The purpose of this study is to evaluate efficacy of golimumab in inducing clinical remission as assessed by the Mayo score, in pediatric participants with moderately to severely active ulcerative colitis (UC). In addition, the safety profile of golimumab, in pediatric participants with moderately to severely active UC will be assessed.

A Study to Assess the Efficacy and Safety of Golimumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Colitis, Ulcerative

Ulcerative Colitis

Gastrointestinal diseases

Immune system conditions

Golimumab

Infliximab

A Phase 3 Randomized, Open-Label Study to Assess the Efficacy, Safety, and Pharmacokinetics of Golimumab Treatment, a Human Anti-TNFα Monoclonal Antibody, Administered Subcutaneously in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Percentage of participants with clinical remission at Week 6 as assessed by the Mayo score was reported. Clinical remission was defined as a Mayo score of less than or equal to (<=) 2 point, with no individual sub-score greater than (>) 1. The Mayo score was sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each rated on a scale from 0 to 3, with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants with symptomatic remission at Week 54 was reported. Symptomatic remission was defined as a Mayo stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0. Mayo stool frequency subscore was determined on the average number of stools more than normal in 24 hours, score ranged from 0 (normal number of stools) to 3 (5 or more stools more than normal), higher score indicated more severity. Mayo rectal bleeding subscore ranged from 0 (no blood seen) to 3 (blood alone passed), higher score indicated more severity.

Percentage of participants with clinical remission at Week 54 was reported. Clinical remission was defined as a Mayo score <= 2 points, with no individual subscore >1. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each rated on a scale from 0 to 3, with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants with clinical remission at Week 54 as assessed by PUCAI score was reported. Clinical remission as measured by the PUCAI score was a PUCAI score <10. PUCAI score was intended for pediatric participants with UC. PUCAI consisted of the following 6 subscores with scores as: abdominal pain (no pain =0, pain can be ignored =5, pain cannot be ignored =10); rectal bleeding (none =0, small amount only [in less than 50 percent (%) of stools] =10, small amount with most stools =20, large amount [>50% of the stool content] =30); stool consistency of most stools (formed =0, partially formed =5, completely unformed =10); number of stools per 24 hours (0-2 =0, 3-5 =5, 6-8 =10, >8 =15); nocturnal bowel movement (no =0, yes =10); activity level (no limitation of activity =0, occasional limitation of activity =5, severely restricted activity =10). PUCAI score = sum of scores of 6 items; score range of 0= no severity to 85= extreme severity.

Percentage of participants with clinical remission at Week 6 as assessed by PUCAI score was reported. Clinical remission as measured by the PUCAI score was a PUCAI score <10. PUCAI score was intended for pediatric participants with UC. PUCAI consisted of the following 6 subscores with scores as: abdominal pain (no pain =0, pain can be ignored =5, pain cannot be ignored =10); rectal bleeding (none =0, small amount only [in less than 50% of stools] =10, small amount with most stools =20, large amount [>50% of the stool content] =30); stool consistency of most stools (formed =0, partially formed =5, completely unformed =10); number of stools per 24 hours (0-2 =0, 3-5 =5, 6-8 =10, >8 =15); nocturnal bowel movement (no =0, yes =10); activity level (no limitation of activity =0, occasional limitation of activity =5, severely restricted activity =10). PUCAI score = sum of scores of 6 items; score range of 0= no severity to 85= extreme severity.

Clinical response was defined as a decrease from baseline in the Mayo score by >= 30% and >= 3 points, with either a decrease from baseline in the rectal bleeding subscore of >= 1 or a rectal bleeding subscore of 0 or 1. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each rated on a scale from 0 to 3, with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants with endoscopic healing at Week 6 as assessed by the Mayo score was reported. Endoscopic healing was defined by an endoscopy subscore of the Mayo score of 0 or 1 based on local endoscopy. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each subscore rated on a scale from 0 (normal) to 3 (severe), with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants with endoscopic healing at Week 54 as assessed by the Mayo score was reported. Endoscopic healing is defined as an endoscopy subscore of the Mayo score of 0 or 1 based on local endoscopy. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each subscore rated on a scale from 0 (normal) to 3 (severe), with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants with clinical remission at Week 54 as assessed by the Mayo score for participants who were in clinical remission at week 6 was reported. Clinical remission was defined as a Mayo score <=2 points, with no individual subscore >1. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each rated on a scale from 0 to 3, with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Percentage of participants who were not receiving corticosteroids for at least 12 weeks prior to Week 54 and in clinical remission at week 54 was reported. Clinical remission was defined as a Mayo score <=2 points, with no individual subscore >1. The Mayo score was the sum of 4 sub-scores (that is, stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment); each rated on a scale from 0 to 3, with higher scores indicating more severe disease. The total score was calculated as the sum of the 4 sub scores and values ranged from 0 to 12. A score of 3 to 5 points indicated mildly active disease; a score of 6 to 10 indicated moderately active disease; and a score of 11 to 12 indicated severe disease.

Participants will receive subcutaneous (SC) golimumab through Week 50. Doses will be based on body surface area. Following the Week 54 evaluations (end of main pivotal study) participants who are benefiting from golimumab at the discretion of the investigator may continue to receive SC golimumab in an extension period until end of study.

Participants will receive infliximab intravenous (IV) through Week 46. Doses will be based on body weight. After the Week 54 evaluations, participants receiving infliximab will be withdrawn from study participation and transition to local standard of care which may include continued commercially available infliximab at the discretion of their physician.

