A Study to Evaluate the Safety and Efficacy of Ustekinumab Induction and Maintenance Therapy in Participants With Moderately to Severely Active Ulcerative Colitis

NCT ID: NCT02407236

Last Updated: 2025-04-29

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

961 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-10

Study Completion Date

2021-11-30

Brief Summary

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The purpose of this study is to evaluate the efficacy and safety of ustekinumab as intravenous (IV: into the vein) infusion in induction study in participants with moderately to severely active Ulcerative Colitis (UC) and as subcutaneous (SC) administration in maintenance study in participants with moderately to severely active Ulcerative Colitis (UC) who have demonstrated a clinical response to Induction treatment with IV ustekinumab.

Detailed Description

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This is a Phase 3, randomized (assignment of study drug by chance), double-blind (neither the participant or study staff will know the identity of study drugs), placebo-controlled (placebo is an inactive substance that is compared with a drug to test whether the drug has a real effect in a clinical trial), parallel-group (a medical research study comparing the response in 2 or more groups of participants receiving different interventions), multi-center (more than one clinical site will work on a medical research study), protocol of ustekinumab. The protocol will consist of 2 studies: an Induction study and a Maintenance study with unique endpoints. Screening period will be up to 8 Weeks. Induction study will be at least 8 weeks duration for each participant. Participant with clinical response in the Induction study will be eligible for the Maintenance study. The Maintenance study will be 44 weeks duration. After completion of the maintenance study, a long term extension will follow eligible participants for an additional 3 years. Clinical remission will be evaluated at Week 8 in the Induction study. Clinical remission among ustekinumab Induction responders will be evaluated at week 44 in the Maintenance study. Participants' safety will be monitored throughout.

Conditions

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Colitis, Ulcerative Inflammatory Bowel Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Induction Study - Placebo Intravenous (IV)

Participants will be randomized to receive single dose of placebo as Intravenous (IV: into the vein) infusion at Week 0. Participants with clinical response at Week 8 will be eligible to enter the Maintenance study, but will not be randomized.

Group Type PLACEBO_COMPARATOR

Placebo IV

Intervention Type DRUG

Placebo will be administered as intravenous infusion.

Induction Study - Ustekinumab 130 milligram (mg) IV

Participants will be randomized to receive single dose of ustekinumab 130 mg as IV infusion at Week 0. Participants with clinical response at Week 8 will be eligible to enter the Maintenance study and will be randomized.

Group Type EXPERIMENTAL

Ustekinumab IV

Intervention Type DRUG

Ustekinumab will be administered as intravenous infusion at Week 0 or Week 8 in Induction Study.

Induction Study - Ustekinumab 6 mg/kg IV

Participants will be randomized to receive ustekinumab approximating 6 mg/kg of body weight, as intravenous infusion at Week 0. Participants with clinical response at Week 8 will be eligible to enter the Maintenance study and will be randomized.

Group Type EXPERIMENTAL

Ustekinumab IV

Intervention Type DRUG

Ustekinumab will be administered as intravenous infusion at Week 0 or Week 8 in Induction Study.

Induction Study- Placebo- Nonresponsders at Week 8

Participants without clinical response to placebo at Week 8 will receive a single IV infusion of ustekinumab approximating 6mg/kg along with matching subcutaneous (SC) placebo (to maintain the blind). Participants in clinical response at Week 16 will be eligible to enter Maintenance study and will be randomized.

Group Type OTHER

Placebo SC

Intervention Type DRUG

Placebo will be administered Subcutaneously.

Ustekinumab IV

Intervention Type DRUG

Ustekinumab will be administered as intravenous infusion at Week 0 or Week 8 in Induction Study.

Induction study-Ustekinumab Nonresponders at Week 8

Participants without clinical response to ustekinumab (130 mg or 6 mg/kg \[IV\]) at Week 8 will receive a single dose of ustekinumab 90 mg subcutaneously along with matching placebo intravenously (to maintain the blind). Participants in clinical response at Week 16 (that is, delayed responders) will be eligible to enter Maintenance study, but will not be randomized.

Group Type OTHER

Placebo IV

Intervention Type DRUG

Placebo will be administered as intravenous infusion.

Ustekinumab SC

Intervention Type DRUG

Ustekinumab will be administered as subcutaneously.

Maintenance Study - Placebo Subcutaneous (SC)

Participants in clinical response (at Week 8 or Week 16) to Induction treatment with single IV infusion of Ustekinumab will be randomized to receive placebo subcutaneously, beginning Week 0 of Maintenance study through Week 44.

Group Type PLACEBO_COMPARATOR

Placebo SC

Intervention Type DRUG

Placebo will be administered Subcutaneously.

