Table of contents

• Trial overview
• Conditions studied
• Who the studies include
• Trial phases and study design
• Main endpoints and what they mean
• Special study types and comparisons
• Pediatric studies

Trial overview

The trial data show that Ustekinumab is being studied in many interventional clinical trials, mostly in Phase 3 and some in Phase 2 or long-term safety settings.^[1] These studies are authorised or completed and focus on diseases where the immune system plays a major role.^[1]

The main purpose of these trials is to measure whether treatment strategies that include Ustekinumab can improve disease control, reduce symptoms, and support remission in adults and children.^[1]

Conditions studied

Ustekinumab trials in the data include psoriatic arthritis, Crohn’s disease, ulcerative colitis, plaque psoriasis, juvenile psoriatic arthritis, and folliculitis decalvans.^[1] Some studies focus on active disease, while others focus on stable disease, long-term follow-up, or disease that came back after earlier treatment or surgery.^[1]

Several trials also study inflammatory bowel disease in special situations, such as after bowel surgery or after loss of response to earlier Ustekinumab treatment.^[1]

Who the studies include

The target populations are mostly adults with active or moderate to severe disease, but some studies are designed for children and teenagers aged 2 to under 18 years or 6 to under 18 years.^[1] Some trials include people who are biologic-naïve, meaning they have not used biologic treatment before, while others include patients who already tried other treatments and still have active disease.^[1]

Other trials include people with stable minimal disease activity, people with refractory disease, meaning disease that is hard to treat, and patients with Crohn’s disease who had surgery and have risk factors for recurrence.^[1]

Trial phases and study design

Most studies are Phase 3 trials, which are larger studies that compare treatment effects and safety in more patients.^[1] The data also include Phase 2 studies, which are earlier studies that look for a first signal of benefit and collect safety information.^[1]

Some studies are open-label, meaning everyone knows the treatment being used, and some are randomised, meaning participants are assigned to groups by chance.^[1] There is also a low-intervention long-term safety study and an open-label extension study, which follows patients for a longer time after earlier trials.^[1]

Main endpoints and what they mean

The trials measure different endpoints, or study results, depending on the disease.^[1] In psoriatic arthritis, one study measures minimal disease activity and PASDAS at 12 months, while another measures ACR 20 response at Week 16.^[1]

In Crohn’s disease and ulcerative colitis, common endpoints include clinical remission, endoscopic remission, corticosteroid-free remission, and changes in scores such as CDAI, SES-CD, and PRO-2.^[1] In psoriasis studies, common endpoints include PASI 90, PASI 75, and IGA 0/1, which show how much the skin improved.^[1]

Some studies also measure safety outcomes, such as adverse events, serious adverse events, laboratory tests, injection-site reactions, and treatment changes because of loss of response.^[1]

Special study types and comparisons

Several trials compare Ustekinumab with other advanced therapies such as infliximab, vedolizumab, risankizumab, guselkumab, deucravacitinib, and other biologic or targeted treatments.^[1] Some studies test whether combination therapy works better than Ustekinumab alone, especially in Crohn’s disease and ulcerative colitis.^[1]

Other studies use Ustekinumab as an active control, which means it is the standard treatment used for comparison against a newer medicine.^[1] A few studies also look at therapeutic drug monitoring, which means measuring drug levels to guide treatment decisions.^[1]

Pediatric studies

Some of the most important Ustekinumab trials in the data are pediatric studies for children and teenagers with moderate to severe plaque psoriasis, moderately to severely active Crohn’s disease, moderately to severely active ulcerative colitis, and juvenile psoriatic arthritis.^[1] These studies look at both efficacy and safety, and some also measure pharmacokinetics, which means how the body handles the treatment over time.^[1]

Long-term extension studies in pediatric participants are also included to collect safety data over time and to follow patients who had already been in earlier clinical studies.^[1]