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

Sponsor
Janssen Research & Development, LLC (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05923073
Collaborator
(none)
120
Enrollment
83
Locations
5
Arms
52
Anticipated Duration (Months)
1.4
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

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 Design

Study Type:
Interventional
Anticipated Enrollment :
120 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Multicenter, Randomized, Platform Study of p19 Inhibition of the IL-23 Pathway to Establish Efficacy in Pediatric Crohn's Disease
Anticipated Study Start Date :
Sep 19, 2023
Anticipated Primary Completion Date :
Oct 27, 2027
Anticipated Study Completion Date :
Jan 18, 2028

Arms and Interventions

Arm Intervention/Treatment
Experimental: Open-label induction phase: Guselkumab IV

Participants will receive guselkumab dose intravenously (IV) based on their body weight (BW) at Weeks 0, 4, and 8 during the 12-week open-label induction phase.

Drug: Guselkumab
Guselkumab will be administered either intravenously or subcutaneously.
Other Names:
  • CNTO1959
  • TREMFYA

    		•
    Experimental: Open-label induction phase: Guselkumab SC

    Participants will receive guselkumab dose subcutaneously (SC) based on their BW at Weeks 0, 4, and 8 during the 12-week open-label induction phase.

    Drug: Guselkumab
    Guselkumab will be administered either intravenously or subcutaneously.
    Other Names:
  • CNTO1959
  • TREMFYA

    		•
    Experimental: Double-blind maintenance phase: Guselkumab SC q8w

    At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose SC based on their BW every 8 weeks (q8w) up to Week 48. In addition, participants will also receive placebo (matching guselkumab q8w dose) SC at protocol specified timepoints to maintain the blinding.

    Drug: Guselkumab
    Guselkumab will be administered either intravenously or subcutaneously.
    Other Names:
  • CNTO1959
  • TREMFYA

    • Drug: Placebo
    Placebo matching to guselkumab will be administered subcutaneously.
    Experimental: Double-blind maintenance phase: Guselkumab SC q4w

    At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose SC based on their BW every 4 weeks (q4w) up to Week 48.

    Drug: Guselkumab
    Guselkumab will be administered either intravenously or subcutaneously.
    Other Names:
  • CNTO1959
  • TREMFYA

    		•
    Experimental: Open-label maintenance phase: Guselkumab SC q4w

    Week 12 non-responders will enter open-label maintenance phase to receive guselkumab maintenance dose SC q4w up to Week 48.

    Drug: Guselkumab
    Guselkumab will be administered either intravenously or subcutaneously.
    Other Names:
  • CNTO1959
  • TREMFYA

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Percentage of Participants with Clinical Remission at Week 52 [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.

    2. Percentage of Participants Who Achieve Endoscopic Response at Week 52 [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 from simplified endoscopic score-Crohn's Disease (SES-CD) score at baseline.

    Secondary Outcome Measures

    1. Percentage of Participants with Clinical Response at Week 12 [Week 12]

      Percentage of participants with clinical response at Week 12 will be assessed. Clinical responder is defined as a decrease from baseline/loss of response (LOR) in the PCDAI score of >=12.5 points with a total PCDAI score <=30.

    2. Percentage of Participants with Clinical Response at Week 52 [Week 52]

      Percentage of participants with clinical response at Week 52 will be assessed. Clinical responder is defined as a decrease from baseline/LOR in the PCDAI score of >=12.5 points with a total PCDAI score <=30.

    3. Percentage of Participants with Clinical Remission at Week 12 [Week 12]

      Percentage of participants with clinical remission at Week 12 will be assessed. Clinical remission is defined as PCDAI score <=10.

    4. Percentage of Participants Who Achieve Endoscopic Response at Week 12 [Week 12]

      Percentage of participants who achieve endoscopic response at Week 12 will be assessed. Endoscopic response is defined as >=50% reduction from SES-CD score at baseline.

    5. Percentage of Participants with Endoscopic Remission at Week 52 [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.

    6. Percentage of Participants with Corticosteroid-free Remission at Week 52 [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.

    7. Percentage of Participants with Sustained Clinical Remission at Weeks 12, 24, and 52 [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.

    8. Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO) [Week 12 and/or Week 52]

      Percentage of participants with clinical remission by PRO will be assessed. Clinical remission by PRO is defined as stool frequency (SF) <=3 and abdominal pain (AP) <=1 and no worsening of SF and AP from baseline.

    9. Serum Concentration of Guselkumab During Induction Phase [From Week 0 to Week 12]

      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.