Maintenance Study - Ustekinumab 90mg SC every 12 weeks

Participants in clinical response (at Week 8 or Week 16) to Induction treatment with single IV infusion of Ustekinumab will be randomized to receive ustekinumab 90 mg subcutaneously every 12 weeks, beginning Week 0 of Maintenance study through Week 44.

Group Type EXPERIMENTAL

Ustekinumab SC

Intervention Type DRUG

Ustekinumab will be administered as subcutaneously.

Maintenance Study - Ustekinumab 90mg SC every 8 weeks (q8w)

Participants in clinical response (at Week 8 or Week 16) to Induction treatment with single IV infusion of Ustekinumab will be randomized to receive ustekinumab 90 mg subcutaneously every 8 weeks, beginning Week 0 of Maintenance study through Week 44.

Group Type EXPERIMENTAL

Ustekinumab SC

Intervention Type DRUG

Ustekinumab will be administered as subcutaneously.

Maintenance Study - Placebo IV - Responder - Placebo SC

Participants in clinical response to Induction treatment with IV Placebo will receive placebo subcutaneously, beginning Week 0 of Maintenance study through Week 44. Participants are not randomized.

Group Type OTHER

Placebo SC

Intervention Type DRUG

Placebo will be administered Subcutaneously.

Maintenance Study-Delayed Responder-Ustekinumab 90mg SC q8w

Participants without clinical response to induction treatment ustekinumab (130 mg or 6 mg/kg \[IV\]) at Week 8 but in clinical response at Week 16 after receiving Induction Ustekinumab at week 8 (delayed responders) will receive ustekinumab 90 mg subcutaneously every 8 weeks, beginning Week 0 of Maintenance study through Week 44. Participants are not randomized.

Group Type OTHER

Ustekinumab SC

Intervention Type DRUG

Ustekinumab will be administered as subcutaneously.

Interventions

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Placebo IV

Placebo will be administered as intravenous infusion.

Intervention Type DRUG

Placebo SC

Placebo will be administered Subcutaneously.

Intervention Type DRUG

Ustekinumab IV

Ustekinumab will be administered as intravenous infusion at Week 0 or Week 8 in Induction Study.

Intervention Type DRUG

Ustekinumab SC

Ustekinumab will be administered as subcutaneously.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Has a clinical diagnosis of Ulcerative Colitis (UC) at least 3 months before Screening
* Has moderately to severely active UC, defined as a Baseline (Week 0) Mayo score of 6 to 12, including a Screening endoscopy subscore of the Mayo score greater than or equal to (\>=) 2 as determined by a central reading of the video endoscopy
* Have failed biologic therapy, that is, have received treatment with 1 or more tumour necrosis factor (TNF) antagonists or vedolizumab at a dose approved for the treatment of UC, and have a documented history of failure to respond to or tolerate such treatment; OR Be naïve to biologic therapy (TNF antagonists or vedolizumab) or have received biologic therapy but have not demonstrated a history of failure to respond to, or tolerate, a biologic therapy and have a prior or current UC medication history that includes at least 1 of the following: a. Inadequate response to or failure to tolerate current treatment with oral corticosteroids or immunomodulators (6-mercaptopurine \[6-MP\] or azathioprine \[AZA\]) OR b. History of failure to respond to, or tolerate, at least 1 of the following therapies: oral or IV corticosteroids or immunomodulators (6-MP or AZA) OR c. History of corticosteroid dependence (that is, an inability to successfully taper corticosteroids without a return of the symptoms of UC)
* Before the first administration of study agent, the following conditions must be met: vedolizumab must have been discontinued for at least 4 months and anti-tumor necrosis factors (TNFs) for at least 8 weeks

Exclusion Criteria

* Has severe extensive colitis and is at imminent risk of colectomy
* Has UC limited to the rectum only or to \< 20 centimeters (cm) of the colon
* Presence of a stoma or history of a fistula
* Participants with history of extensive colonic resection (for example, less than 30 cm of colon remaining) that would prevent adequate evaluation of the effect of study agent on clinical disease activity
* Participants with history of colonic mucosal dysplasia. Participants will not be excluded from the study because of a pathology finding of "indefinite dysplasia with reactive atypia''
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Janssen Research & Development, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Janssen Research & Development, LLC Clinical Trial

Role: STUDY_DIRECTOR

Janssen Research & Development, LLC

Locations

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Birmingham, Alabama, United States

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La Mirada, California, United States

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Los Angeles, California, United States

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Newport Beach, California, United States

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Torrance, California, United States

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Vallejo, California, United States

