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

NCT04630028 | Completed Phase 3 DMC FDA Drug

• 4 other identifiers: CR108865CNTO1275PUC30012019-004224-382023-504977-19-00
• Study Type: interventional
• Target: 112
• Locations: 9 countries
• Sites: 58

Brief Summary

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.

Conditions

Colitis, Ulcerative

Outcome Measures

Primary Outcomes (9)

• 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 Outcomes (14)

• Number of Participants With Clinical Response at I-8 Visit

  Week 8

• Number of Participants with Symptomatic Remission at I-8 Visit

  Week 8

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

  Week 8

• Endoscopic Improvement at I-8 Visit

  Week 8

• Histologic-endoscopic Mucosal Improvement at Week I-8

  Week 8

Study Arms (3)

Induction Period (I): Ustekinumab

EXPERIMENTAL

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

Maintenance (M) Period: Ustekinumab once every 8 Week (q8w)

EXPERIMENTAL

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; Drug: Matching Placebo

Maintenance (M) Period: Ustekinumab once every 12 Week (q12w)

EXPERIMENTAL

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; Drug: Matching Placebo

Interventions

Ustekinumab Dose Based on BSA and Body Weight DRUG

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

Also known as: STELARA

Induction Period (I): Ustekinumab; Maintenance (M) Period: Ustekinumab once every 12 Week (q12w); Maintenance (M) Period: Ustekinumab once every 8 Week (q8w)

Matching Placebo DRUG

Placebo will be administered subcutaneously.

Maintenance (M) Period: Ustekinumab once every 12 Week (q12w); Maintenance (M) Period: Ustekinumab once every 8 Week (q8w)

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (58)

Nemours DuPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's Center for Digestive Health Care

Atlanta, Georgia, 30342, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Morristown Memorial Hospital

Morristown, New Jersey, 07962, United States

Location

Levine Childrens at Atrium Health

Charlotte, North Carolina, 28207, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17033, United States

Location

Cook Childrens Medical Center

Fort Worth, Texas, 76104, United States

Location

Pediatric Specialists Of Virginia

Fairfax, Virginia, 22031, United States

Location

Universitair Kinderziekenhuis Koningin Fabiola

Brussels, 1020, Belgium

Location

Cliniques Universitaires Saint Luc

Brussels, 1200, Belgium

Location

UZ Gent

Ghent, 9000, Belgium

Location

UZ Brussel

Jette, 1090, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Universitätsklinikum Aachen

Aachen, 52074, Germany

Location

Charite-Universitätsmedizin Berlin - Berlin

Berlin, 13353, Germany

Location

Universitatsklinikum Erlangen

Erlangen, 91054, Germany

Location

Universitatsklinikum Essen

Essen, 45147, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Dr. von Haunersches Kinderspital

Munich, 80337, Germany

Location

KUNO Klinik St. Hedwig

Regensburg, 93049, Germany

Location

Universitatsklinikum Ulm

Ulm, 89075, Germany

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, 4032, Hungary

Location

Borsod Abauj Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktato Korhaz

Miskolc, 3526, Hungary

Location

Szabolcs Szatmar Bereg Varmegyei Oktatokorhaz

Nyíregyháza, 4400, Hungary

Location

Szegedi Tudományegyetem, Gyermekgyógyászati Klinika és Gyermekegészségügyi Centrum

Szeged, 6720, Hungary

Location

Shamir Medical Center Assaf Harofeh

Be’er Ya‘aqov, 70300, Israel

Location

Rambam Medical Center

Haifa, 3109601, Israel

Location

Carmel Medical Center

Haifa, 3436212, Israel

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

Schneider Children's Medical Center

Petah Tikva, 4920235, Israel

Location

Sheba Medical Center

Ramat Gan, 30700, Israel

Location

Juntendo University Hospital

Bunkyō City, 113 8431, Japan

Location

Gunma University Hospital

Gunma, 371-0034, Japan

Location

Kindai University Nara Hospital

Ikoma, 630-0293, Japan

Location

Kurume University Hospital

Kurume, 830-0011, Japan

Location

Saitama Childrens Medical Center

Saitama Shi, 330-8777, Japan

Location

Miyagi Children's Hospital

Sendai, 989-3126, Japan

Location

National Center for Child Health and Development

Setagaya Ku, 157 8535, Japan

Location

Jichi Medical University Hospital

Shimotsuke, 329-0498, Japan

Location

Mie University Hospital

Tsu, 514 8507, Japan

Location

Szpital im. M. Kopernika

Gdansk, 80 803, Poland

Location

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30 663, Poland

Location

Korczowski Bartosz Gabinet Lekarski

Rzeszów, 35-302, Poland

Location

GASTROMED Sp. z o.o.

Torun, 87 100, Poland

Location

WIP Warsaw IBD Point Profesor Kierkus

Warsaw, 04 501, Poland

Location

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, 04 730, Poland

Location

Kazan State Medical University

Kazan', 420138, Russia

Location

Russian National Research Medical University named after N.I.Pirogov

Moscow, 119571, Russia

Location

FSBI 'Scientific Centre of Children Health' of the Russian Academy of Medical Sciences

Moscow, 119991, Russia

Location

Privolzhsky Research Medical University of Ministry of Health of Russian Federation

Nizhny Novgorod, 603950, Russia

Location

Saratov State Medical University

Saratov, 410054, Russia

Location

Yaroslavl Regional Children's Clinical Hospital

Yaroslavl, 150032, Russia

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8BJ, United Kingdom

Location

Cambridge University Hospitals NHS Foundation Trust

Cambridge, CB2 0QQ, United Kingdom

Location

Royal London Hospital

London, E1 2AT, United Kingdom

Location

MeSH Terms

Conditions

Colitis, Ulcerative

Interventions

Weights and Measures; Ustekinumab

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Inflammatory Bowel Diseases; Colonic Diseases; Intestinal Diseases

Intervention Hierarchy (Ancestors)

Investigative Techniques; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2020
• First Posted: November 16, 2020
• Study Start: March 17, 2021
• Primary Completion: May 8, 2025
• Study Completion: June 5, 2025
• Last Updated: May 14, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

BE (5); RU (6); US (9); DE (8); PL (6); GB (4); IL (6); HU (5); JP (9)