    10. Trough Plasma Concentration (Ctrough) of Guselkumab During Maintenance Phase [At Weeks 16, 24, 36, 48 and 52]

      Ctrough is defined as the serum concentration of guselkumab immediately prior (pre-dose) to the next drug administration.

    11. Change from Baseline in Body Weight at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in body weight at Weeks 12, 24, and 52 will be assessed.

    12. Change from Baseline in Body Weight Percentiles at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in body weight percentiles at Weeks 12, 24, and 52 will be assessed.

    13. Change from Baseline in Body Weight z-scores at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in body weight z-scores at Weeks 12, 24, and 52 will be assessed.

    14. Change from Baseline in Height at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in height at Weeks 12, 24, and 52 will be assessed.

    15. Change from Baseline in Height Percentiles at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in height percentiles at Weeks 12, 24, and 52 will be assessed.

    16. Change from Baseline in Height z-scores at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in height z-scores at Weeks 12, 24, and 52 will be assessed.

    17. Change from Baseline in Height Velocity at Weeks 12, 24, and 52 [Baseline, Weeks 12, 24, and 52]

      Change from baseline in height velocity at Weeks 12, 24, and 52 will be assessed.

    18. Percentage of Participants with Clinical Remission [Week 52]

      Percentage of participants with clinical remission who were assigned to q4w maintenance therapy and did not receive rescue therapy at Week 52 will be assessed. Clinical remission is defined as PCDAI score <=10.

    19. Percentage of Participants Who Achieve Endoscopic Response [Week 52]

      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 from SES-CD score at baseline.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 17 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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 [>] 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) during screening into this study

    • Participants must have a prior or current CD medication history that includes either inadequate response, loss of response to or failure to tolerate current treatment immunomodulators or with oral or intravenously (IV) corticosteroids or have received biologic therapy/JAK inhibitor for the treatment of CD and have a documented history of inadequate response, loss of response (LOR), or intolerance to the biologic therapy/JAK inhibitor

