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

A Phase 3 Study of the Efficacy, Safety and Pharmacokinetics of Ustekinumab as Open-label Intravenous Induction Treatment Followed by Randomized Double-blind Subcutaneous Ustekinumab Maintenance in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

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 Overview

• Status: Completed
• Conditions: Colitis, Ulcerative
• Intervention / Treatment: Drug: Ustekinumab Dose Based on BSA and Body Weight; Drug: Matching Placebo

• Study Type: Interventional
• Enrollment (Actual): 112
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium, 1200
    • Cliniques Universitaires Saint Luc
  • Brussels, Belgium, 1020
    • Universitair Kinderziekenhuis Koningin Fabiola
  • Ghent, Belgium, 9000
    • UZ Gent
  • Jette, Belgium, 1090
    • UZ Brussel
  • Leuven, Belgium, 3000
    • UZ Leuven
• Germany
  • Aachen, Germany, 52074
    • Universitätsklinikum Aachen
  • Berlin, Germany, 13353
    • Charite-Universitätsmedizin Berlin - Berlin
  • Erlangen, Germany, 91054
    • Universitätsklinikum Erlangen
  • Essen, Germany, 45147
    • Universitätsklinikum Essen
  • Hanover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Munich, Germany, 80337
    • Dr. von Haunersches Kinderspital
  • Regensburg, Germany, 93049
    • KUNO Klinik St. Hedwig
  • Ulm, Germany, 89075
    • Universitätsklinikum Ulm
• Hungary
  • Budapest, Hungary, 1083
    • Semmelweis Egyetem
  • Debrecen, Hungary, 4032
    • Debreceni Egyetem Klinikai Kozpont
  • Miskolc, Hungary, 3526
    • Borsod Abauj Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktato Korhaz
  • Nyíregyháza, Hungary, 4400
    • Szabolcs Szatmar Bereg Varmegyei Oktatokorhaz
  • Szeged, Hungary, 6720
    • Szegedi Tudományegyetem, Gyermekgyógyászati Klinika és Gyermekegészségügyi Centrum
• Israel
  • Be’er Ya‘aqov, Israel, 70300
    • Shamir Medical Center Assaf Harofeh
  • Haifa, Israel, 3109601
    • Rambam Medical Center
  • Haifa, Israel, 3436212
    • Carmel Medical Center
  • Jerusalem, Israel, 9103102
    • Shaare Zedek Medical Center
  • Petah Tikva, Israel, 4920235
    • Schneider Children's Medical Center
  • Ramat Gan, Israel, 30700
    • Sheba Medical Center
• Japan
  • Bunkyō City, Japan, 113 8431
    • Juntendo University Hospital
  • Gunma, Japan, 371-0034
    • Gunma University Hospital
  • Ikoma, Japan, 630-0293
    • Kindai University Nara Hospital
  • Kurume, Japan, 830-0011
    • Kurume University Hospital
  • Saitama Shi, Japan, 330-8777
    • Saitama Childrens Medical Center
  • Sendai, Japan, 989-3126
    • Miyagi Children's Hospital
  • Setagaya Ku, Japan, 157 8535
    • National Center for Child Health and Development
  • Shimotsuke, Japan, 329-0498
    • Jichi Medical University Hospital
  • Tsu, Japan, 514 8507
    • Mie University Hospital
• Poland
  • Gdansk, Poland, 80 803
    • Szpital im. M. Kopernika
  • Krakow, Poland, 30 663
    • Uniwersytecki Szpital Dzieciecy w Krakowie
  • Rzeszów, Poland, 35-302
    • Korczowski Bartosz Gabinet Lekarski
  • Torun, Poland, 87 100
    • GASTROMED Sp. z o.o.
  • Warsaw, Poland, 04 501
    • WIP Warsaw IBD Point Profesor Kierkus
  • Warsaw, Poland, 04 730
    • Instytut Pomnik Centrum Zdrowia Dziecka
• Russia
  • Kazan', Russia, 420138
    • Kazan State Medical University
  • Moscow, Russia, 119991
    • FSBI 'Scientific Centre of Children Health' of the Russian Academy of Medical Sciences
  • Moscow, Russia, 119571
    • Russian National Research Medical University named after N.I.Pirogov
  • Nizhny Novgorod, Russia, 603950
    • Privolzhsky Research Medical University of Ministry of Health of Russian Federation
  • Saratov, Russia, 410054
    • Saratov State Medical University
  • Yaroslavl, Russia, 150032
    • Yaroslavl Regional Children's Clinical Hospital
• United Kingdom
  • Birmingham, United Kingdom, B4 6NH
    • Birmingham Children's Hospital
  • Bristol, United Kingdom, BS2 8BJ
    • University Hospitals Bristol and Weston NHS Foundation Trust
  • Cambridge, United Kingdom, CB2 0QQ
    • Cambridge University Hospitals NHS Foundation Trust
  • London, United Kingdom, E1 2AT
    • Royal London Hospital
• United States
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Nemours DuPont Hospital for Children
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Children's Center for Digestive Health Care
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • New Jersey
    • Morristown, New Jersey, United States, 07962
      • Morristown Memorial Hospital
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Levine Childrens at Atrium Health
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Hershey Children's Hospital
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Childrens Medical Center
  • Virginia
    • Fairfax, Virginia, United States, 22031
      • Pediatric Specialists Of Virginia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

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

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: Induction Period (I): Ustekinumab; All participants will receive a single intravenous (IV) administration of ustekinumab at induction Week 0 (I-0) based on body surface area (BSA) (milligram per meter square [mg/m^2]) or weight-tiered induction dose (milligram per kilogram [mg/kg]).	Drug: Ustekinumab Dose Based on BSA and Body Weight; As per BSA and body weight Ustekinumab will be administered SC and IV.; Other Names: STELARA
Experimental: Maintenance (M) Period: Ustekinumab once every 8 Week (q8w); Participants will receive subcutaneous (SC) administration of ustekinumab every 8 weeks (q8w) based on BSA (mg/m^2) or weight-tiered induction dose (mg/kg) at Weeks M-0, M-8, M-16, M-24, M-32, M-40 and matching placebo at Weeks M-12 and M-36 to maintain the blind.	Drug: Ustekinumab Dose Based on BSA and Body Weight; As per BSA and body weight Ustekinumab will be administered SC and IV.; Other Names: STELARA; Drug: Matching Placebo; Placebo will be administered subcutaneously.
Experimental: Maintenance (M) Period: Ustekinumab once every 12 Week (q12w); Participants will receive SC administration of ustekinumab every 12 weeks (q12w) based on BSA (mg/m^2) or weight-tiered induction dose (mg/kg) at Weeks M-0, M-12, M-24, M-36 and matching placebo at Weeks M-8, M-16, M-32, and M-40 to maintain the blind.	Drug: Ustekinumab Dose Based on BSA and Body Weight; As per BSA and body weight Ustekinumab will be administered SC and IV.; Other Names: STELARA; Drug: Matching Placebo; Placebo will be administered subcutaneously.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	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.	Up to 74 weeks
Number of Participants with Laboratory Abnormalities	Number of participants with laboratory abnormalities related to hematology, serum chemistry, and coagulation will be reported.	Up to 74 weeks
Reactions Temporally Associated with an Intravenous (IV) Infusion and Subcutaneous (SC) Injection-site Reactions	Reactions temporally associated with an IV infusion (induction period) and SC injection-site reactions (maintenance period) will be reported.	Up to 74 weeks
Serum Concentration of Ustekinumab	Serum samples will be analyzed to determine concentrations of ustekinumab.	Up to 74 weeks
US Specific: Clinical Remission at M-44 for Participants who are in Clinical Response at I-8	Clinical remission at M-44 for participants who are in clinical response 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.	Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	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).	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.	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.	Week 52
Clinical Remission at M-44 as Assessed by the PUCAI Score	Clinical remission is defined as a PUCAI score less than < 10.	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.	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.	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.	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.	Week 52
US Specific: Number of Participants with Clinical Remission at 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.	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).	Week 52

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC
• Investigators: Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

Study Major Dates

• Study Start (Actual): March 17, 2021
• Primary Completion (Actual): May 8, 2025
• Study Completion (Actual): June 5, 2025

Study Registration Dates

• First Submitted: November 13, 2020
• First Submitted That Met QC Criteria: November 13, 2020
• First Posted (Actual): November 16, 2020

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 7, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Colitis; Colitis, Ulcerative; Amino Acids, Peptides, and Proteins; Proteins; Investigative Techniques; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Ustekinumab; Weights and Measures
• Other Study ID Numbers: CR108865; 2019-004224-38 (EudraCT Number); CNTO1275PUC3001 (Other Identifier: Janssen Research & Development, LLC); 2023-504977-19-00 (Registry Identifier: EUCT number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/ transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

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