A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease (MACARONI-23)

A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23 Pathway to Establish Efficacy in Pediatric Crohn's Disease

The purpose of this study is to evaluate the clinical and endoscopic efficacy of guselkumab in pediatric participants with Crohn's Disease (CD) at the end of maintenance therapy (Week 52) among participants who were in clinical response to guselkumab at Week 12.

Study Overview

• Status: Recruiting
• Conditions: Crohn's Disease
• Intervention / Treatment: Drug: Guselkumab; Drug: Guselkumab

Detailed Description

Participants screened in the MACARONI-23 platform study could be randomized to guselkumab to participate in this intervention specific arm of the study.

• Study Type: Interventional
• Enrollment (Estimated): 120
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Study Contact; Phone Number: 844-434-4210; Email: Participate-In-This-Study1@its.jnj.com

Study Locations

• Australia
  • Nedlands, Australia, 6009
    • Recruiting
    • Perth Children's Hospital
  • South Brisbane, Australia, 4101
    • Recruiting
    • Mater Hospital Brisbane
  • Westmead, Australia, 2145
    • Recruiting
    • The Children's Hospital at Westmead
• Austria
  • Vienna, Austria, 1090
    • Recruiting
    • AKH - Medizinische Universitat Wien
• Belgium
  • Brussels, Belgium, 1090
    • Recruiting
    • Universitair Ziekenhuis Brussel
  • Brussels, Belgium, 1200
    • Recruiting
    • Cliniques Universitaires Saint Luc
  • Ghent, Belgium, 9000
    • Recruiting
    • Universitair Ziekenhuis Gent
  • Leuven, Belgium, 3000
    • Recruiting
    • UZ Leuven
• Brazil
  • Campinas, Brazil, 13060-904
    • Recruiting
    • Sociedade Campineira de Educacao e Instrucao Hospital e Maternidade Celso Pierro
  • Curitiba, Brazil, 80250-060
    • Recruiting
    • Associacao Hospitalar de Protecao a Infancia Dr. Raul Carneiro
  • Goiânia, Brazil, 74605-020
    • Recruiting
    • Universidade Federal de Goias - Hospital das Clinicas da UFG
  • Porto Alegre, Brazil, 90035-074
    • Recruiting
    • Irmandade Santa Casa de Misericordia de Porto Alegre
  • Porto Alegre, Brazil, 90035-007
    • Recruiting
    • Hospital de Clínicas de Porto Alegre
  • Vitória, Brazil, 29040-091
    • Recruiting
    • Centro de Ciencias e Saude da Universidade Federal do Espirito Santo-Nucleo de Doencas Infecciosas
  • Votuporanga, Brazil, 100000
    • Recruiting
    • Integral Pesquisa e Ensino
• Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3K 6R8
      • Recruiting
      • IWK Health Centre
  • Ontario
    • London, Ontario, Canada, N6A 5W9
      • Recruiting
      • London Health Sciences Centre Victoria Hospital
    • Toronto, Ontario, Canada, M5G 1X8
      • Recruiting
      • Hospital for Sick Children
• France
  • Amiens, France, 80054
    • Recruiting
    • CHU Amiens-Hopital Nord
  • Lille, France, 59037
    • Recruiting
    • Hôpital Jeanne de FLANDRE, CHRU LILLE
  • Paris, France, 75019
    • Recruiting
    • Hopital Robert Debre
  • Paris, France, 75015
    • Recruiting
    • Hopital Necker - Enfants Malades
• Israel
  • Beersheba, Israel, 84101
    • Recruiting
    • Soroka University Medical Center
  • Be’er Ya‘aqov, Israel, 70300
    • Recruiting
    • Shamir Medical Center Assaf Harofeh
  • Jerusalem, Israel, 9103102
    • Recruiting
    • Shaare Zedek Medical Center
  • Jerusalem, Israel, 9124001
    • Recruiting
    • Hadassah Medical Center
  • Petah Tikva, Israel, 4920235
    • Recruiting
    • Schneider Children's Medical Center
  • Rehovot, Israel, 761001
    • Recruiting
    • Kaplan Medical Center
• Italy
  • Alessandria, Italy, 15121
    • Recruiting
    • A.O. Santi Antonio e Biagio e Cesare Arrigo
  • Bergamo, Italy, 24127
    • Recruiting
    • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)
  • Bologna, Italy, 40124
    • Recruiting
    • Ospedale Bellaria, U.O.Cardiologia Az. USL di Bologna
  • Florence, Italy, 50139
    • Recruiting
    • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
  • Milan, Italy, 20154
    • Recruiting
    • Azienda Socio Sanitaria Territoriale Fatebenefratelli Presidio Ospedale dei Bambini Vittore Buzzi
  • Roma, Italy, 161
    • Recruiting
    • AOU Policlinico Umberto I
• Japan
  • Hirosaki, Japan, 036-8563
    • Recruiting
    • Hirosaki University Hospital
  • Kagoshima, Japan, 890-8544
    • Recruiting
    • Kagoshima University Hospital
  • Kashiwa-shi, Japan, 277-0871
    • Recruiting
    • Tsujinaka Hospital Kashiwanoha
  • Kobe, Japan, 650 0017
    • Recruiting
    • Kobe University Hospital
  • Saga, Japan, 849-8501
    • Recruiting
    • Saga University Hospital
  • Sendai, Japan, 989-3126
    • Recruiting
    • Miyagi Childrens Hospital
  • Setagaya Ku, Japan, 157 8535
    • Recruiting
    • National Center for Child Health and Development
  • Tokyo, Japan, 113-8431
    • Recruiting
    • Juntendo University Hospital
  • Tsu, Japan, 514 8507
    • Recruiting
    • Mie University Hospital
  • Ube, Japan, 755-8505