The purpose of this study is to evaluate efficacy of golimumab in inducing clinical remission
      as assessed by the Mayo score, in pediatric participants with moderately to severely active
      ulcerative colitis (UC). In addition, the safety profile of golimumab, in pediatric
      participants with moderately to severely active UC will be assessed.

This is a multicenter study in pediatric participants aged 2 to 17 years with moderately to
      severely active UC, defined as a baseline Mayo score of 6 through 12, inclusive, with an
      endoscopy subscore of greater than or equal to (>=)2. This 54-week study will consist of a
      6-week short-term phase and a 48-week long-term phase followed by a study extension (Week 54
      to end of study). At Week 58, participants who are eligible will continue receiving golimumab
      in the study extension. The primary hypothesis is that golimumab is an effective therapy in
      pediatric UC relative to historical placebo control as assessed by clinical remission based
      on Mayo score. Safety will be monitored throughout the study.

University of California San Francisco

Children's Hospital Colorado and University of Colorado

Rocky Mountain Pediatric Gastroenterology

Connecticut Children's Medical Center

Nemours DuPont Hospital for Children

Children's Center for Digestive Health Care

Mayo Clinic

Columbia University Medical Center

GI For Kids

Children's Medical Center of Dallas

Cook Childrens Medical Center

DHAT Research Institute

Universitair Kinderziekenhuis Koningin Fabiola

Cliniques Universitaires Saint-Luc

UZ Brussel

MK Blumenau Pesquisa Clínica

Hospital Pequeno Principe

Irmandade Santa Casa de Misericordia de Porto Alegre

BR Trials

Hopital Pellegrin CHU Bordeaux

Hôpital Necker

Rambam Medical Center

Shaare Zedek Medical Center

Schneider Children's Medical Center

Sheba Medical Center

Assaf Harofeh Medical Center

Sourasky Medical Center

Azienda USL di Bologna - Ospedale Maggiore

AOU Meyer

AOU Policlinico G.Martino

AOU Policlinico Umberto I

IRCCS Ospedale Pediatrico Bambino Gesu

Casa Sollievo della Sofferenza, IRCCS

IRCCS Materno Infantile Burlo Garofolo

Kyungpook National University Chilgok Hospital

Seoul National University Hospital

Severance Hospital, Yonsei University Health System

Asan Medical Center

Samsung Medical Center

Emma Children's Hospital Academic Medical Center

Isala Kliniek

Szpital Uniwersytecki NR 1 IM. Dr. Antoniego Jurasza

Szpital im. M. Kopernika

Uniwersytecki Szpital Dzieciecy w Krakowie

Wojewodzki Specjalistyczny Szpital Dziecięcy im. Prof. Stanislawa Popowskiego

Korczowski Bartosz, Gabinet Lekarski

Centrum Zdrowia Matki, Dziecka i Młodzieży

Szpital Pomnik Centrum Zdrowia Dziecka

Hosp. Univ. de Cruces

Hosp. Sant Joan de Deu

Hosp. Infantil Univ. Niño Jesus

Hosp. Gral. Univ. Gregorio Maranon

Hosp. Univ. 12 de Octubre

Hosp. Regional Univ. de Malaga

Hosp. Virgen Del Rocio

National Taiwan University Hospital

Taipei Veterans General Hospital

Chang Gung Memorial Hospital- Linkou

Clinical remission is defined as a Mayo score less than or equal to (<=) 2 points, with no individual sub score greater than (>) 1. The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.

Symptomatic remission is defined as Mayo stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0.

Clinical remission is defined as a Mayo score <=2 points, with no individual subscore >1 (based on Mayo endoscopy subscore assigned by the local endoscopist). The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.

Clinical remission is defined as a PUCAI score less than (<)10. The PUCAI is a noninvasive measure of UC disease activity. It comprises 6 scales and total score ranges between 0 and 85 points. The scales are: abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI total score is calculated as the sum of the 6 subscores. Higher scores indicate a more severe disease.

Clinical remission is defined as a PUCAI score <10. The PUCAI is a noninvasive measure of ulcerative colitis (UC) disease activity. It comprises 6 scales and total score ranges between 0 and 85 points. The scales are: abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI total score is calculated as the sum of the 6 subscores. Higher scores indicate a more severe disease.

Clinical response is defined as a decrease from baseline in the Mayo score of greater than or equal to (>=)30 percent (%) and >=3 points, with either a decrease from baseline in the rectal bleeding subscore of >=1 or a rectal bleeding subscore of 0 or 1. The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.

Endoscopic healing is defined as an endoscopy subscore of the Mayo score of 0 (normal or inactive disease) or 1 (mild disease).

Endoscopic healing is defined as an endoscopy subscore of the Mayo score of 0 (normal or active disease) or 1 (mild disease).

Number of participants who were not receiving corticosteroids for at least 12 Weeks prior to Week 54 and in clinical remission at Week 54 will be reported.

Participants will receive subcutaneous (SC) golimumab through Week 50. Doses will be based on body surface area. After the Week 54 evaluations, at the discretion of investigator, participants benefiting from continued SC golimumab will continue to receive SC golimumab in the extension until end of study.

A Study to Assess the Efficacy and Safety of Golimumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Trials at a glance

Trials at a glance

A Study to Assess the Efficacy and Safety of Golimumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Outcome measures

Primary outcome measures

Secondary outcome measures