A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease
- Registration Number
- NCT05923073
- Lead Sponsor
- Janssen Research & Development, LLC
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 120
- 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 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Open-label induction phase: Guselkumab Intravenously (IV) Guselkumab Participants will receive guselkumab dose IV based on their body weight during the 12-week open-label induction phase. Open-label induction phase: Guselkumab Subcutaneously (SC) Guselkumab Participants will receive guselkumab dose SC based on their body weight during the 12-week open-label induction phase. Double-blind maintenance phase: Guselkumab SC Dose Regimen 1 Guselkumab 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. Double-blind Maintenance Phase: Guselkumab SC Dose Regimen 2 Guselkumab 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. Open-label maintenance phase: Guselkumab SC Guselkumab 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.
- Primary Outcome Measures
Name Time Method 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.
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
Name Time Method 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 in the PCDAI score of \>=12.5 points with a total PCDAI score \<30.
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 in the PCDAI score of \>=12.5 points with a total PCDAI score \<30.
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.
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.
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.
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.
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.
Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO-2) Week 12 and/or Week 52 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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Trial Locations
- Locations (67)
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
Sociedade Campineira de Educacao e Instrucao Hospital e Maternidade Celso Pierro
🇧🇷Campinas, Brazil
Associacao Hospitalar de Protecao a Infancia Dr. Raul Carneiro
🇧🇷Curitiba, Brazil
Universidade Federal de Goias - Hospital das Clinicas da UFG
🇧🇷Goiania, Brazil
Irmandade Santa Casa de Misericordia de Porto Alegre
🇧🇷Porto Alegre, Brazil
Centro de Ciências e Saúde da Universidade Federal do Espirito Santo-Núcleo de Doenças Infecciosas
🇧🇷Vitória, Brazil
INTEGRAL Pesquisa e Ensino
🇧🇷Votuporanga, Brazil
CHU Amiens-Hopital Nord
🇫🇷Amiens Cedex 1, France
Hôpital Jeanne de FLANDRE, CHRU LILLE
🇫🇷Lille, France
Hôpital Necker - Enfants Malades
🇫🇷Paris cedex 15, France
Hôpital Robert Debré
🇫🇷Paris, France
Shamir Medical Center Assaf Harofeh
🇮🇱Be Er Ya Akov, Israel
Soroka University Medical Center
🇮🇱Beersheba, Israel
Shaare Zedek Medical Center
🇮🇱Jerusalem, Israel
Hadassah Medical Center
🇮🇱Jerusalem, Israel
Schneider Children's Medical Center
🇮🇱Petach-Tikva, Israel
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)
🇮🇹Bergamo, Italy
Ospedale Bellaria, U.O.Cardiologia Az. USL di Bologna
🇮🇹Bologna, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
🇮🇹Firenze, Italy
Azienda Socio Sanitaria Territoriale Fatebenefratelli Presidio Ospedale dei Bambini Vittore Buzzi
🇮🇹Milano, Italy
AOU Policlinico Umberto I
🇮🇹Roma, Italy
Hirosaki University Hospital
🇯🇵Hirosaki, Japan
Tsujinaka Hospital Kashiwanoha
🇯🇵Kashiwa-shi, Japan
Saga University Hospital
🇯🇵Saga, Japan
Miyagi Children's Hospital
🇯🇵Sendai, Japan
National Center for Child Health and Development
🇯🇵Setagaya Ku, Japan
Juntendo University Hospital
🇯🇵Tokyo, Japan
Yokohama City University Medical Center
🇯🇵Yokohama-shi, Japan
Inje University Haeundae Paik Hospital
🇰🇷Busan, Korea, Republic of
Kyungpook National University Chilgok Hospital
🇰🇷Daegu, Korea, Republic of
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Samsung Medical Center
🇰🇷Seoul, Korea, Republic of
Severance Hospital Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
Erasmus Medisch Centrum
🇳🇱Rotterdam, Netherlands
Akershus Universitetssykehus HF
🇳🇴Nordbyhagen, Norway
Oslo University Hospital
🇳🇴Oslo, Norway
Universitetssykehuset Nord-Norge HF
🇳🇴Tromsø, Norway
St. Olavs Hospital
🇳🇴Trondheim, Norway
Korczowski Bartosz Gabinet Lekarski
🇵🇱Rzeszow, Poland
WIP Warsaw IBD Point Profesor Kierkus
🇵🇱Warszawa, Poland
Instytut Pomnik Centrum Zdrowia
🇵🇱Warszawa, Poland
Hospital de Braga
🇵🇹Braga, Portugal
Centro Hospitalar de Lisboa Norte Hospital Santa Maria
🇵🇹Lisboa, Portugal
Centro Hospitalar de Sao Joao E.P.E.
🇵🇹Porto, Portugal
Hosp Reina Sofia
🇪🇸Córdoba, Spain
Corporacio Sanitari Parc Tauli
🇪🇸Sabadell, Spain
Hosp. Univ. I Politecni La Fe
🇪🇸Valencia, Spain
Royal Hospital for Sick Children
🇬🇧Glasgow, United Kingdom
Great Ormond Street Hospital
🇬🇧London, United Kingdom
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
Sheffield Children's Hospital
🇬🇧Sheffield, United Kingdom
Nowgen Centre, Research and Innovation
🇬🇧Manchester, United Kingdom
Connecticut Children's Medical Center
🇺🇸Hartford, Connecticut, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Children's Center for Digestive Health Care
🇺🇸Atlanta, Georgia, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Weill Cornell Medical College - Judith Jaffe Multiple Sclerosis Center
🇺🇸New York, New York, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
The Children's Medical Center of Dayton
🇺🇸Dayton, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Cook Childrens Medical Center
🇺🇸Fort Worth, Texas, United States
University of Vermont Medical Center
🇺🇸Colchester, Vermont, United States
AKH - Medizinische Universitat Wien
🇦🇹Vienna, Austria
Universitair Ziekenhuis Brussel
🇧🇪Brussels, Belgium
Cliniques Universitaires Saint Luc
🇧🇪Bruxelles, Belgium