search
Back to results

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

Primary Purpose

Crohn's Disease

Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Guselkumab
Placebo
Sponsored by
Janssen Research & Development, LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Crohn's Disease focused on measuring Pediatric, Inflammatory bowel disease

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm 4

    Arm 5

    Arm Type

    Experimental

    Experimental

    Experimental

    Experimental

    Experimental

    Arm Label

    Open-label induction phase: Guselkumab IV

    Open-label induction phase: Guselkumab SC

    Double-blind maintenance phase: Guselkumab SC q8w

    Double-blind maintenance phase: Guselkumab SC q4w

    Open-label maintenance phase: Guselkumab SC q4w

    Arm Description

    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.

    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.

    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.

    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.

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

    Outcomes

    Primary Outcome Measures

    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.
    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 from simplified endoscopic score-Crohn's Disease (SES-CD) score at baseline.

    Secondary Outcome Measures

    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/loss of response (LOR) in the PCDAI score of >=12.5 points with a total PCDAI score <=30.
    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/LOR in the PCDAI score of >=12.5 points with a total PCDAI score <=30.
    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.
    Percentage of Participants Who Achieve Endoscopic Response at 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.
    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.
    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.
    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.
    Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO)
    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.
    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.
    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.
    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.
    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.
    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.
    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.
    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.
    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.
    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.
    Percentage of Participants with Clinical Remission
    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.
    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 from SES-CD score at baseline.

    Full Information

    First Posted
    June 20, 2023
    Last Updated
    October 10, 2023
    Sponsor
    Janssen Research & Development, LLC
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT05923073
    Brief Title
    A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease
    Acronym
    MACARONI-23
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    November 15, 2023 (Anticipated)
    Primary Completion Date
    October 27, 2027 (Anticipated)
    Study Completion Date
    January 18, 2028 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Janssen Research & Development, LLC

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. 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.
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Crohn's Disease
    Keywords
    Pediatric, Inflammatory bowel disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigator
    Allocation
    Randomized
    Enrollment
    120 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Open-label induction phase: Guselkumab IV
    Arm Type
    Experimental
    Arm Description
    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.
    Arm Title
    Open-label induction phase: Guselkumab SC
    Arm Type
    Experimental
    Arm Description
    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.
    Arm Title
    Double-blind maintenance phase: Guselkumab SC q8w
    Arm Type
    Experimental
    Arm Description
    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.
    Arm Title
    Double-blind maintenance phase: Guselkumab SC q4w
    Arm Type
    Experimental
    Arm Description
    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.
    Arm Title
    Open-label maintenance phase: Guselkumab SC q4w
    Arm Type
    Experimental
    Arm Description
    Week 12 non-responders will enter open-label maintenance phase to receive guselkumab maintenance dose SC q4w up to Week 48.
    Intervention Type
    Drug
    Intervention Name(s)
    Guselkumab
    Other Intervention Name(s)
    CNTO1959, TREMFYA
    Intervention Description
    Guselkumab will be administered either intravenously or subcutaneously.
    Intervention Type
    Drug
    Intervention Name(s)
    Placebo
    Intervention Description
    Placebo matching to guselkumab will be administered subcutaneously.
    Primary Outcome Measure Information:
    Title
    Percentage of Participants with Clinical Remission at Week 52
    Description
    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.
    Time Frame
    Week 52
    Title
    Percentage of Participants Who Achieve Endoscopic Response at Week 52
    Description
    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.
    Time Frame
    Week 52
    Secondary Outcome Measure Information:
    Title
    Percentage of Participants with Clinical Response at Week 12
    Description
    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.
    Time Frame
    Week 12
    Title
    Percentage of Participants with Clinical Response at Week 52
    Description
    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.
    Time Frame
    Week 52
    Title
    Percentage of Participants with Clinical Remission at Week 12
    Description
    Percentage of participants with clinical remission at Week 12 will be assessed. Clinical remission is defined as PCDAI score <=10.
    Time Frame
    Week 12
    Title
    Percentage of Participants Who Achieve Endoscopic Response at Week 12
    Description
    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.
    Time Frame
    Week 12
    Title
    Percentage of Participants with Endoscopic Remission at Week 52
    Description
    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.
    Time Frame
    Week 52
    Title
    Percentage of Participants with Corticosteroid-free Remission at Week 52
    Description
    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.
    Time Frame
    Week 52
    Title
    Percentage of Participants with Sustained Clinical Remission at Weeks 12, 24, and 52
    Description
    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.
    Time Frame
    Weeks 12, 24, and 52
    Title
    Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO)
    Description
    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.
    Time Frame
    Week 12 and/or Week 52
    Title
    Serum Concentration of Guselkumab During Induction Phase
    Description
    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.
    Time Frame
    From Week 0 to Week 12
    Title
    Trough Plasma Concentration (Ctrough) of Guselkumab During Maintenance Phase
    Description
    Ctrough is defined as the serum concentration of guselkumab immediately prior (pre-dose) to the next drug administration.
    Time Frame
    At Weeks 16, 24, 36, 48 and 52
    Title
    Change from Baseline in Body Weight at Weeks 12, 24, and 52
    Description
    Change from baseline in body weight at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Body Weight Percentiles at Weeks 12, 24, and 52
    Description
    Change from baseline in body weight percentiles at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Body Weight z-scores at Weeks 12, 24, and 52
    Description
    Change from baseline in body weight z-scores at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Height at Weeks 12, 24, and 52
    Description
    Change from baseline in height at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Height Percentiles at Weeks 12, 24, and 52
    Description
    Change from baseline in height percentiles at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Height z-scores at Weeks 12, 24, and 52
    Description
    Change from baseline in height z-scores at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Change from Baseline in Height Velocity at Weeks 12, 24, and 52
    Description
    Change from baseline in height velocity at Weeks 12, 24, and 52 will be assessed.
    Time Frame
    Baseline, Weeks 12, 24, and 52
    Title
    Percentage of Participants with Clinical Remission
    Description
    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.
    Time Frame
    Week 52
    Title
    Percentage of Participants Who Achieve Endoscopic Response
    Description
    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.
    Time Frame
    Week 52

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    17 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Study Contact
    Phone
    844-434-4210
    Email
    Participate-In-This-Study@its.jnj.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Clinical Trial
    Organizational Affiliation
    Janssen Research & Development, LLC
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Plan to Share IPD
    Yes
    IPD Sharing Plan Description
    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
    IPD Sharing URL
    https://www.janssen.com/clinical-trials/transparency

    Learn more about this trial

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

    We'll reach out to this number within 24 hrs