Crohn DiseasePhase 3UNITI JrNCT04673357
A Study of Ustekinumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease
A Phase 3 study testing Ustekinumab in people with Crohn Disease, run by Janssen Research & Development, LLC at 53 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate the efficacy of ustekinumab dosing in inducing clinical remission (Global) and in maintaining clinical remission (US); to evaluate the safety profile and ustekinumab exposure (pharmacokinetics [PK]) in pediatric participants with moderately to severely active Crohn's disease. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Crohn Disease
- What is being tested?
- Ustekinumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 101 participants
- Where
- 53 sitesin 9 countries
- Study dates
- 6 Apr 2021to 3 Mar 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Have Crohn's disease or fistulizing Crohn's disease with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by endoscopy and histology
- Must have moderately to severely active Crohn's disease (as defined by a baseline Pediatric Crohn's Disease Activity Index [PCDAI] score greater than [>] 30); have ileocolonoscopy with evidence of active Crohn's disease defined as presence of ulceration (which is equal to Simple Endoscopic Score for Crohn's disease [SES-CD] score greater than or equals to [>=] 3) during screening into this study. The ileocolonoscopy procedure must occur within approximately 3 weeks prior to the administration of study intervention at Week 0 (Induction Period). A video ileocolonoscopy recorded within 3 months prior to the Week 0 (Induction Period) visit may be used in case of rescreening of a participant who had an ileocolonoscopy but failed the initial screening for another reason, on a case-by-case basis, after consultation with the sponsor. If unable to evaluate ulceration due to stricture or inadequate bowel preparation, at least one of the following criteria may instead be applied: an abnormal C-reactive protein (CRP) (> 0.3 milligram per deciliter [mg/dL] or 3.0 milligram per liter [mg/L] at screening) or; fecal calprotectin of >= 250 milligram per kilogram [mg/kg] or >= 250 microgram per gram [mcg/g] at screening
- If receiving enteral nutrition, must have been on a stable regimen for at least 2 weeks prior to induction week 0 (Week I-0)
- 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
Who cannot join
- Has complications of Crohn's disease such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery, that could preclude the use of the PCDAI to assess response to therapy or would possibly confound the ability to assess the effect of treatment with ustekinumab
- 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
- Have a history of moderate or severe progressive or uncontrolled liver or renal insufficiency; or significant cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, psychiatric (including suicidality), or metabolic disturbances
- Received an investigational intervention including any investigational vaccines or used an invasive investigational medical device within 3 months before the planned first dose of study intervention or is currently enrolled in an investigational study; receipt of an investigational vaccine for Coronavirus Disease 2019 (COVID-19) is not an automatic exclusion criterion
Trial locations and contacts
53 sites in 9 countries.
53 sites in 9 countries
United States9 sites
Children's Center for Digestive Health Care
Atlanta, Georgia, 30342
Levine Childrens at Atrium Health