    • Recruiting
    • Yamaguchi University Hospital
  • Yokohama, Japan, 232-0024
    • Recruiting
    • Yokohama City University Medical Center
  • Yokohama, Japan, 230-8765
    • Recruiting
    • Saiseikai Yokohamashi Tobu Hospital
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Recruiting
    • Erasmus Medisch Centrum
• Norway
  • Nordbyhagen, Norway, 1474
    • Recruiting
    • Akershus Universitetssykehus HF
  • Oslo, Norway, 424
    • Recruiting
    • Oslo University Hospital
  • Tromsø, Norway, 9038
    • Recruiting
    • Universitetssykehuset Nord-Norge HF
  • Trondheim, Norway, 7030
    • Recruiting
    • St. Olavs hospital
• Poland
  • Rzeszów, Poland, 35-302
    • Recruiting
    • Korczowski Bartosz Gabinet Lekarski
  • Warsaw, Poland, 04 501
    • Recruiting
    • WIP Warsaw IBD Point Profesor Kierkus
  • Warsaw, Poland, 04 730
    • Recruiting
    • Instytut Pomnik Centrum Zdrowia
• Portugal
  • Braga, Portugal, 4710-243
    • Recruiting
    • Hospital de Braga
  • Lisbon, Portugal, 1649-035
    • Recruiting
    • Centro Hospitalar de Lisboa Norte Hospital Santa Maria
  • Porto, Portugal, 4200-319
    • Recruiting
    • Centro Hospitalar de Sao Joao E.P.E.
• South Korea
  • Busan, South Korea, 48108
    • Recruiting
    • Inje University Haeundae Paik Hospital
  • Daegu, South Korea, 41404
    • Recruiting
    • Kyungpook National University Chilgok Hospital
  • Seoul, South Korea, 03080
    • Recruiting
    • Seoul National University Hospital
  • Seoul, South Korea, 06351
    • Recruiting
    • Samsung Medical Center
  • Seoul, South Korea, 120-752
    • Recruiting
    • Severance Hospital Yonsei University Health System
• Spain
  • Córdoba, Spain, 14004
    • Recruiting
    • Hosp Reina Sofia
  • Sabadell, Spain, 8208
    • Recruiting
    • Corporacio Sanitari Parc Tauli
  • Valencia, Spain, 46026
    • Recruiting
    • Hosp. Univ. I Politecni La Fe
• United Kingdom
  • Glasgow, United Kingdom, G51 4TF
    • Recruiting
    • Royal Hospital for Sick Children
  • London, United Kingdom, WC1N 3JH
    • Recruiting
    • Great Ormond Street Hospital
  • Manchester, United Kingdom, M13 9WL
    • Recruiting
    • Royal Manchester Children's Hospital
  • Manchester, United Kingdom, M13 9WL
    • Recruiting
    • Nowgen Centre, Research and Innovation
  • Oxford, United Kingdom, OX3 9DU
    • Recruiting
    • John Radcliffe Hospital
  • Sheffield, United Kingdom, S10 2TH
    • Recruiting
    • Sheffield Children's Hospital
• United States
  • California
    • Los Angeles, California, United States, 90048
      • Recruiting
      • Cedars Sinai Medical Center
  • Connecticut
    • Hartford, Connecticut, United States, 06106
      • Recruiting
      • Connecticut Children's Medical Center
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Active, not recruiting
      • Emory University
    • Atlanta, Georgia, United States, 30342
      • Recruiting
      • Children's Center for Digestive Health Care
  • Indiana
    • Indianapolis, Indiana, United States, 46202-5225
      • Recruiting
      • Riley Hospital for Children
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Childrens Hospital
  • New Jersey
    • Morristown, New Jersey, United States, 07960
      • Recruiting
      • Goryeb Children's Hospital
  • New York
    • New York, New York, United States, 10029
      • Recruiting
      • Icahn School of Medicine at Mount Sinai
    • New York, New York, United States, 10032
      • Recruiting
      • Columbia University Medical Center
    • New York, New York, United States, 10021-5663
      • Recruiting
      • Weill Cornell Medical College - Judith Jaffe Multiple Sclerosis Center
  • Ohio
    • Dayton, Ohio, United States, 45404
      • Recruiting
      • The Children's Medical Center of Dayton
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Recruiting
      • Cook Childrens Medical Center
  • Vermont
    • Colchester, Vermont, United States, 05446
      • Recruiting
      • University of Vermont Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants must have a diagnosis of Crohn's Disease (CD) or fistulizing CD, with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by clinical, endoscopic, and histologic criteria.
• Participants must have moderately to severely active CD (as defined by a baseline Pediatric Crohn's Disease Activity Index [PCDAI] score greater than or equal to [>=] 30)
• Participants must have endoscopy with evidence of active CD defined as Simple Endoscopic Score for Crohn's Disease (SES-CD) score greater than or equal to (>=) 6 (or >=4 for participants with isolated ileal disease) within 1 month of receiving study intervention at Week 0
• Participants must have a history of inadequate response, loss of response, or intolerance to immunomodulators (6-MP, AZA, or MTX), oral or IV corticosteroids, or biologic therapy/JAK inhibitor therapy; OR have a history of corticosteroid dependence; OR have a history of inadequate response to exclusive enteral nutrition (EEN)