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Lone Tree, Colorado, United States

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Farmington, Connecticut, United States

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Washington D.C., District of Columbia, United States

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Gainesville, Florida, United States

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Largo, Florida, United States

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Miami, Florida, United States

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Port Orange, Florida, United States

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Tampa, Florida, United States

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Winter Park, Florida, United States

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Zephyrhills, Florida, United States

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Atlanta, Georgia, United States

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Decatur, Georgia, United States

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Macon, Georgia, United States

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Suwanee, Georgia, United States

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Idaho Falls, Idaho, United States

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Chicago, Illinois, United States

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Evanston, Illinois, United States

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Urbana, Illinois, United States

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Indianapolis, Indiana, United States

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Pratt, Kansas, United States

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Crestview Hills, Kentucky, United States

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Lexington, Kentucky, United States

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Louisville, Kentucky, United States

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Houma, Louisiana, United States

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Shreveport, Louisiana, United States

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Columbia, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Chesterfield, Michigan, United States

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Troy, Michigan, United States

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Ypsilanti, Michigan, United States

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Rochester, Minnesota, United States

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Jackson, Mississippi, United States

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Marlton, New Jersey, United States

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Morristown, New Jersey, United States

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Brooklyn, New York, United States

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Mineola, New York, United States

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New York, New York, United States

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Poughkeepsie, New York, United States

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Rochester, New York, United States

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The Bronx, New York, United States

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Cincinnati, Ohio, United States

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Mentor, Ohio, United States

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Portland, Oregon, United States

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Doylestown, Pennsylvania, United States

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Hershey, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Sayre, Pennsylvania, United States

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Germantown, Tennessee, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Houston, Texas, United States

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Irving, Texas, United States

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San Antonio, Texas, United States

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Southlake, Texas, United States

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Tyler, Texas, United States

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Salt Lake City, Utah, United States

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West Jordan, Utah, United States

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Chesapeake, Virginia, United States

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Fairfax, Virginia, United States

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Roanoke, Virginia, United States

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Seattle, Washington, United States

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Bedford, , Australia

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Clayton, , Australia

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Concord, , Australia

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Fitzroy, , Australia

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Five Dock, , Australia

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Garran, , Australia

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Heidelberg, , Australia

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Liverpool, , Australia

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Melbourne, , Australia

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South Brisbane, , Australia

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Salzburg, , Austria

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Vienna, , Austria

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Antwerp, , Belgium

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Ghent, , Belgium

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Kortrijk, , Belgium

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Leuven, , Belgium

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Liège, , Belgium

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Roeselaere, , Belgium

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Pleven, , Bulgaria

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Rousse, , Bulgaria

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Sevlievo, , Bulgaria

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Sofia, , Bulgaria

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Varna, , Bulgaria

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Vancouver, British Columbia, Canada

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Victoria, British Columbia, Canada

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Brandon, Manitoba, Canada

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Winnipeg, Manitoba, Canada

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Greater Sudbury, Ontario, Canada

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London, Ontario, Canada

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Montreal, Quebec, Canada

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Hradec Králové, , Czechia

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Pilsen, , Czechia

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Prague, , Czechia

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Aarhus, , Denmark

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Odense, , Denmark

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Amiens, , France

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Bordeaux, , France

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Lille, , France

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Lyon, , France

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Marseille, , France

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Montpellier, , France

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Pierre-Bénite, , France

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Reims, , France

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Rennes, , France

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Saint-Etienne, , France

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Toulouse, , France

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Berlin, , Germany

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Essen, , Germany

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Freiburg im Breisgau, , Germany