Charlotte, North Carolina, 28207
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Pediatric Specialists Of Virginia
Fairfax, Virginia, 22031
Cook Childrens Medical Center
Fort Worth, Texas, 76104
Penn State Hershey Children's Hospital
Hershey, Pennsylvania, 17033
Morristown Memorial Hospital
Morristown, New Jersey, 07962
Mayo Clinic
Rochester, Minnesota, 55905
Nemours DuPont Hospital for Children
Wilmington, Delaware, 19803
Japan9 sites
Juntendo University Hospital
Bunkyō City, 113 8431
Gunma University Hospital
Gunma, 371-0034
Kindai University Nara Hospital
Ikoma, 630-0293
Kurume University Hospital
Kurume, 830-0011
Saitama Childrens Medical Center
Saitama Shi, 330-8777
Miyagi Children's Hospital
Sendai, 989-3126
National Center for Child Health and Development
Setagaya Ku, 157 8535
Jichi Medical University Hospital
Shimotsuke, 329-0498
Mie University Hospital
Tsu, 514 8507
Germany7 sites
Universitätsklinikum Aachen
Aachen, 52074
Charite-Universitätsmedizin Berlin - Berlin
Berlin, 13353
Universitatsklinikum Essen
Essen, 45147
Medizinische Hochschule Hannover
Hanover, 30625
Dr. von Haunersches Kinderspital
Munich, 80337
KUNO Klinik St. Hedwig
Regensburg, 93049
Universitatsklinikum Ulm
Ulm, 89075
Belgium5 sites
Universitair Kinderziekenhuis Koningin Fabiola
Brussels, 1020
Cliniques Universitaires Saint Luc
Brussels, 1200
UZ Gent
Ghent, 9000
UZ Brussel
Jette, 1090
UZ Leuven
Leuven, 3000
Hungary5 sites
Semmelweis Egyetem
Budapest, 1083
Debreceni Egyetem Klinikai Kozpont
Debrecen, 4032
Borsod Abauj Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktato Korhaz
Miskolc, 3526
Szabolcs Szatmar Bereg Varmegyei Oktatokorhaz
Nyíregyháza, 4400
Szegedi Tudományegyetem, Gyermekgyógyászati Klinika és Gyermekegészségügyi Centrum
Szeged, 6720
Poland5 sites
Szpital im. M. Kopernika
Gdansk, 80 803
Uniwersytecki Szpital Dzieciecy w Krakowie
Krakow, 30 663
Korczowski Bartosz Gabinet Lekarski
Rzeszów, 35-302
WIP Warsaw IBD Point Profesor Kierkus
Warsaw, 04 501
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04 730
United Kingdom5 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, BS2 8BJ
Cambridge University Hospitals NHS Foundation Trust
Cambridge, CB2 0QQ
Royal Hospital for Children and Young People
Edinburgh, EH16 4TJ
Royal London Hospital
London, E1 2AT
Israel4 sites
Yitzhak Shamir Medical Center
Be’er Ya‘aqov, 70300
Carmel Medical Center
Haifa, 34362
Shaare Zedek Medical Center
Jerusalem, 9103102
Schneider Children's Medical Center
Petah Tikva, 4920235
Russia4 sites
Kazan State Medical University
Kazan', 420138
Russian National Research Medical University named after N.I.Pirogov
Moscow, 119571
Privolzhsky Research Medical University of Ministry of Health of Russian Federation
Nizhny Novgorod, 603950
Yaroslavl Regional Children's Clinical Hospital
Yaroslavl, 150032
About this study
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 Crohn's Disease
The purpose of this study is to evaluate the efficacy of ustekinumab dosing in inducing clinical remission (Global) and in maintaining clinical remission (US); to evaluate the safety profile and ustekinumab exposure (pharmacokinetics [PK]) in pediatric participants with moderately to severely active Crohn's disease.
- Conditions
- Crohn Disease
- Being tested
- Ustekinumab, Placebo
- Keywords
- Pediatric
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UstekinumabDrug
Ustekinumab will be administered intravenously in induction period and subcutaneously in maintenance period.
PlaceboDrug
Matching placebo will be administered as SC injection.
| Study group | Role | What they receive |
|---|---|---|
| Open- Label Ustekinumab Intravenous (IV): Induction Period | Experimental | All participants will receive a single 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]). Receives: Drug: Ustekinumab |
| Ustekinumab Subcutaneous (SC) Every 8 Weeks (q8w): Maintenance Period | Experimental | Participants will receive SC administration of ustekinumab q8w based on BSA (mg/m^2) or weight-tiered induction dose (mg/kg) at maintenance weeks (Weeks M)-0, M-8, M-16, M-24, M 32, and M-40 and matching placebo at Weeks M-12 and M-36 to maintain the blind. Receives: Drug: Ustekinumab; Drug: Placebo |
| Ustekinumab SC Every 12 Weeks (q12w): Maintenance Period | Experimental | Participants will receive SC administration of ustekinumab 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. Receives: Drug: Ustekinumab; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Global Outcome Measure: Percentage of Participants With Clinical Remission at Induction Week 8 (Week I-8)
Clinical remission was defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score <=10. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency, and general well-being); laboratory scores (3 items: hematocrit [HCT], erythrocyte sedimentation rate [ESR], Albumin); growth scores (2 items: weight and height); physical examination scores (2 items: abdomen and perirectal disease); and extraintestinal manifestations. The category of severity for each index item is assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score =5. For albumin level, the maximum score = 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
Measured: Induction Week 8 (Week I-8)
US-specific Outcome Measure: Percentage of Participants With Clinical Remission at Maintenance Week 44
Clinical remission was defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score <=10. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency, and general well-being); laboratory scores (3 items: HCT, ESR, Albumin); growth scores (2 items: weight and height); physical examination scores (2 items: abdomen and perirectal disease); and extraintestinal manifestations. The category of severity for each index item was assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score= 5. For albumin level, the maximum score= 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
Measured: Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Number of participants with TEAEs were reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. TEAEs were defined as AEs occurring at or after the initial administration of the study intervention.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
Number of participants with TESAEs was reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; or congenital anomaly. TESAEs were defined as any SAE occurring at or after the initial administration of the study intervention.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With AEs Leading to Discontinuation of Study Intervention
Number of participants with AEs leading to discontinuation of study intervention were reported.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 40 (Study Week 48)
Global and US-specific Outcome Measures: Number of Participants With AEs of Special Interest (AESI)
An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. AESI were defined as any newly identified malignancy, or case of active TB, or opportunistic infection occurring after the first administration of study intervention.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With Abnormalities in Clinical Laboratory Parameters: Hematology
Number of participants with abnormalities in clinical laboratory parameters (hematology) were reported. It included hemoglobin (Hb). Grades 0-4 were based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), where Grade 0 was normal, Grade 1 was mild, Grade 2 was moderate, Grade 3 was severe or medically significant but not immediately life-threatening, and Grade 4 was life-threatening consequences. Higher grades showed more severe abnormalities. Only abnormalities where atleast one participant had data are reported. Inc = increased, Dec= decreased.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With Abnormalities in Clinical Laboratory Parameters: Chemistry
Number of participants with abnormalities in clinical laboratory parameters were reported. It included chemistry measures: alanine aminotransferase (ALT), aspartate aminotransferase (AST),blood bilirubin (BB). Grades 0-4 were based on NCI-CTCAE version 5.0, where Grade 0 was Normal, Grade 1 was mild, Grade 2 was moderate, Grade 3 was severe or medically significant but not immediately life-threatening, and Grade 4 was life-threatening consequences. Higher grades showed more severe abnormalities. Only abnormalities where at least one participant had data are reported. Inc = increased, Dec= decreased.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Number of Participants With Reactions Temporally Associated With Intravenous (IV) Infusion (Induction Period) and Subcutaneous (SC) Injection-Site Reactions (Maintenance Period)
Number of participants with reactions temporally associated with intravenous (IV) infusion (Induction Period) and subcutaneous (SC) Injection-Site Reactions (Maintenance Period) were reported.
Measured: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Global and US-specific Outcome Measures: Serum Ustekinumab Concentrations
Serum ustekinumab concentrations were reported.
Measured: Induction Period: Week 0 (Pre-infusion), Week I-0 (1-hour post infusion), Week I-3, Week I-6, and Week I-8; Maintenance Period: Week M-4, Week M-8, Week M-12, Week M-16, Week M-24, Week M-32, Week M-36 and Week M-44
Secondary outcomes (9)
Global Outcome Measure: Percentage of Participants With Clinical Remission at Induction Week 6 as Assessed by Short Pediatric Crohn's Disease Activity Index (sPCDAI)
Clinical remission was defined as a short Pediatric Crohn's Disease Activity Index (sPCDAI) score <=10. The sPCDAI was composed of six components: abdominal pain, stool frequency, patient general well-being, body weight, abdominal examination (abdominal mass and tenderness), and extraintestinal manifestations. Each component was assigned a score of 0, 5, 10, or 20, depending on the severity of findings, with higher scores indicating greater disease activity, while lower scores (closer to 0) reflected clinical remission or minimal disease activity. Individual component scores were summed to derive the total sPCDAI score, which ranged from 0 to 90. Higher score indicate greater severity. The sPCDAI score was calculated only when >=3 of the 6 components were available.
Measured: Induction Week 6 (Week I-6)
Global Outcome Measure: Percentage of Participants With Clinical Response at Induction Week 8
Assessment of response in children with Crohn's disease was evaluated using the PCDAI. Clinical response was defined as a reduction from baseline of >=12.5 points in the PCDAI score, calculated by summing weighted scores from 11 items across five domains: symptom history (abdominal pain, stool frequency, and general well-being); laboratory scores (hematocrit, erythrocyte sedimentation rate, and albumin); growth scores (weight and height); physical examination scores (abdominal and perirectal disease); and extraintestinal manifestations. Each index item was assigned a severity score of 0 (normal), 5 (mild abnormality), or 10 (severe abnormality). For HCT and ESR, maximum score was 5, and for albumin level, maximum score was 10. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity. Lower PCDAI scores reflected lower disease activity, with a resulting score of <=30 on the 0-100 scale.
Measured: Induction Week 8
Global and US-specific Outcome Measures: Percentage of Participants With Clinical Response at Induction Week 6 as Assessed by sPCDAI
sPCDAI clinical response was defined as reduction from baseline in the sPCDAI score of >=10. The 6-item sPCDAI score ranges from 0 (no disease activity) to 90 (severe disease activity),calculated by summing weighted scores for abdominal pain, stool frequency, general well-being, body weight, abdominal exam, and extraintestinal manifestations. Each component was assigned a score of 0, 5, 10, or 20, depending on the severity of findings, with higher scores indicating greater disease activity, while lower scores (closer to 0) reflected minimal disease activity. Individual component scores were summed to derive the total sPCDAI score, which ranged from 0 to 90. Higher score indicate greater severity. The sPCDAI score was only calculated when >=3 of the 6 components were available.
Measured: Induction Week 6
Global Outcome Measure: Percentage of Participants With Endoscopic Response at Maintenance Week 8 as Assessed by Simplified Endoscopic Score-Crohn's Disease (SES-CD)
Endoscopic response was assessed using the Simplified Endoscopic Activity Score for Crohn's Disease (SES-CD), a validated endoscopic scoring system based on four components: size of ulcers, extent of ulcerated surface, extent of affected surface, and presence of narrowing. Each component was scored from 0 to 3 across five intestinal sections: ileum, right colon, transverse colon, left (descending and sigmoid) colon, and rectum. Component scores were summed across all sections to derive a total SES-CD score ranging from 0 to 56, with higher scores indicating greater endoscopic disease severity. Endoscopic response was defined as a >=50% reduction from baseline in SES-CD score or achievement of an SES-CD score <=2 in participants with a baseline SES-CD score >=3.
Measured: Maintenance Period Week 8 (Study Week 16)
Global Outcome Measure: Percentage of Participants With Clinical Response at Maintenance Week 8
Clinical response was defined as a reduction from baseline of >=12.5 points in PCDAI score, calculated by summing weighted scores from 11 items across five domains: symptom history(abdominal pain, stool frequency, and general well-being); laboratory scores (hematocrit, erythrocyte sedimentation rate, and albumin); growth scores(weight and height); physical examination scores(abdominal and perirectal disease); extraintestinal manifestations. Each index item was assigned a severity score of 0(normal), 5(mild abnormality), or 10(severe abnormality). For HCT and ESR, maximum score was 5, for albumin level, maximum score was 10. Total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity. Lower PCDAI scores reflected lower disease activity, with resulting score of <=30 on 0-100 scale.
