance to ustekinumab or its excipients

Locations (58)

Outcomes

Primary Outcomes

Global: Number of Participants with Clinical Remission at Induction Week 8 (I-8) Visit

Clinical remission is defined as Mayo stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.

Time frame: Week 8

Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability

An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

Time frame: Up to 74 weeks

Number of Participants with Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability

SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect, and suspects transmission of any infectious agent via a medicinal product.

Time frame: Up to 74 weeks

Number of Participants with AEs Leading to Discontinuation of Study Intervention

Number of Participants with discontinuation of study intervention due to an AE, infections, injection-site reactions, and AEs during or within 1 hour of an infusion will be reported.

Time frame: Up to 74 weeks

Number of Participants with AEs of Special Interest (AESI) as a Measure of Safety and Tolerability

AESI of any newly identified malignancy, case of active tuberculosis (TB), or opportunistic infection occurring after the first administration of study intervention(s) in participants will be reported.

Time frame: Up to 74 weeks

Number of Participants with Laboratory Abnormalities

Data from ClinicalTrials.gov

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Nemours DuPont Hospital for Children

Wilmington, Delaware, United States

Children's Center for Digestive Health Care

Atlanta, Georgia, United States

Mayo Clinic

Rochester, Minnesota, United States

Morristown Memorial Hospital

Morristown, New Jersey, United States

Levine Childrens at Atrium Health

Charlotte, North Carolina, United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, United States

Cook Childrens Medical Center

Fort Worth, Texas, United States

Pediatric Specialists Of Virginia

Fairfax, Virginia, United States

Universitair Kinderziekenhuis Koningin Fabiola

Brussels, Belgium

...and 48 more locations

Secondary Outcomes

Number of Participants With Clinical Response at I-8 Visit

Clinical response is defined as decrease from baseline in the modified Mayo score by \>= 30 percent (%) and \>=2 points, with either a decrease from baseline in the rectal bleeding subscore of \>= 1 or a rectal bleeding subscore of 0 or 1.

Time frame: Week 8

Number of Participants with Symptomatic Remission at I-8 Visit

Symptomatic remission is defined as Mayo stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.

Time frame: Week 8

Clinical Remission at I-8 as Assessed by the Pediatric Ulcerative Colitis Activity Index Score (PUCAI) Score

Clinical remission is defined as a PUCAI score less than (\<)10.

Time frame: Week 8

Endoscopic Improvement at I-8 Visit

Endoscopic improvement is defined as a Mayo endoscopy subscore of 0 or \<= 1 with no friability present on the endoscopy.

Time frame: Week 8

Histologic-endoscopic Mucosal Improvement at Week I-8

Histologic-endoscopic mucosal improvement is defined as achieving a combination of histologic (neutrophil infiltration in less than \[\<\] 5% of crypts, no crypt destruction, and no erosions, ulcerations or granulation tissue according to the Geboes grading system) and endoscopic improvement (endoscopy subscore of 0 or 1 with no friability present on the endoscopy).

Time frame: Week 8

Number of Participants with Clinical Remission at Week 44 (M-44) Visit

Clinical remission is defined as a Mayo stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.

Time frame: Week 52

Number of Participants with Symptomatic Remission at M-44 Visit

Symptomatic remission is defined as Mayo stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.

Time frame: Week 52

Clinical Remission at M-44 as Assessed by the PUCAI Score

Clinical remission is defined as a PUCAI score less than \< 10.

Time frame: Week 52

Endoscopic Improvement at M-44 Visit

Endoscopic improvement is defined as a Mayo endoscopy subscore of 0 or \<= 1 with no friability present on the endoscopy.

Time frame: Week 52

Corticosteroid-free Clinical Remission at Week M-44

Corticosteroid-free clinical remission is defined as a Mayo stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline; and not receiving corticosteroids for at least 90 days prior to Week M-44.

Time frame: Week 52

Clinical Remission at M-44 and not Receiving Corticosteroids for at Least 90 Days Prior to M-44 Among Participants who Received Corticosteroids at M-0

Clinical remission is defined as a PUCAI score less than \< 10. Clinical remission at M-44 and in participants not receiving corticosteroids for at least 90 days prior to M-44 among participants who received corticosteroids at M-0 as assessed by PUCAI score will be reported.

Time frame: Week 52

Clinical Remission at M-44 for Participants who are in Clinical Remission at I-8

Clinical remission at M-44 for participants who are in clinical remission at I-8 will be reported. Clinical remission is defined as a Mayo stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.

Time frame: Week 52

US Specific: Number of Participants with Clinical Remission at I-8 Visit

Time frame: Week 8

Histologic-endoscopic Mucosal Improvement at Week M-44

Histologic-endoscopic mucosal improvement is defined as achieving a combination of histologic (neutrophil infiltration in \< 5% of crypts, no crypt destruction, and no erosions, ulcerations or granulation tissue according to the Geboes grading system) and endoscopic improvement (endoscopy subscore of 0 or 1 with no friability present on the endoscopy).

Time frame: Week 52