Back to resultsA 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
Primary Purpose
Colitis, Ulcerative
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Golimumab
Sponsored by
About this trial
This is an interventional treatment trial for Colitis, Ulcerative focused on measuring Colitis, Ulcerative, Ulcerative Colitis, Moderately to Severely Active Ulcerative Colitis, Golimumab, Simponi, Human anti-TNFα Antibody, Pediatric participants, Safety and Pharmacokinetics
Eligibility Criteria
Inclusion criteria:
- Moderate to severe Ulcerative Colitis (UC) defined by a Mayo score (a score used to assess the treatment for UC) of 6 to 12 inclusive, including an endoscopic subscore of 2 or more.
- Must either be currently receiving treatment with, or have a history of having failed to respond to, or have a medical contraindication to at least 1 of the following therapies: oral or intravenous corticosteroids, 6-mercaptopurine and azathioprine OR must either have or have had a history of corticosteroid dependency (ie, an inability to successfully taper corticosteroids without a return of the symptoms of UC) OR required more than 3 courses of corticosteroids in the past year
- No history of latent or active tuberculosis prior to screening
- Positive protective antibody titers to varicella and measles prior to the first administration of study agent
Exclusion criteria:
- Have severe extensive UC that is likely to require a colectomy (surgical removal of the colon) within 12 weeks of study entry
- Have UC limited to the rectum only or to less than 20 cm of the colon
- Presence of a stoma
- Presence or history of a fistula
- Have evidence of Crohn's disease (an inflammatory large intestine disease)
- Previous exposure to anti-tumor necrosis factor therapy
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Golimumab
Arm Description
Outcomes
Primary Outcome Measures
Serum golimumab concentrations at Week 6
Sample will be collected prior to the administration of study medication.
Secondary Outcome Measures
Number of participants who will achieve clinical response at 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
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
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
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
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.
Full Information
NCT ID
NCT01900574
First Posted
July 12, 2013
Last Updated
May 9, 2023
Sponsor
Janssen Research & Development, LLC
1. Study Identification
Unique Protocol Identification Number
NCT01900574
Brief Title
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
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 Active Ulcerative Colitis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
August 9, 2013 (Actual)
Primary Completion Date
February 18, 2015 (Actual)
Study Completion Date
September 1, 2022 (Actual)
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
Data Monitoring Committee
Yes
5. Study Description
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 Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Colitis, Ulcerative
Keywords
Colitis, Ulcerative, Ulcerative Colitis, Moderately to Severely Active Ulcerative Colitis, Golimumab, Simponi, Human anti-TNFα Antibody, Pediatric participants, Safety and Pharmacokinetics
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
35 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Golimumab
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Golimumab
Other Intervention Name(s)
Simponi
Intervention Description
Golimumab will be administered subcutaneously. For participants weighing less than 45 kg, the dose of golimumab at Week 0 will be 90 mg/m2 (up to 200 mg) and 45 mg/m2 (up to 100 mg) at Weeks 2, 6, and 10. For participants weighing 45 kg or more, the dose of golimumab at Week 0 will be 200 mg and 100 mg at Weeks 2, 6, and 10. From Week 14, the dose of golimumab will be 45 mg/m2 and 100 mg, every 4 weeks for participants weighing less than 45 kg and 45 kg or more, respectively. From Week 18, participants weighing 45 kg or more have the option to administer golimumab at home after being properly trained. At Week 14 or at any time after, the participants have the option to decrease their dose of golimumab to 50 mg (weighing 45 kg or more) or 22.5 mg/square meter (less than 45 kg) at the discretion of the investigator. A single dose increase back to 100 mg or 45 mg/m2 is permitted based on the investigator's assessment of an increase in a participant's ulcerative colitis disease activity.
Primary Outcome Measure Information:
Title
Serum golimumab concentrations at Week 6
Description
Sample will be collected prior to the administration of study medication.
Time Frame
Week 6
Secondary Outcome Measure Information:
Title
Number of participants who will achieve clinical response at Week 6
Description
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.
Time Frame
Week 6
Title
Number of participants who will achieve clinical remission at Week 6 measured by the Mayo score
Description
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.
Time Frame
Week 6
Title
Number of participants who will achieve clinical remission at Week 6 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score
Description
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.
Time Frame
Week 6
Title
Number of participants with mucosal healing at Week 6
Description
Mucosal healing is calculated by endoscopy subscore of the Mayo score of 0 (normal or inactive disease) or 1 (mild disease).
Time Frame
Week 6
Title
Number of participants who will achieve clinical remission at Week 54 and Week 110 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score
Description
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.
Time Frame
Week 54 and Week 110
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:
Moderate to severe Ulcerative Colitis (UC) defined by a Mayo score (a score used to assess the treatment for UC) of 6 to 12 inclusive, including an endoscopic subscore of 2 or more.
Must either be currently receiving treatment with, or have a history of having failed to respond to, or have a medical contraindication to at least 1 of the following therapies: oral or intravenous corticosteroids, 6-mercaptopurine and azathioprine OR must either have or have had a history of corticosteroid dependency (ie, an inability to successfully taper corticosteroids without a return of the symptoms of UC) OR required more than 3 courses of corticosteroids in the past year
No history of latent or active tuberculosis prior to screening
Positive protective antibody titers to varicella and measles prior to the first administration of study agent
Exclusion criteria:
Have severe extensive UC that is likely to require a colectomy (surgical removal of the colon) within 12 weeks of study entry
Have UC limited to the rectum only or to less than 20 cm of the colon
Presence of a stoma
Presence or history of a fistula
Have evidence of Crohn's disease (an inflammatory large intestine disease)
Previous exposure to anti-tumor necrosis factor therapy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Janssen Research & Development, LLC Clinical Trial
Organizational Affiliation
Janssen Research & Development, LLC
Official's Role
Study Director
Facility Information:
City
Phoenix
State/Province
Arizona
Country
United States
City
Los Angeles
State/Province
California
Country
United States
City
San Francisco
State/Province
California
Country
United States
City
Hartford
State/Province
Connecticut
Country
United States
City
Atlanta
State/Province
Georgia
Country
United States
City
Chicago
State/Province
Illinois
Country
United States
City
Rochester
State/Province
Minnesota
Country
United States
City
Morristown
State/Province
New Jersey
Country
United States
City
Seattle
State/Province
Washington
Country
United States
City
Graz
Country
Austria
City
Wien
Country
Austria
City
Brussels
Country
Belgium
City
Brussel
Country
Belgium
City
Edmonton
State/Province
Alberta
Country
Canada
City
Halifax
State/Province
Nova Scotia
Country
Canada
City
Toronto
State/Province
Ontario
Country
Canada
City
Aarhus
Country
Denmark
City
Hvidovre
Country
Denmark
City
Paris
Country
France
City
Aachen
Country
Germany
City
Muenchen
Country
Germany
City
Beer Sheva
Country
Israel
City
Haifa
Country
Israel
City
Jerusalem
Country
Israel
City
Nijmegen
Country
Netherlands
City
Rotterdam
Country
Netherlands
City
Rzeszow Poland
Country
Poland
City
Warszawa
Country
Poland
12. IPD Sharing Statement
Learn more about this 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
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