Measured: Maintenance Week 8 (Study Week 16)
Global and US-specific Outcome Measures: Percentage of Participants With Endoscopic Response at Maintenance Week 44 as Assessed by SES-CD
Endoscopic response was assessed using the Simplified Endoscopic Activity Score for Crohn's Disease (SES-CD), a validated endoscopic scoring system based on four components: size of ulcers, extent of ulcerated surface, extent of affected surface, and presence of narrowing. Each component was scored from 0 to 3 across five intestinal sections: ileum, right colon, transverse colon, left (descending and sigmoid) colon, and rectum. Component scores were summed across all sections to derive a total SES-CD score ranging from 0 to 56, with higher scores indicating greater endoscopic disease severity. Endoscopic response was defined as a >=50% reduction from baseline in SES-CD score or achievement of an SES-CD score <=2 in participants with a baseline SES-CD score >=3.
Measured: Maintenance Week 44 (Study Week 52)
US-specific Outcome Measure: Percentage of Participants With Clinical Response at Maintenance Week 44
Clinical response was defined as a reduction from baseline of >=12.5 points in PCDAI score, calculated by summing weighted scores from 11 items across five domains: symptom history(abdominal pain, stool frequency, and general well-being); laboratory scores (hematocrit, erythrocyte sedimentation rate, and albumin); growth scores(weight and height); physical examination scores(abdominal and perirectal disease); extraintestinal manifestations. Each index item was assigned a severity score of 0(normal), 5(mild abnormality), or 10(severe abnormality). For HCT and ESR, maximum score was 5, for albumin level, maximum score was 10. Total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity. Lower PCDAI scores reflected lower disease activity, with resulting score of <=30 on 0-100 scale.
Measured: Maintenance Week 44 (Study Week 52)
US-specific Outcome Measure: Percentage of Participants With Corticosteroid-free Clinical Remission at Maintenance Week 44
Corticosteroid-free remission was defined as PCDAI score of <=10 points and not receiving corticosteroids for at least 90 days prior to Week M-44. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency; and general well-being); laboratory scores (3 items: HCT, ESR, Albumin); growth scores (2 items: weight and height), physical examination scores (2 items: abdomen and perirectal disease), and extraintestinal manifestations. The category of severity for each index item was assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score= 5. For albumin level, the maximum score= 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
Measured: Maintenance Period Week 44 (Study Week 52)
Global Outcome Measure: Percentage of Participants With Clinical Remission at Maintenance Week 44 Who Had Clinical Remission at Induction Week 8
Clinical remission was defined as Pediatric Crohn's Disease Activity Index (PCDAI) score <=10. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency; and general well-being); laboratory scores (3 items: HCT, ESR, Albumin); growth scores (2 items: weight and height), physical examination scores (2 items: abdomen and perirectal disease), and extraintestinal manifestations. The category of severity for each index item was assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score= 5. For albumin level, the maximum score= 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
Measured: Maintenance Week 44 (Study Week 52)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2020
- Study start6 Apr 2021
- Primary completion28 Nov 2024
- Study completion3 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Crohn DiseaseStudies of the same condition, open and under way.
- More trials run by Janssen Research & Development, LLCThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- Janssen Research & Development, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Janssen Research & Development, LLC Clinical Trial, Study director, Janssen Research & Development, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04673357
- Sponsor's protocol ID
- CR108864
- Other IDs
- CNTO1275CRD3004, 2019-004225-24, 2023-504978-38-00, 2023, 2019
- Acronym
- UNITI Jr
- Status last verified
- Jul 2026
- First submitted
- 14 Dec 2020
- Last updated
- 30 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
De Greef E, Turner D, Kierkus J, Korczowski B, Meglicka M, Cohen SA, Hyams JS, Griffiths AM, Rosh JR, Strauss R, Van Limbergen E, Adedokun OJ, Kim L, Volger S; UNITI Jr study Group. Ustekinumab therapy for moderately to severely active pediatric Crohn's disease: UNITI Jr study safety and efficacy results in patients weighing at least 40 kg. J Crohns Colitis. 2026 Mar 10;20(3):jjag011. doi: 10.1093/ecco-jcc/jjag011.
PubMed 41843763
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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 Crohn's Disease
- ClinicalTrials.gov ID
- NCT04673357
- Acronym / sponsor ID
- UNITI Jr / CR108864
- Conditions
- Crohn Disease
- Keywords
- Pediatric
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- Janssen Research & Development, LLCIndustry (pharma, biotech, devices)
- Study start
- 6 Apr 2021 (actual)
- Primary completion
- 28 Nov 2024 (actual)
- Study completion
- 3 Mar 2025 (actual)
- First posted
- 17 Dec 2020 (actual)
- Results
- Posted 30 Jul 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy of ustekinumab dosing in inducing clinical remission (Global) and in maintaining clinical remission (US); to evaluate the safety profile and ustekinumab exposure (pharmacokinetics [PK]) in pediatric participants with moderately to severely active Crohn's disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Open- Label Ustekinumab Intravenous (IV): Induction Period | Experimental | Drug: UstekinumabAll participants will receive a single 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]). |
| Ustekinumab Subcutaneous (SC) Every 8 Weeks (q8w): Maintenance Period | Experimental | Drug: Ustekinumab, Drug: PlaceboParticipants will receive SC administration of ustekinumab q8w based on BSA (mg/m^2) or weight-tiered induction dose (mg/kg) at maintenance weeks (Weeks M)-0, M-8, M-16, M-24, M 32, and M-40 and matching placebo at Weeks M-12 and M-36 to maintain the blind. |
| Ustekinumab SC Every 12 Weeks (q12w): Maintenance Period | Experimental | Drug: Ustekinumab, Drug: PlaceboParticipants will receive SC administration of ustekinumab 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. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Have Crohn's disease or fistulizing Crohn's disease with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by endoscopy and histology
- Must have moderately to severely active Crohn's disease (as defined by a baseline Pediatric Crohn's Disease Activity Index [PCDAI] score greater than [>] 30); have ileocolonoscopy with evidence of active Crohn's disease defined as presence of ulceration (which is equal to Simple Endoscopic Score for Crohn's disease [SES-CD] score greater than or equals to [>=] 3) during screening into this study. The ileocolonoscopy procedure must occur within approximately 3 weeks prior to the administration of study intervention at Week 0 (Induction Period). A video ileocolonoscopy recorded within 3 months prior to the Week 0 (Induction Period) visit may be used in case of rescreening of a participant who had an ileocolonoscopy but failed the initial screening for another reason, on a case-by-case basis, after consultation with the sponsor. If unable to evaluate ulceration due to stricture or inadequate bowel preparation, at least one of the following criteria may instead be applied: an abnormal C-reactive protein (CRP) (> 0.3 milligram per deciliter [mg/dL] or 3.0 milligram per liter [mg/L] at screening) or; fecal calprotectin of >= 250 milligram per kilogram [mg/kg] or >= 250 microgram per gram [mcg/g] at screening
- If receiving enteral nutrition, must have been on a stable regimen for at least 2 weeks prior to induction week 0 (Week I-0)
- 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
Key exclusion criteria (5 listed)
- Has complications of Crohn's disease such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery, that could preclude the use of the PCDAI to assess response to therapy or would possibly confound the ability to assess the effect of treatment with ustekinumab
- 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
- Have a history of moderate or severe progressive or uncontrolled liver or renal insufficiency; or significant cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, psychiatric (including suicidality), or metabolic disturbances
- Received an investigational intervention including any investigational vaccines or used an invasive investigational medical device within 3 months before the planned first dose of study intervention or is currently enrolled in an investigational study; receipt of an investigational vaccine for Coronavirus Disease 2019 (COVID-19) is not an automatic exclusion criterion
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UstekinumabUstekinumab will be administered intravenously in induction period and subcutaneously in maintenance period. | Drug | Open- Label Ustekinumab Intravenous (IV): Induction Period, Ustekinumab SC Every 12 Weeks (q12w): Maintenance Period, Ustekinumab Subcutaneous (SC) Every 8 Weeks (q8w): Maintenance Period |
| PlaceboMatching placebo will be administered as SC injection. | Drug | Ustekinumab SC Every 12 Weeks (q12w): Maintenance Period, Ustekinumab Subcutaneous (SC) Every 8 Weeks (q8w): Maintenance Period |
Primary outcome
Global Outcome Measure: Percentage of Participants With Clinical Remission at Induction Week 8 (Week I-8)
Time frame: Induction Week 8 (Week I-8)
Clinical remission was defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score <=10. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency, and general well-being); laboratory scores (3 items: hematocrit [HCT], erythrocyte sedimentation rate [ESR], Albumin); growth scores (2 items: weight and height); physical examination scores (2 items: abdomen and perirectal disease); and extraintestinal manifestations. The category of severity for each index item is assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score =5. For albumin level, the maximum score = 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
US-specific Outcome Measure: Percentage of Participants With Clinical Remission at Maintenance Week 44
Time frame: Maintenance Week 44 (Study Week 52)
Clinical remission was defined as a Pediatric Crohn's Disease Activity Index (PCDAI) score <=10. The 11-item PCDAI covered five general disease activity domains: symptom history scores (3 items: abdominal pain, stool frequency, and general well-being); laboratory scores (3 items: HCT, ESR, Albumin); growth scores (2 items: weight and height); physical examination scores (2 items: abdomen and perirectal disease); and extraintestinal manifestations. The category of severity for each index item was assigned a score: 0=normal;5=mild abnormality; 10= severe abnormality. For hematocrit and erythrocyte SR, the maximum score= 5. For albumin level, the maximum score= 10. All items were summed to derive the total score. The total PCDAI score ranged from 0 to 100, with higher scores indicating greater disease activity.
Global and US-specific Outcome Measures: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Number of participants with TEAEs were reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. TEAEs were defined as AEs occurring at or after the initial administration of the study intervention.
Global and US-specific Outcome Measures: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Number of participants with TESAEs was reported. An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; or congenital anomaly. TESAEs were defined as any SAE occurring at or after the initial administration of the study intervention.
Global and US-specific Outcome Measures: Number of Participants With AEs Leading to Discontinuation of Study Intervention
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 40 (Study Week 48)
Number of participants with AEs leading to discontinuation of study intervention were reported.
Global and US-specific Outcome Measures: Number of Participants With AEs of Special Interest (AESI)
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
An AE was any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non-investigational) product. An AE did not necessarily have a causal relationship with the intervention. AESI were defined as any newly identified malignancy, or case of active TB, or opportunistic infection occurring after the first administration of study intervention.
Global and US-specific Outcome Measures: Number of Participants With Abnormalities in Clinical Laboratory Parameters: Hematology
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Number of participants with abnormalities in clinical laboratory parameters (hematology) were reported. It included hemoglobin (Hb). Grades 0-4 were based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), where Grade 0 was normal, Grade 1 was mild, Grade 2 was moderate, Grade 3 was severe or medically significant but not immediately life-threatening, and Grade 4 was life-threatening consequences. Higher grades showed more severe abnormalities. Only abnormalities where atleast one participant had data are reported. Inc = increased, Dec= decreased.
Global and US-specific Outcome Measures: Number of Participants With Abnormalities in Clinical Laboratory Parameters: Chemistry
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Number of participants with abnormalities in clinical laboratory parameters were reported. It included chemistry measures: alanine aminotransferase (ALT), aspartate aminotransferase (AST),blood bilirubin (BB). Grades 0-4 were based on NCI-CTCAE version 5.0, where Grade 0 was Normal, Grade 1 was mild, Grade 2 was moderate, Grade 3 was severe or medically significant but not immediately life-threatening, and Grade 4 was life-threatening consequences. Higher grades showed more severe abnormalities. Only abnormalities where at least one participant had data are reported. Inc = increased, Dec= decreased.
