Clinical Trial: A Study to Assess the Safety and Pharmacokinetics of Subcutaneously Administered Golimumab, a Human Anti-TNFα Antibody, in Pediatric Patients With Moderate to Severe Active Ulcerative Colitis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 1b Open-Label Study to Assess the Safety and Pharmacokinetics of Subcutaneously Administered Golimumab, a Human Anti-TNFα Antibody, in Pediatric Subjects With Moderately to Severely Activ

Brief Summary: The purpose of this study is to evaluate the pharmacokinetics (what the body does to the study medication) and safety of subcutaneously (under the skin) administered golimumab in pediatric participants (aged 2 to 17 years) with moderately to severely active Ulcerative Colitis (UC).

Detailed Summary: This is an open-label (both [participants and investigator] know what treatment participants will receive) and multicenter study. The study is divided into 2 parts: Part 1: pharmacokinetics portion (Week 0 to Week 14); Part 2: study extension (Week 14 to Week 114). The focus of this study is to evaluate the pharmacokinetics and safety of golimumab. Additionally the efficacy of short-term therapy with golimumab will be evaluated. Participants in clinical response at Week 6 will continue to receive open label golimumab maintenance therapy and will enter the study extension at Week 14. Participants who are not in clinical response at Week 6 will be withdrawn from further treatment with golimumab. At Week 114, participants who, in the opinion of the investigator, may benefit from continued treatment will be eligible to continue to receive golimumab every 4 weeks until marketing authorization is obtained in the treatment of pediatric UC in that country, or until a decision has been made not to pursue an indication in pediatric UC. Approximately 30 participants will be enrolled in this study. The total duration of study participation for a participant will be approximately 126 weeks or longer.
Sponsor: Janssen Research & Development, LLC

Current Primary Outcome:

  • Serum golimumab concentrations at Week 6 [ Time Frame: Week 6 ]
    Sample will be collected prior to the administration of study medication.
  • Area under the curve for serum golimumab concentration from Week 0 through Week 6 [ Time Frame: Week 0 to Week 6 ]
    Sample will be collected prior to the administration of study medication.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of participants with adverse events as a measure of safety [ Time Frame: Up to Week 126 ]
  • Number of participants who will achieve clinical response at Week 6 [ Time Frame: Week 6 ]
    Clinical response is defined as a decrease from baseline in the Mayo score by greater than or equal to 30 percent and greater than or equal to 3 points, with either a decrease from baseline in the rectal bleeding subscore of 1 or more or a rectal bleeding subscore of 0 or 1. The Mayo score consists of 4 subscores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), rated as 0 (normal) to 3 (severe). Total score is calculated as the sum of 4 subscores and values range from 0 to 12 scores, where 3 to 5 = mild; 6 to 10 = moderate; and 11 to 12 = severe. Higher scores indicate worsening of the disease.
  • Number of participants who will achieve clinical remission at Week 6 measured by the Mayo score [ Time Frame: Week 6 ]
    Clinical remission is defined as a Mayo score less than or equal to 2 points, with no individual subscore greater than 1. The Mayo score consists of 4 subscores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), rated as 0 (normal) to 3 (severe). Total score is calculated as the sum of 4 subscores and values range from 0 to 12 scores, where 3 to 5 = mild; 6 to 10 = moderate; and 11 to 12 = severe. Higher scores indicate worsening of the disease.
  • Number of participants who will achieve clinical remission at Week 6 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score [ Time Frame: Week 6 ]
    Clinical remission is defined as a score less than 10 and it will be measured by the PUCAI scores, a noninvasive measure of ulcerative colitis disease activity. The PUCAI score consists of 6 scales (abdominal pain [points 0 to 10], rectal bleeding [points 0 to 30], stool consistency [points 0 to 10], number of stools [points 0 to 15], nocturnal bowel movement [points 0 to 10], and activity level [points 0 to 10]). The total score ranges from 0 and 85 points, where it is calculated as the sum of the 6 scales and decrease of 20 points is considered a minimally clinically important change. Higher scores indicates a more severe disease.
  • Number of participants with mucosal healing at Week 6 [ Time Frame: Week 6 ]
    Mucosal healing is calculated by endoscopy subscore of the Mayo score of 0 (normal or inactive disease) or 1 (mild disease).
  • Number of participants who will achieve clinical remission at Week 54 and Week 110 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score [ Time Frame: Week 54 and Week 110 ]
    Clinical remission is defined as a score less than 10 and it will be measured by the PUCAI score, a noninvasive measure of ulcerative colitis disease activity. The PUCAI score consists of 6 scales (abdominal pain [points 0 to 10], rectal bleeding [points 0 to 30], stool consistency [points 0 to 10], number of stools [points 0 to 15], nocturnal bowel movement [points 0 to 10], and activity level [points 0 to 10]). The total score ranges from 0 and 85 points, where it is calculated as the sum of the 6 scales and decrease of 20 points is considered a minimally clinically important change. Higher scores indicates a more severe disease.


Original Secondary Outcome: Same as current

Information By: Janssen Research & Development, LLC

Dates:
Date Received: July 12, 2013
Date Started: August 2013
Date Completion: August 2023
Last Updated: March 21, 2017
Last Verified: March 2017