Exclusion Criteria:

• Participants has complications of CD such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery.
• Participants must not have an abscess
• Participants must not have any kind of bowel resection within 26 weeks or any other intra-abdominal surgery within 12 weeks of baseline

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open-label induction phase: Guselkumab Intravenously (IV); Participants will receive guselkumab dose IV based on their body weight during the 12-week open-label induction phase.	Drug: Guselkumab; Guselkumab will be administered subcutaneously.; Other Names: CNTO1959; TREMFYA; Drug: Guselkumab; Guselkumab will be administered intravenously.; Other Names: CNTO1959; TREMFYA
Experimental: Open-label induction phase: Guselkumab Subcutaneously (SC); Participants will receive guselkumab dose SC based on their body weight during the 12-week open-label induction phase.	Drug: Guselkumab; Guselkumab will be administered subcutaneously.; Other Names: CNTO1959; TREMFYA; Drug: Guselkumab; Guselkumab will be administered intravenously.; Other Names: CNTO1959; TREMFYA
Experimental: Double-blind maintenance phase: Guselkumab SC Dose Regimen 1; At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 1 SC based on their body weight up to Week 48.	Drug: Guselkumab; Guselkumab will be administered subcutaneously.; Other Names: CNTO1959; TREMFYA; Drug: Guselkumab; Guselkumab will be administered intravenously.; Other Names: CNTO1959; TREMFYA
Experimental: Double-blind Maintenance Phase: Guselkumab SC Dose Regimen 2; At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 2 SC based on their body weight up to Week 48.	Drug: Guselkumab; Guselkumab will be administered subcutaneously.; Other Names: CNTO1959; TREMFYA; Drug: Guselkumab; Guselkumab will be administered intravenously.; Other Names: CNTO1959; TREMFYA
Experimental: Open-label maintenance phase: Guselkumab SC; Week 12 non-responders will not be randomized and will enter an open-label maintenance phase to receive guselkumab SC dosing regimen based on their body weight up to Week 48.	Drug: Guselkumab; Guselkumab will be administered subcutaneously.; Other Names: CNTO1959; TREMFYA; Drug: Guselkumab; Guselkumab will be administered intravenously.; Other Names: CNTO1959; TREMFYA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants with Clinical Remission at Week 52	Percentage of participants with clinical remission at Week 52 will be assessed. Clinical remission is defined as pediatric Crohn's Disease activity index (PCDAI) less than or equal to (<=) 10.	Week 52
Percentage of Participants Who Achieve Endoscopic Response at Week 52	Percentage of participants who achieve endoscopic response at Week 52 will be assessed. Endoscopic response is defined as greater than or equal to (>=) 50 percent (%) reduction (global) and greater than (>) 50% reduction (U.S specific) from simplified endoscopic score-Crohn's Disease (SES-CD) score at baseline.	Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants with Clinical Remission at Week 12	Percentage of participants with clinical remission at Week 12 will be assessed. Clinical remission is defined as PCDAI score <=10.	Week 12
Percentage of Participants with Endoscopic Remission at Week 52	Percentage of participants with endoscopic remission at Week 52 will be assessed. Endoscopic remission is defined as SES-CD total score <=4 and at least a 2-point reduction from baseline and no subscore >1.	Week 52
Percentage of Participants with Corticosteroid-free Remission at Week 52	Percentage of participants with corticosteroid-free remission at Week 52 will be assessed. Corticosteroid-free remission is defined as PCDAI score <=10 at Week 52 and not receiving corticosteroids for at least 90 days before Week 52.	Week 52
Percentage of Participants with Sustained Clinical Remission at Weeks 12, 24, and 52	Percentage of participants with sustained clinical remission at Weeks 12, 24, and 52 will be assessed. Sustained clinical remission is defined as PCDAI <=10 at Weeks 12, 24, and 52.	Weeks 12, 24, and 52
