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

Crohn's Disease Clinical Trial

— 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05923073
• Other study ID #: CR109212
• Secondary ID: 2021-006282-37CN
• Status: Recruiting
• Phase: Phase 3
• Start date: March 13, 2024
• Est. completion date: April 27, 2028

Study information

• Verified date: June 2024
• Source: Janssen Research & Development, LLC
• Contact: Study Contact
• Phone: 844-434-4210
• Email: Participate-In-This-Study[@]its.jnj.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: April 27, 2028
• Est. primary completion date: October 27, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Crohn Disease
• Crohn's Disease

Intervention

Drug:
• Guselkumab
Guselkumab will be administered either intravenously or subcutaneously.
• Placebo
Week 12 induction responders will be administered placebo (matching guselkumab up to Week 48) SC at protocol specified time points to maintain the blind.

Locations

Country	Name	City	State
Italy	Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer	Firenze
Italy	AOU Policlinico Umberto I	Roma
Japan	Hirosaki University Hospital	Hirosaki
Japan	Tsujinaka Hospital Kashiwanoha	Kashiwa-shi
Japan	Saga University Hospital	Saga
Japan	Miyagi Children's Hospital	Sendai
Japan	Juntendo University Hospital	Tokyo
Japan	Yokohama City University Medical Center	Yokohama-shi
Norway	Akershus Universitetssykehus HF	Nordbyhagen
Norway	Oslo University Hospital	Oslo
Norway	Universitetssykehuset Nord-Norge HF	Tromsø
Norway	St. Olavs Hospital	Trondheim
Poland	Korczowski Bartosz Gabinet Lekarski	Rzeszow
Poland	Centrum Zdrowia MDM	Warszawa
Poland	WIP Warsaw IBD Point Profesor Kierkus	Warszawa
Spain	Hosp. Univ. I Politecni La Fe	Valencia
United States	Children's Center for Digestive Health Care	Atlanta	Georgia
United States	Emory University	Atlanta	Georgia
United States	Riley Hospital for Children	Indianapolis	Indiana

Sponsors (1)

Lead Sponsor	Collaborator
Janssen Research & Development, LLC

Countries where clinical trial is conducted

United States,  Italy,  Japan,  Norway,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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.	Week 52
Secondary	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.	Week 12
Secondary	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.	Week 52
Secondary	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
Secondary	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.	Week 12
Secondary	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
Secondary	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
Secondary	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
Secondary	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.	Week 12 and/or Week 52
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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.	Week 52
Secondary	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.	Week 52