Guselkumab for Pediatric Ulcerative Colitis

(QUASAR Jr Trial)

Recruiting at 89 trial locations

Overseen By Study Contact

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Janssen Research & Development, LLC

Disqualifiers: Severe colitis, Crohn's disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

120 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of the drug guselkumab (Tremfya) for children with moderate to severe ulcerative colitis, a condition causing inflammation and sores in the colon. The study aims to determine if guselkumab can maintain improvement after an initial response. Participants will receive different doses, either through injections or infusions, to identify the most effective method. Children with long-standing ulcerative colitis who have not responded well to other treatments might be suitable for this trial. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to help bring a new treatment to market.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that guselkumab is generally well-tolerated. In studies involving children with moderate to severe ulcerative colitis, the safety of guselkumab over 52 weeks matched that observed in adults and older children, indicating no unexpected safety issues for this age group.

Other studies found that fewer patients taking guselkumab reported side effects compared to those on a placebo. Specifically, 42% of patients on guselkumab reported side effects, while 68% of those on placebo did.

The FDA has approved this treatment for other conditions, suggesting a known safety record. However, individual experiences may vary. Always discuss any concerns with a doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ulcerative colitis?

Researchers are excited about guselkumab for pediatric ulcerative colitis because it offers a different approach compared to standard treatments like aminosalicylates, corticosteroids, and biologics targeting TNF-alpha. Unlike these treatments, guselkumab specifically targets interleukin-23 (IL-23), a key player in the inflammatory process, potentially providing a more precise method of reducing inflammation. Additionally, guselkumab's flexibility in administration, with both intravenous (IV) and subcutaneous (SC) options, allows for personalized treatment strategies based on patient needs and preferences. This unique mechanism and delivery method could offer new hope for better managing ulcerative colitis in children.

What evidence suggests that guselkumab might be an effective treatment for pediatric ulcerative colitis?

Research has shown that guselkumab, also known as TREMFYA, may help treat ulcerative colitis in children. In one study, 23% of children who took guselkumab were symptom-free after 12 weeks. Another study found that 36.7% of children experienced both symptom relief and healthier-looking colon tissue after using guselkumab for 48 weeks. Guselkumab targets a protein called IL-23, which is involved in inflammation, helping to reduce symptoms of ulcerative colitis. This makes guselkumab a promising option for children with this condition. Participants in this trial will receive different dosing regimens of guselkumab, either intravenously or subcutaneously, during the induction and maintenance phases. ^{2 3 6 7 8}

Who Is on the Research Team?

Janssen Research & Development, LLC Clinical Trial

Principal Investigator

Janssen Research & Development, LLC

Are You a Good Fit for This Trial?

This trial is for children and teenagers with moderately to severely active ulcerative colitis, a type of inflammatory bowel disease. Participants must have shown some response to earlier treatment phases to qualify.

Inclusion Criteria

I have a confirmed diagnosis of ulcerative colitis with biopsy proof.

My ulcerative colitis is moderate to severe, confirmed by specific tests.

My health is stable according to my recent medical check-up.

Exclusion Criteria

My ulcerative colitis affects only my rectum or less than 20cm of my colon.

I have a stoma.

I have not had bowel surgery in the last 6 months or any abdominal surgery in the last 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Induction

Participants receive guselkumab intravenously or subcutaneously based on their body weight during the 12-week open-label induction phase

12 weeks

Double-blind Maintenance

Week 12 responders are randomized to receive guselkumab dose regimen 1 or 2 subcutaneously based on their body weight up to Week 56

44 weeks

Open-label Maintenance

Week 12 non-responders enter an open-label maintenance phase to receive guselkumab subcutaneously based on their body weight up to Week 56

44 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Guselkumab

Trial Overview

The study tests the effectiveness of Guselkumab, a medication, in young patients with ulcerative colitis. It compares Guselkumab against a placebo (a substance with no therapeutic effect) over time.

How Is the Trial Designed?

Group I: Open-label Maintenance Phase: Guselkumab SC Experimental Treatment 1 Intervention

Week 12 non-responders will not be randomized and will enter an open-label maintenance phase to receive guselkumab SC dosing regimen based on their body weight up to Week 56.

Group II: Open-label Induction Phase: Guselkumab Subcutaneously (SC) Experimental Treatment 1 Intervention

Participants will receive a guselkumab dose SC based on their BW during the 12-week open-label induction phase.

Group III: Open-label Induction Phase: Guselkumab Intravenously (IV) Experimental Treatment 1 Intervention

Participants will receive a guselkumab dose IV based on their body weight (BW) during the 12-week open-label induction phase.

Group IV: Double-blind Maintenance Phase: Guselkumab Dose Regimen 2 Experimental Treatment 1 Intervention

At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 2 SC based on their BW up to Week 56.

Group V: Double-blind Maintenance Phase: Guselkumab Dose Regimen 1 Experimental Treatment 1 Intervention

At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 1 SC based on their BW up to Week 56.

Guselkumab is already approved in United States, European Union for the following indications:

Approved in United States as Tremfya for:

• Moderate to severe plaque psoriasis
• Psoriatic arthritis
• Moderately to severely active ulcerative colitis

Approved in European Union as Tremfya for:

• Moderate to severe plaque psoriasis
• Psoriatic arthritis
• Moderately to severely active ulcerative colitis

Who Is Running the Clinical Trial?

Janssen Research & Development, LLC

Lead Sponsor

Trials

1,022

Recruited

6,408,000+

Joaquin Duato

Janssen Research & Development, LLC

Chief Executive Officer since 2022

MBA from ESADE, Master of International Management from Thunderbird School of Global Management

Dr. Jijo James, MD

Janssen Research & Development, LLC

Chief Medical Officer since 2014

MD from St. Johns Medical College, MPH from Columbia University

Published Research Related to This Trial

In a study of 337 pediatric patients with ulcerative colitis followed for a median of 7.2 years, the introduction of immunosuppressants and anti-TNF therapies significantly reduced the risk of colectomy from 18% in the pre-anti-TNF era to 9% in the anti-TNF era.

Despite the increased use of these therapies, the risk of flare-related hospitalizations rose from 23% to 42%, indicating that while treatment has improved surgical outcomes, it may not fully prevent disease exacerbations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Therapeutic Strategies Are Associated With a Significant Decrease in Colectomy Rate in Pediatric Ulcerative Colitis. Ley, D., Leroyer, A., Dupont, C., et al.[2023]

Tofacitinib, a small molecule JAK inhibitor, was successfully used to treat a 13-year-old girl with severe ulcerative colitis (UC) who had not responded to other biologic therapies, leading to complete clinical and endoscopic remission.

This case suggests that tofacitinib may be a safe and effective treatment option for pediatric patients with refractory UC, potentially reducing the need for surgical interventions like proctocolectomy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib for a Child with Refractory Steroid-Dependent Ulcerative Colitis: A Case Report and Review of the Literature. Alajmi, R., Alabdulhadi, M., Alali, AA., et al.[2021]

In the QUASAR Phase 2b Induction Study, guselkumab, an interleukin-23p19 antagonist, demonstrated significant efficacy in treating moderately to severely active ulcerative colitis, with 61.4% and 60.7% of patients achieving clinical response at week 12 for the 200 mg and 400 mg doses, respectively, compared to only 27.6% for placebo.

The safety profile of guselkumab was comparable to that of the placebo, indicating that it is a safe treatment option for patients who have not responded adequately to other therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Guselkumab in Patients With Moderately to Severely Active Ulcerative Colitis: QUASAR Phase 2b Induction Study. Peyrin-Biroulet, L., Allegretti, JR., Rubin, DT., et al.[2023]

Children with ulcerative colitis often require more aggressive treatments like immunomodulators or biologics, as standard mesalamine therapy is less effective, achieving remission in less than 50% of cases.

The review highlights the use of biologic therapies such as infliximab, adalimumab, golimumab, and vedolizumab in pediatric patients, emphasizing the importance of therapeutic drug monitoring for optimizing treatment plans, despite a lack of extensive controlled clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Special considerations for biologic medications in pediatric ulcerative colitis. Jerger, L., Hyams, JS.[2020]

Infliximab was found to be effective in inducing a response in 73.3% of children aged 6-17 with moderately to severely active ulcerative colitis who had not responded to conventional treatments, as measured by significant decreases in Mayo scores at week 8.

The study indicated that maintenance therapy with infliximab every 8 weeks led to a higher remission rate at week 54 compared to every 12 weeks, although both regimens had similar safety profiles with no serious adverse events reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Induction and maintenance therapy with infliximab for children with moderate to severe ulcerative colitis. Hyams, J., Damaraju, L., Blank, M., et al.[2022]

In a study of 35 pediatric patients aged 6 to 17 with moderately-to-severely active ulcerative colitis, subcutaneous golimumab showed promising clinical benefits, with 60% achieving a clinical response and 54% achieving mucosal healing by week 6.

The pharmacokinetics of golimumab in these children were comparable to those observed in adults, and no significant safety concerns were reported, suggesting it may be a safe and effective treatment option for this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subcutaneous Golimumab in Pediatric Ulcerative Colitis: Pharmacokinetics and Clinical Benefit. Hyams, JS., Chan, D., Adedokun, OJ., et al.[2018]

In a study involving 214 adults with moderately-to-severely active ulcerative colitis, combination therapy with guselkumab and golimumab resulted in a higher clinical response rate at week 12 (83%) compared to golimumab monotherapy (61%) and guselkumab monotherapy (75%).

The safety profile was generally acceptable, with no deaths or serious complications reported during the treatment period, although some adverse events like infections and anemia were noted, indicating the need for monitoring in future studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Guselkumab plus golimumab combination therapy versus guselkumab or golimumab monotherapy in patients with ulcerative colitis (VEGA): a randomised, double-blind, controlled, phase 2, proof-of-concept trial. Feagan, BG., Sands, BE., Sandborn, WJ., et al.[2023]

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U.S. FDA approves TREMFYA® (guselkumab) for the ...

Safety data through Week 16 showed that 42% of patients receiving guselkumab and 68% of patients receiving placebo reported adverse events (AEs) ...