Global and US-specific Outcome Measures: Number of Participants With Reactions Temporally Associated With Intravenous (IV) Infusion (Induction Period) and Subcutaneous (SC) Injection-Site Reactions (Maintenance Period)
Time frame: Induction Period: From induction Week 0 up to induction Week 8; Maintenance Period: From Maintenance Week 0 (Study Week 8) up to Maintenance Week 44 (Study Week 52)
Number of participants with reactions temporally associated with intravenous (IV) infusion (Induction Period) and subcutaneous (SC) Injection-Site Reactions (Maintenance Period) were reported.
Global and US-specific Outcome Measures: Serum Ustekinumab Concentrations
Time frame: Induction Period: Week 0 (Pre-infusion), Week I-0 (1-hour post infusion), Week I-3, Week I-6, and Week I-8; Maintenance Period: Week M-4, Week M-8, Week M-12, Week M-16, Week M-24, Week M-32, Week M-36 and Week M-44
Serum ustekinumab concentrations were reported.
9 secondary outcomes
Global Outcome Measure: Percentage of Participants With Clinical Remission at Induction Week 6 as Assessed by Short Pediatric Crohn's Disease Activity Index (sPCDAI)
Time frame: Induction Week 6 (Week I-6)
Global Outcome Measure: Percentage of Participants With Clinical Response at Induction Week 8
Time frame: Induction Week 8
Global and US-specific Outcome Measures: Percentage of Participants With Clinical Response at Induction Week 6 as Assessed by sPCDAI
Time frame: Induction Week 6
Global Outcome Measure: Percentage of Participants With Endoscopic Response at Maintenance Week 8 as Assessed by Simplified Endoscopic Score-Crohn's Disease (SES-CD)
Time frame: Maintenance Period Week 8 (Study Week 16)
Global Outcome Measure: Percentage of Participants With Clinical Response at Maintenance Week 8
Time frame: Maintenance Week 8 (Study Week 16)
Global and US-specific Outcome Measures: Percentage of Participants With Endoscopic Response at Maintenance Week 44 as Assessed by SES-CD
Time frame: Maintenance Week 44 (Study Week 52)
US-specific Outcome Measure: Percentage of Participants With Clinical Response at Maintenance Week 44
Time frame: Maintenance Week 44 (Study Week 52)
US-specific Outcome Measure: Percentage of Participants With Corticosteroid-free Clinical Remission at Maintenance Week 44
Time frame: Maintenance Period Week 44 (Study Week 52)
Global Outcome Measure: Percentage of Participants With Clinical Remission at Maintenance Week 44 Who Had Clinical Remission at Induction Week 8
Time frame: Maintenance Week 44 (Study Week 52)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41843763De Greef E, Turner D, Kierkus J, Korczowski B, Meglicka M, Cohen SA, Hyams JS, Griffiths AM, Rosh JR, Strauss R, Van Limbergen E, Adedokun OJ, Kim L, Volger S; UNITI Jr study Group. Ustekinumab therapy for moderately to severely active pediatric Crohn's disease: UNITI Jr study safety and efficacy results in patients weighing at least 40 kg. J Crohns Colitis. 2026 Mar 10;20(3):jjag011. doi: 10.1093/ecco-jcc/jjag011.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Janssen Research & Development, LLC Clinical Trial study director
Janssen Research & Development, LLC
53 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ustekinumab in people with Crohn Disease, run by Janssen Research & Development, LLC at 53 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in United States, Japan, Germany, Belgium, Hungary and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jul 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Global Outcome Measure: Percentage of Participants With Clinical Remission at Induction Week 8 (Week I-8) (time frame: Induction Week 8 (Week I-8)). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04673357, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2026.