Clinical trial NCT02968108: A Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
Official title: A Randomized Double-blind Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Crohn Disease
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2016-11-18
Enrollment: 45
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the pharmacokinetics (PK) of ustekinumab in subjects from 2 through less than (\<) 18 years old in the USA, or 6 through less than (\<) 18 years old in other countries and determine if it is similar to that observed in adults with moderately to severely active Crohn's disease (CD). Also to assess the safety, immunogenicity and efficacy of ustekinumab in the treatment of moderately to severely active CD. The main part of the study continues to Week 16, at which point all subjects who are receiving benefit from ustekinumab maintenance therapy (as determined by the investigator) are eligible to enter the long-term extension (LTE) and continue to receive ustekinumab. The study extension ends at Week 268 or upon availability of the LTE basket study (CNTO1275ISD3001) whichever occurs first. If participants do not consent/assent to the LTE basket study, they will continue safety follow-up for approximately 20 weeks after the last study agent administration.


Eligibility criteria: Inclusion Criteria:

* Be a pediatric subject 2 to less than (\<) 18 years old in the US, 6 to \<18 years old elsewhere, of either gender with a body weight of greater than or equal to (\>=) 10 kilogram (kg)
* Have Crohn's disease (CD) or fistulizing CD of at least 3 months duration, with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by radiography, histology, and/or endoscopy
* Must have moderately to severely active CD defined by: Baseline pediatric Crohn's disease activity index (PCDAI) score of greater than (\>)30 and at least one of the following: An abnormal C-reactive protein (CRP) \>0.3 milligram per deciliter (mg/dL) or 3.0 milligram per liter (mg/L) at screening) or fecal calprotectin \>250 milligram per kilogram (mg/kg) at screening or ileocolonoscopy with evidence of active CD (defined as ulcerations in the ileum and/or colon) during screening into this study including at the baseline visit
* Prior or current medication for CD must include at least 1 of the following: Current treatment with at least 1 of the following therapies: oral corticosteroids, the immunomodulators azathioprine, 6-MP, or methotrexate, or currently have or have had a history of corticosteroid dependency, or have a history of failure to respond to, or tolerate, at least 1 of the following therapies including oral or IV corticosteroids or the immunomodulators 6-mercaptopurine, azathioprine, or methotrexate,or have required more than 3 courses of oral or IV corticosteroids in the past year
* Have negative stool results for enteric pathogens. Stool studies must include a stool culture and Clostridium difficile toxin assay. These must have been performed during screening or the current episode of disease exacerbation as long as the stool studies were performed within 4 months prior to the first administration of study agent

Exclusion Criteria:

* Has complications of CD such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery, could preclude the use of the PCDAI to assess response to therapy, or would possibly confound the ability to assess the effect of treatment with ustekinumab
* Currently has or is suspected to have an abscess. Recent cutaneous and perianal abscesses are not exclusionary if drained and adequately treated at least 3 weeks prior to baseline, or 8 weeks prior to baseline for intra-abdominal abscesses, provided that there is no anticipated need for any further surgery. Participant with active fistulas may be included if there is no anticipation of a need for surgery and there are currently no abscesses identified
* Has had any kind of bowel resection within 6 months or any other intra-abdominal surgery within 3 months prior to baseline
* Has a draining (that is (i.e.), functioning) stoma or ostomy
* Presence or history of any malignancy including presence or history of lymphoproliferative disease including lymphoma, or signs and symptoms suggestive of possible lymphoproliferative disease, such as lymphadenopathy of unusual size or location (example, nodes in the posterior triangle of the neck, infraclavicular, epitrochlear, or periaortic areas), or clinically significant hepatomegaly or splenomegaly

Primary outcomes:
measure: Serum Ustekinumab Concentrations Over Time
timeFrame: Up to Week 16
Secondary outcomes:
measure: Clinical Response as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score
timeFrame: Week 6
measure: Clinical Remission as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score
timeFrame: Week 8
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars Sinai Medical Center
state: California
city: Hartford
country: United States
facility: Connecticut Childrens Medical Center
state: Connecticut
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Atlanta
country: United States
facility: Children's Center For Digestive Healthcare, Llc
state: Georgia
city: Peoria
country: United States
facility: Methodist Medical Center of Illinois
state: Illinois
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: Mount Sinai
state: New York
city: Philadelphia
country: United States
facility: Childrens Hospital Of Philadelphia
state: Pennsylvania
city: Greenville
country: United States
facility: Center For Digestive Health Systems-Greenville
state: South Carolina
city: Brussel
country: Belgium
facility: Universitair Kinderziekenhuis Koningin Fabiola
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint Luc
city: Gent
country: Belgium
facility: UZ Gent
city: Jette
country: Belgium
facility: UZ Brussel
city: Leuven
country: Belgium
facility: UZ Leuven
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
state: Alberta
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: London
country: Canada
facility: Children's Hospital of Western Ontario
state: Ontario
city: Toronto
country: Canada
facility: Hospital For Sick Children
state: Ontario
city: Montreal
country: Canada
facility: Centre Hospitalier Sainte Justine
state: Quebec
city: Paris
country: France
facility: Hôpital Necker
city: Paris
country: France
facility: Hôpital Robert Debré
city: Munich
country: Germany
facility: Dr. von Haunersches Kinderspital
city: Wuppertal
country: Germany
facility: HELIOS Klinikum Wuppertal GmbH
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowia Matki Polki
city: Warszawa
country: Poland
facility: WIP Warsaw IBD Point Profesor Kierkus
city: Wroclaw
country: Poland
facility: Uniwersytecki Szpital Kliniczny im Jana Mikulicza Radeckiego we Wroclawiu
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: