Clinical trial NCT01900574: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Colitis, Ulcerative
Keywords: Colitis, Ulcerative
Ulcerative Colitis
Moderately to Severely Active Ulcerative Colitis
Golimumab
Simponi
Human anti-TNFα Antibody
Pediatric participants
Safety and Pharmacokinetics
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2013-07-16
Enrollment: 35
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 Participants who are not in clinical response at Week 6 will be withdrawn from further treatment with golimumab.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.

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

Primary outcomes:
measure: Serum golimumab concentrations at Week 6
timeFrame: Week 6
Secondary outcomes:
measure: Number of participants who will achieve clinical response at Week 6
timeFrame: Week 6
measure: Number of participants who will achieve clinical remission at Week 6 measured by the Mayo score
timeFrame: Week 6
measure: Number of participants who will achieve clinical remission at Week 6 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score
timeFrame: Week 6
measure: Number of participants with mucosal healing at Week 6
timeFrame: Week 6
measure: Number of participants who will achieve clinical remission at Week 54 and Week 110 measured by Pediatric Ulcerative Colitis Activity Index (PUCAI) score
timeFrame: Week 54 and Week 110
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Los Angeles
country: United States
state: California
city: San Francisco
country: United States
state: California
city: Hartford
country: United States
state: Connecticut
city: Atlanta
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Rochester
country: United States
state: Minnesota
city: Morristown
country: United States
state: New Jersey
city: Seattle
country: United States
state: Washington
city: Graz
country: Austria
city: Wien
country: Austria
city: Brussels
country: Belgium
city: Brussel
country: Belgium
city: Edmonton
country: Canada
state: Alberta
city: Halifax
country: Canada
state: Nova Scotia
city: Toronto
country: Canada
state: Ontario
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
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: