Clinical trial NCT04630028: A Study of Ustekinumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis (UC)
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Colitis, Ulcerative
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2020-11-16
Enrollment: 112
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Global: Number of Participants with Clinical Remission at Induction Week 8 (I-8) Visit
timeFrame: Week 8
measure: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
timeFrame: Up to 74 weeks
measure: Number of Participants with Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability
timeFrame: Up to 74 weeks
measure: Number of Participants with AEs Leading to Discontinuation of Study Intervention
timeFrame: Up to 74 weeks
measure: Number of Participants with AEs of Special Interest (AESI) as a Measure of Safety and Tolerability
timeFrame: Up to 74 weeks
measure: Number of Participants with Laboratory Abnormalities
timeFrame: Up to 74 weeks
measure: Reactions Temporally Associated with an Intravenous (IV) Infusion and Subcutaneous (SC) Injection-site Reactions
timeFrame: Up to 74 weeks
measure: Serum Concentration of Ustekinumab
timeFrame: Up to 74 weeks
measure: US Specific: Clinical Remission at M-44 for Participants who are in Clinical Response at I-8
timeFrame: Week 52
Secondary outcomes:
measure: Number of Participants With Clinical Response at I-8 Visit
timeFrame: Week 8
measure: Number of Participants with Symptomatic Remission at I-8 Visit
timeFrame: Week 8
measure: Clinical Remission at I-8 as Assessed by the Pediatric Ulcerative Colitis Activity Index Score (PUCAI) Score
timeFrame: Week 8
measure: Endoscopic Improvement at I-8 Visit
timeFrame: Week 8
measure: Histologic-endoscopic Mucosal Improvement at Week I-8
timeFrame: Week 8
measure: Number of Participants with Clinical Remission at Week 44 (M-44) Visit
timeFrame: Week 52
measure: Number of Participants with Symptomatic Remission at M-44 Visit
timeFrame: Week 52
measure: Clinical Remission at M-44 as Assessed by the PUCAI Score
timeFrame: Week 52
measure: Endoscopic Improvement at M-44 Visit
timeFrame: Week 52
measure: Corticosteroid-free Clinical Remission at Week M-44
timeFrame: Week 52
measure: 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
timeFrame: Week 52
measure: Clinical Remission at M-44 for Participants who are in Clinical Remission at I-8
timeFrame: Week 52
measure: US Specific: Number of Participants with Clinical Remission at I-8 Visit
timeFrame: Week 8
measure: Histologic-endoscopic Mucosal Improvement at Week M-44
timeFrame: Week 52
Other outcomes:
Locations:
city: Wilmington
country: United States
facility: Nemours DuPont Hospital for Children
state: Delaware
city: Atlanta
country: United States
facility: Children's Center for Digestive Health Care
state: Georgia
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Morristown
country: United States
facility: Morristown Memorial Hospital
state: New Jersey
city: Charlotte
country: United States
facility: Levine Childrens at Atrium Health
state: North Carolina
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Hershey
country: United States
facility: Penn State Hershey Children's Hospital
state: Pennsylvania
city: Fort Worth
country: United States
facility: Cook Childrens Medical Center
state: Texas
city: Fairfax
country: United States
facility: Pediatric Specialists Of Virginia
state: Virginia
city: Brussel
country: Belgium
facility: Universitair Kinderziekenhuis Koningin Fabiola
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint Luc
city: Gent
country: Belgium
facility: UZ Gent
city: Jette
country: Belgium
facility: UZ Brussel
city: Leuven
country: Belgium
facility: UZ Leuven
city: Aachen
country: Germany
facility: Universitätsklinikum Aachen
city: Berlin
country: Germany
facility: Charite-Universitätsmedizin Berlin - Berlin
city: Erlangen
country: Germany
facility: Universitatsklinikum Erlangen
city: Essen
country: Germany
facility: Universitatsklinikum Essen
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Munich
country: Germany
facility: Dr. von Haunersches Kinderspital
city: Regensburg
country: Germany
facility: KUNO Klinik St. Hedwig
city: Ulm
country: Germany
facility: Universitatsklinikum Ulm
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Miskolc
country: Hungary
facility: Borsod Abauj Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktato Korhaz
city: Nyiregyhaza
country: Hungary
facility: Szabolcs Szatmar Bereg Varmegyei Oktatokorhaz
city: Szeged
country: Hungary
facility: Szegedi Tudományegyetem, Gyermekgyógyászati Klinika és Gyermekegészségügyi Centrum
city: Be Er Ya Akov
country: Israel
facility: Shamir Medical Center Assaf Harofeh
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Haifa
country: Israel
facility: Carmel Medical Center
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Petah Tikva
country: Israel
facility: Schneider Children's Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Bunkyo Ku
country: Japan
facility: Juntendo University Hospital
city: Gunma
country: Japan
facility: Gunma University Hospital
city: Ikoma
country: Japan
facility: Kindai University Nara Hospital
city: Kurume
country: Japan
facility: Kurume University Hospital
city: Saitama shi
country: Japan
facility: Saitama Childrens Medical Center
city: Sendai
country: Japan
facility: Miyagi Children's Hospital
city: Setagaya Ku
country: Japan
facility: National Center for Child Health and Development
city: Shimotsuke
country: Japan
facility: Jichi Medical University Hospital
city: Tsu
country: Japan
facility: Mie University Hospital
city: Gdansk
country: Poland
facility: Szpital im. M. Kopernika
city: Krakow
country: Poland
facility: Uniwersytecki Szpital Dzieciecy w Krakowie
city: Rzeszow
country: Poland
facility: Korczowski Bartosz Gabinet Lekarski
city: Torun
country: Poland
facility: GASTROMED Sp. z o.o.
city: Warszawa
country: Poland
facility: WIP Warsaw IBD Point Profesor Kierkus
city: Warszawa
country: Poland
facility: Instytut Pomnik Centrum Zdrowia Dziecka
city: Kazan
country: Russian Federation
facility: Kazan State Medical University
city: Moscow
country: Russian Federation
facility: Russian National Research Medical University named after N.I.Pirogov
city: Moscow
country: Russian Federation
facility: FSBI 'Scientific Centre of Children Health' of the Russian Academy of Medical Sciences
city: Nizhny Novgorod
country: Russian Federation
facility: Privolzhsky Research Medical University of Ministry of Health of Russian Federation
city: Saratov
country: Russian Federation
facility: Saratov State Medical University
city: Yaroslavl
country: Russian Federation
facility: Yaroslavl Regional Children's Clinical Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: Royal London Hospital
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: