A Pharmacokinetic Study of Ustekinumab in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

NCT02968108 | Completed Phase 1 FDA Drug

• 3 other identifiers: CR108233CNTO1275CRD10012016-001956-22
• Study Type: interventional
• Target: 45
• Locations: 6 countries
• Sites: 26

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.

Conditions

Crohn Disease

Outcome Measures

Primary Outcomes (1)

• Serum Ustekinumab Concentrations Over Time

  Serum samples will be collected to measure seum concentrations of Ustekinumab.

  Up to Week 16

Secondary Outcomes (2)

• Clinical Response as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score

  Week 6

• Clinical Remission as Measured by the Pediatric Crohn's Disease Activity Index (PCDAI) Score

  Week 8

Study Arms (2)

Group1: Ustekinumab Dose Regimen 1

EXPERIMENTAL

Subjects will receive a single intravenous (IV) induction dose of 3 milligram per kilogram (mg/kg) for subjects less than \< 40 kilogram (kg) or 130 milligram (mg) for subjects greater than or equal to \>= 40 kg at Week 0 followed by subcutaneous (SC) maintenance dose of 2 mg/kg for subjects \< 40 kg or 90 mg for subjects \>= 40 kg at week 8.

Drug: Ustekinumab

Group2: Ustekinumab Dose Regimen 2

EXPERIMENTAL

Subjects will receive a single Intravenous (IV) dose of 9 mg/kg for subjects \<40 kg or 390 mg for subjects \>= 40 kg at Week 0 followed by SC maintenance dose of 2 mg/kg for subjects \<40 kg or 90 mg for subjects \>= 40 kg at week 8.

Drug: Ustekinumab

Interventions

Ustekinumab DRUG

Subjects will receive a single IV administration of ustekinumab (3 mg/kg for subjects \<40 kg or 130 mg for subjects \>= 40 kg in Group 1 and 9 mg/kg for subjects \< 40 kg or 390 mg for subjects \>= 40 kg in group 2) at week 0 followed by SC administration of ustekinumab (2 mg/kg for subjects \< 40 kg or 90 mg for subjects \>= 40 kg at Week 8.

Group1: Ustekinumab Dose Regimen 1; Group2: Ustekinumab Dose Regimen 2

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (26)

Cedars Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Connecticut Childrens Medical Center

Hartford, Connecticut, 06106, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Children's Center For Digestive Healthcare, Llc

Atlanta, Georgia, 30342, United States

Location

Methodist Medical Center of Illinois

Peoria, Illinois, 61602, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Mount Sinai

New York, New York, 10029, United States

Location

Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Center For Digestive Health Systems-Greenville

Greenville, South Carolina, 29615, United States

Location

Universitair Kinderziekenhuis Koningin Fabiola

Brussels, 1020, Belgium

Location

Cliniques Universitaires Saint Luc

Brussels, 1200, Belgium

Location

UZ Gent

Ghent, 9000, Belgium

Location

UZ Brussel

Jette, 1090, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Stollery Children's Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H3V4, Canada

Location

Children's Hospital of Western Ontario

London, Ontario, N6A 5W9, Canada

Location

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Centre Hospitalier Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Hôpital Necker

Paris, 75015, France

Location

Hôpital Robert Debré

Paris, 75019, France

Location

Dr. von Haunersches Kinderspital

Munich, 80337, Germany

Location

HELIOS Klinikum Wuppertal GmbH

Wuppertal, 42283, Germany

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, 93 338, Poland

Location

WIP Warsaw IBD Point Profesor Kierkus

Warsaw, 00-728, Poland

Location

Uniwersytecki Szpital Kliniczny im Jana Mikulicza Radeckiego we Wroclawiu

Wroclaw, 50 369, Poland

Location

Related Publications (1)

• Hasskamp J, Meinhardt C, Timmer A. Anti-IL-12/23p40 antibodies for induction of remission in Crohn's disease. Cochrane Database Syst Rev. 2025 May 13;5(5):CD007572. doi: 10.1002/14651858.CD007572.pub4.

  PMID: 40357993 DERIVED

MeSH Terms

Conditions

Crohn Disease

Interventions

Ustekinumab

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2016
• First Posted: November 18, 2016
• Study Start: January 18, 2017
• Primary Completion: September 19, 2018
• Study Completion: March 18, 2022
• Last Updated: April 27, 2025

Record last verified: 2025-04

Locations

BE (5); US (9); CA (5); DE (2); FR (2); PL (3)