Serum Concentration of Guselkumab During Induction Phase	Serum concentrations of guselkumab will be assessed. Serum samples will be analyzed to determine concentrations of guselkumab using a validated, specific, and sensitive immunoassay method.	From Week 0 to Week 12
Trough Plasma Concentration (Ctrough) of Guselkumab During Maintenance Phase	Ctrough is defined as the serum concentration of guselkumab immediately prior (pre-dose) to the next drug administration.	At Weeks 16, 24, 36, 48 and 52
Change from Baseline in Body Weight at Weeks 12, 24, and 52	Change from baseline in body weight at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Body Weight Percentiles at Weeks 12, 24, and 52	Change from baseline in body weight percentiles at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Body Weight z-scores at Weeks 12, 24, and 52	Change from baseline in body weight z-scores at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Height at Weeks 12, 24, and 52	Change from baseline in height at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Height Percentiles at Weeks 12, 24, and 52	Change from baseline in height percentiles at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Height z-scores at Weeks 12, 24, and 52	Change from baseline in height z-scores at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Change from Baseline in Height Velocity at Weeks 12, 24, and 52	Change from baseline in height velocity at Weeks 12, 24, and 52 will be assessed.	Baseline, Weeks 12, 24, and 52
Percentage of Participants with Clinical Response at Week 12	Percentage of participants with clinical response at Week 12 will be assessed. Clinical responder is defined as a decrease from baseline in the PCDAI score of >=12.5 points with a total PCDAI score <30.	Week 12
Percentage of Participants with Clinical Response at Week 52	Percentage of participants with clinical response at Week 52 will be assessed. Clinical responder is defined as a decrease from baseline in the PCDAI score of >=12.5 points with a total PCDAI score <30.	Week 52
Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO-2)	Percentage of participants with clinical remission by PRO-2 will be assessed. Clinical remission by PRO-2 is defined as stool frequency (SF) <=3 and abdominal pain (AP) <=1 and no worsening of SF and AP from baseline.	Week 12 and/or Week 52
Percentage of Participants with Clinical Remission	Percentage of participants with clinical remission who were assigned to guselkumab dose regimen 1 and did not receive rescue therapy at Week 52 will be assessed. Clinical remission is defined as PCDAI score <=10.	Week 52
Percentage of Participants Who Achieve Endoscopic Response	Percentage of participants who achieve endoscopic response who were assigned to q4w maintenance therapy and did not receive rescue therapy at Week 52 will be assessed. Endoscopic response is defined as >=50% reduction (global) and >50% reduction (U.S specific) from SES-CD score at baseline.	Week 52
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product that does not necessarily have a causal relationship with the intervention. An SAE is is any untoward medical occurrence that at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product and is medically important.	Up to Week 64

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): March 13, 2024
• Primary Completion (Estimated): October 27, 2027
• Study Completion (Estimated): July 12, 2028

Study Registration Dates

• First Submitted: June 20, 2023
• First Submitted That Met QC Criteria: June 20, 2023
• First Posted (Actual): June 28, 2023

Study Record Updates

• Last Update Posted (Actual): June 5, 2026
• Last Update Submitted That Met QC Criteria: June 4, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric, Inflammatory bowel disease
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Crohn Disease; Inflammatory Bowel Diseases; Immunologic Factors; Physiological Effects of Drugs; guselkumab
• Other Study ID Numbers: CR109212; 2021-006282-37 (EudraCT Number); CNTO1959PBCRD3007 (Other Identifier: Janssen Research & Development, LLC); 2023-504735-41-00 (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 Johnson & Johnson Innovative Medicine is available at www.innovativemedicine.jnj.com/our-innovation/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