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Hanover, , Germany

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Kiel, , Germany

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Leipzig, , Germany

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Lüneburg, , Germany

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Mannheim, , Germany

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Minden, , Germany

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Münster, , Germany

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Balatonfüred, , Hungary

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Békéscsaba, , Hungary

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Budapest, , Hungary

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Debrecen, , Hungary

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Miskolc, , Hungary

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Mosonmagyaróvár, , Hungary

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Szekszárd, , Hungary

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Székesfehérvár, , Hungary

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Szombathely, , Hungary

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Vác, , Hungary

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Beersheba, , Israel

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Haifa, , Israel

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Holon, , Israel

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Jerusalem, , Israel

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Kfar Saba, , Israel

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Nahariya, , Israel

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Petach Tikvah, , Israel

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Tel Aviv, , Israel

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Tel Litwinsky, , Israel

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Ageo-shi, , Japan

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Asahikawa, , Japan

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Bunkyō City, , Japan

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Chiba, , Japan

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Chikushinoshi, , Japan

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Fujiidera, , Japan

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Fukuoka-ken, , Japan

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Higashi-Ibaraki, , Japan

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Hirosaki, , Japan

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Hiroshima, , Japan

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Isesaki, , Japan

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Izumo, , Japan

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Kagoshima, , Japan

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Kahoku, , Japan

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Kobe, , Japan

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Kochi, , Japan

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Kurume, , Japan

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Kyoto, , Japan

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Midori-ku, , Japan

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Nagasaki, , Japan

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Nara, , Japan

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Nishinomiya, , Japan

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Numakunai, , Japan

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Osaka, , Japan

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Ōita, , Japan

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Saga, , Japan

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Saga-ken, , Japan

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Saitama, , Japan

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Sakura, , Japan

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Sapporo, , Japan

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Sendai, , Japan

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Shizuoka, , Japan

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Sunto-gun, , Japan

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Takamatsu, , Japan

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Tokorozawa, , Japan

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Tokyo, , Japan

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Toyama, , Japan

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Toyota, , Japan

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Tsu, , Japan

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Tsuchiura, , Japan

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Wakayama, , Japan

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Yamanashi, , Japan

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Amsterdam, , Netherlands

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Maastricht, , Netherlands

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Auckland, , New Zealand

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Christchurch, , New Zealand

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Dunedin, , New Zealand

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Lower Hutt, , New Zealand

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Milford, , New Zealand

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Gdansk, , Poland

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Krakow, , Poland

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Lodz, , Poland

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Puławy, , Poland

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Sopot, , Poland

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Szczecin, , Poland

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Warsaw, , Poland

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Wroclaw, , Poland

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Bucharest, , Romania

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Oradea, , Romania

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Romania, , Romania

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Timișoara, , Romania

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Irkutsk, , Russia

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Kazan', , Russia

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Moscow, , Russia

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Moscva, , Russia

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Novosibirsk, , Russia

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Rostov-on-Don, , Russia

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Ryazan, , Russia

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Saint Petersburg, , Russia

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Stavropol, , Russia

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Ufa, , Russia

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Belgrade, , Serbia

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Kragujevac, , Serbia

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Niš, , Serbia

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Vojvodina, , Serbia

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Bratislava, , Slovakia

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Prešov, , Slovakia

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Daegu, , South Korea

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Guri-si, , South Korea

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Seoul, , South Korea

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Suwon, , South Korea

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Chernivtsi, , Ukraine

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Dnipropetrovsk, , Ukraine

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Ivano-Frankivsk, , Ukraine

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Kharkiv, , Ukraine

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Kiyv, , Ukraine

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Kyiv, , Ukraine

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Lviv, , Ukraine

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Odesa, , Ukraine

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Sumy, , Ukraine

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Uzhhorod, , Ukraine

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Vinnytsia, , Ukraine

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Zaporizhzhia, , Ukraine

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Zhaporozhia, , Ukraine

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Birmingham, , United Kingdom

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Cambridge, , United Kingdom

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Coventry, , United Kingdom

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Doncaster, , United Kingdom

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Edinburgh, , United Kingdom

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Liverpool, , United Kingdom

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London, , United Kingdom

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Salford, , United Kingdom

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Southampton, , United Kingdom

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Sutton in Ashfield, , United Kingdom

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Countries

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United States Australia Austria Belgium Bulgaria Canada Czechia Denmark France Germany Hungary Israel Japan Netherlands New Zealand Poland Romania Russia Serbia Slovakia South Korea Ukraine United Kingdom

References

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Danese S, Leong RW, Sands BE, Ma T, Marano C, Peyrin-Biroulet L. Clinical Trial: Association Between Early Disease Clearance and Long-Term Outcomes-4-Year Results From the Phase 3 UNIFI Study of Ustekinumab in Ulcerative Colitis. Aliment Pharmacol Ther. 2025 Sep;62(5):483-492. doi: 10.1111/apt.70264. Epub 2025 Jul 16.

Reference Type DERIVED
PMID: 40668079 (View on PubMed)

Solitano V, Panaccione R, Sands BE, Wang Z, Hogan M, Zou G, Peyrin-Biroulet L, Danese S, Cornfield LJ, Feagan BG, Singh S, Jairath V, Ma C. Responsiveness of different disease activity indices in moderate-to-severe ulcerative colitis. Med. 2025 Feb 14;6(2):100512. doi: 10.1016/j.medj.2024.09.001. Epub 2024 Oct 4.