    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Phoenix Children's Hospital Phoenix Arizona United States 85016
    2 Arkansas Childrens Hospital Research Institute Little Rock Arkansas United States 72202
    3 Cedars-Sinai Medical Center Los Angeles California United States 90048
    4 University of California San Francisco San Francisco California United States 94143
    5 Connecticut Children's Medical Center Hartford Connecticut United States 06106
    6 Emory University Atlanta Georgia United States 30322
    7 Children's Center for Digestive Health Care Atlanta Georgia United States 30342
    8 Riley Hospital for Children Indianapolis Indiana United States 46202-5225
    9 Boston Children's Hospital Boston Massachusetts United States 02115
    10 The Retina Inst. William Beaumont Hospital Royal Oak Michigan United States 48073
    11 Weill Cornell Medical College - Judith Jaffe Multiple Sclerosis Center New York New York United States 10021-5663
    12 Icahn School of Medicine at Mount Sinai New York New York United States 10029
    13 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27514
    14 The Cleveland Clinic Foundation Cleveland Ohio United States 44195
    15 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    16 Palmetto Pediatric Gastroenterology &amp; Nutrition Columbia South Carolina United States 29203
    17 East Tennessee Children's Hospital Knoxville Tennessee United States 37916
    18 Cook Childrens Medical Center Fort Worth Texas United States 76104
    19 Texas Children's Hospital Houston Texas United States 77030
    20 University of Vermont Medical Center Colchester Vermont United States 05446
    21 Children's Hospital of Wisconsin Milwaukee Wisconsin United States 53226-4874
    22 Perth Children's Hospital Nedlands Australia 6009
    23 Women's and Children's Hospital North Adelaide Australia 5006
    24 Mater Hospital Brisbane South Brisbane Australia 4101
    25 AKH - Medizinische Universität Wien Vienna Austria 1090
    26 Universitair Ziekenhuis Brussel Brussels Belgium 1090
    27 Cliniques Universitaires Saint-Luc Brussel Belgium 1200
    28 Universitair Ziekenhuis Gent Gent Belgium 9000
    29 UZ Leuven Leuven Belgium 3000
    30 Sociedade Campineira de Educacao e Instrucao - Hospital e Maternidade Celso Pierro Campinas Brazil 13060-904
    31 Associacao Hospitalar de Protecao a Infancia Dr. Raul Carneiro Curitiba Brazil 80.250-060
    32 Universidade Federal de Goias - Hospital das Clinicas da UFG Goiânia Brazil 74605-020
    33 Irmandade Santa Casa de Misericordia de Porto Alegre Porto Alegre Brazil 90035-074
    34 Hospital das Clínicas da Faculdade de Medicina de RPUSP - HCRP Ribeirao Preto Brazil 14098-900
    35 Centro de Ciências e Saúde da Universidade Federal do Espirito Santo-Núcleo de Doenças Infecciosas Vitória Brazil 29040-091
    36 British Columbia Children's Hospital Vancouver British Columbia Canada V6H 3N1
    37 Iwk Health Centre Halifax Nova Scotia Canada B3K 6R8
    38 London Health Sciences Centre - Victoria Hospital London Ontario Canada N6A 5W9
    39 Hospital For Sick Children Toronto Ontario Canada M5G 1X8
    40 Fakultni nemocnice v Motole Praha 5 Czechia 150 06
    41 CHU Amiens-Hopital Nord Amiens Cedex 1 France 80054
    42 Hôpital Jeanne de FLANDRE, CHRU LILLE Lille France 59037
    43 Hôpital Necker - Enfants Malades Paris cedex 15 France 75015
    44 Hôpital Robert Debré Paris France 75019
    45 Shamir Medical Center (Assaf Harofeh) Be'er Ya'akov Israel 70300
    46 Soroka University Medical Center Beersheba Israel 84101
    47 Rambam Health Care Campus Haifa Israel 3109601
    48 Shaare Zedek Medical Center Jerusalem Israel 9103102
    49 Hadassah Medical Center Jerusalem Israel 9124001
    50 Schneider Children's Medical Center Petach-Tikva Israel 4920235
    51 Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII) Bergamo Italy 24127
    52 Ospedale Bellaria, U.O.Cardiologia Az. USL di Bologna Bologna Italy 40124
    53 IRCCS Ospedale Pediatrico Bambino Gesu Roma Italy 00165
    54 AOU Policlinico Umberto I Roma Italy 161
    55 Kyungpook National University Chilgok Hospital Daegu Korea, Republic of 41404
    56 Seoul National University Hospital Seoul Korea, Republic of 03080
    57 Samsung Medical Center Seoul Korea, Republic of 06351
    58 Severance Hospital, Yonsei University Health System Seoul Korea, Republic of 120-752
    59 Erasmus Medisch Centrum Rotterdam Netherlands 3015 GD
    60 Akershus Universitetssykehus HF Nordbyhagen Norway 1474
    61 Oslo University Hospital Oslo Norway 424
    62 Universitetssykehuset Nord-Norge HF Tromsø Norway 9038
    63 St. Olavs Hospital Trondheim Norway 7030
    64 Uniwersytecki Szpital Dzieciecy w Krakowie Krakow Poland 30-663
    65 Korczowski Bartosz, Gabinet Lekarski Rzeszow Poland 35-302
    66 Twoja Przychodnia - Szczecinskie Centrum Medyczne Szczecin Poland 71-434
    67 Centrum Zdrowia MDM Warszawa Poland 00-635
    68 WIP Warsaw IBD Point Profesor Kierkus Warszawa Poland 00-728
    69 Hospital de Braga Braga Portugal 4710-243
    70 Centro Hospitalar de Lisboa Norte - Hospital Santa Maria Lisboa Portugal 1649-035
    71 Centro Hospitalar de São João, E.P.E. Porto Portugal 4200-319
    72 Hosp. Reina Sofia Córdoba Spain 14004
    73 Hosp. Infantil Univ. Niño Jesus Madrid Spain 28009
    74 Hosp. Univ. de La Paz Madrid Spain 28046
    75 Corporacio Sanitari Parc Tauli Sabadell Spain 8208
    76 Hosp. Univ. I Politecni La Fe Valencia Spain 46026
    77 INSELSPITAL, Universitätsspital Bern Bern Switzerland 3010
    78 Kinderspital Zürich Zürich Switzerland 8032
    79 King's College Hospital Denmark Hill United Kingdom SE5 9RS
    80 Royal Hospital for Sick Children Glasgow United Kingdom G51 4TF
    81 Nowgen Centre, Research and Innovation Manchester United Kingdom M13 9WL
    82 Royal Manchester Children's Hospital Manchester United Kingdom M13 9WL
    83 Sheffield Children's Hospital Sheffield United Kingdom S1 0 2TH

    Sponsors and Collaborators

    • Janssen Research & Development, LLC

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Janssen Research & Development, LLC
    ClinicalTrials.gov Identifier:
    NCT05923073
    Other Study ID Numbers:
    • CR109212
    • 2021-006282-37
    • CNTO1959PBCRD3007
    First Posted:
    Jun 28, 2023
    Last Update Posted:
    Jun 28, 2023
    Last Verified:
    Jun 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Janssen Research & Development, LLC
    Pediatric, Inflammatory bowel disease
    Additional relevant MeSH terms:
    Crohn Disease

    Study Results

    No Results Posted as of Jun 28, 2023