Reference Type DERIVED
PMID: 39368474 (View on PubMed)

Ghosh S, Feagan BG, Ott E, Gasink C, Godwin B, Marano C, Miao Y, Ma T, Loftus EV Jr, Sandborn WJ, Danese S, Abreu MT, Sands BE. Safety of Ustekinumab in Inflammatory Bowel Disease: Pooled Safety Analysis Through 5 Years in Crohn's Disease and 4 Years in Ulcerative Colitis. J Crohns Colitis. 2024 Aug 6;18(7):1091-1101. doi: 10.1093/ecco-jcc/jjae013.

Reference Type DERIVED
PMID: 38310565 (View on PubMed)

Afif W, Arasaradnam RP, Abreu MT, Danese S, Sandborn WJ, Miao Y, Zhang H, Panaccione R, Hisamatsu T, Scherl EJ, Leong RW, Rowbotham DS, Peyrin-Biroulet L, Sands BE, Marano C. Efficacy and Safety of Ustekinumab for Ulcerative Colitis Through 4 Years: Final Results of the UNIFI Long-Term Maintenance Study. Am J Gastroenterol. 2024 May 1;119(5):910-921. doi: 10.14309/ajg.0000000000002621. Epub 2023 Dec 14.

Reference Type DERIVED
PMID: 38095692 (View on PubMed)

Chen R, Li L, Tie Y, Chen M, Zhang S. Trajectory of fecal lactoferrin for predicting prognosis in ulcerative colitis. Precis Clin Med. 2023 Sep 5;6(3):pbad022. doi: 10.1093/pcmedi/pbad022. eCollection 2023 Sep.

Reference Type DERIVED
PMID: 38025971 (View on PubMed)

Danese S, Sands BE, Abreu MT, O'Brien CD, Bravata I, Nazar M, Miao Y, Wang Y, Rowbotham D, Leong RWL, Arasaradnam RP, Afif W, Marano C. Early Symptomatic Improvement After Ustekinumab Therapy in Patients With Ulcerative Colitis: 16-Week Data From the UNIFI Trial. Clin Gastroenterol Hepatol. 2022 Dec;20(12):2858-2867.e5. doi: 10.1016/j.cgh.2022.02.050. Epub 2022 Mar 8.

Reference Type DERIVED
PMID: 35276329 (View on PubMed)

Panaccione R, Danese S, Sandborn WJ, O'Brien CD, Zhou Y, Zhang H, Adedokun OJ, Tikhonov I, Targan S, Abreu MT, Hisamatsu T, Scherl EJ, Leong RW, Rowbotham DS, Arasaradnam RP, Sands BE, Marano C. Ustekinumab is effective and safe for ulcerative colitis through 2 years of maintenance therapy. Aliment Pharmacol Ther. 2020 Dec;52(11-12):1658-1675. doi: 10.1111/apt.16119. Epub 2020 Oct 21.

Reference Type DERIVED
PMID: 33086438 (View on PubMed)

Sandborn WJ, Feagan BG, Danese S, O'Brien CD, Ott E, Marano C, Baker T, Zhou Y, Volger S, Tikhonov I, Gasink C, Sands BE, Ghosh S. Safety of Ustekinumab in Inflammatory Bowel Disease: Pooled Safety Analysis of Results from Phase 2/3 Studies. Inflamm Bowel Dis. 2021 Jun 15;27(7):994-1007. doi: 10.1093/ibd/izaa236.

Reference Type DERIVED
PMID: 32964215 (View on PubMed)

Li K, Marano C, Zhang H, Yang F, Sandborn WJ, Sands BE, Feagan BG, Rubin DT, Peyrin-Biroulet L, Friedman JR, De Hertogh G. Relationship Between Combined Histologic and Endoscopic Endpoints and Efficacy of Ustekinumab Treatment in Patients With Ulcerative Colitis. Gastroenterology. 2020 Dec;159(6):2052-2064. doi: 10.1053/j.gastro.2020.08.037. Epub 2020 Aug 25.

Reference Type DERIVED
PMID: 32853634 (View on PubMed)

Sands BE, Sandborn WJ, Panaccione R, O'Brien CD, Zhang H, Johanns J, Adedokun OJ, Li K, Peyrin-Biroulet L, Van Assche G, Danese S, Targan S, Abreu MT, Hisamatsu T, Szapary P, Marano C; UNIFI Study Group. Ustekinumab as Induction and Maintenance Therapy for Ulcerative Colitis. N Engl J Med. 2019 Sep 26;381(13):1201-1214. doi: 10.1056/NEJMoa1900750.

Reference Type DERIVED
PMID: 31553833 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2014-005606-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CNTO1275UCO3001

Identifier Type: OTHER

Identifier Source: secondary_id

CR106920

Identifier Type: -

Identifier Source: org_study_id

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