SUMMARY  

• The company cannot recommend any practices, procedures, or usage that deviate from the approved labeling.
• Ongoing phase 3 randomized clinical trials are evaluating the efficacy and safety of TREMFYA in pediatric patients with moderately to severely active ulcerative colitis (UC) or Crohn's disease (CD). Additional details regarding these trials are available at clinicaltrials.gov: NCT06260163 & NCT05923073

clinical data

Ulcerative Colitis (QUASAR Jr): The efficacy, safety, and pharmacokinetics of TREMFYA are currently being evaluated in pediatric patients (ages 2-17 years) with moderately to severely active UC in a phase 3, randomized, open-label induction, double-blind maintenance, parallel-group, multicenter study.¹

Study Design/Methods

• Please refer to clinicaltrials.gov for inclusion and exclusion criteria.

Treatment Arms

Open-label Induction Phase

• TREMFYA intravenously (IV)
  • TREMFYA IV dose based on body weight (BW) during the 12-week open-label induction phase.
• TREMFYA subcutaneously (SC)
  • TREMFYA SC dose based on BW during the 12-week open-label induction phase.

Double-blind Maintenance Phase

• TREMFYA Dose Regimen 1
  • At the end of the induction phase, week 12 responders will be randomized into the double-blind maintenance phase to receive TREMFYA SC dose regimen 1 based on their BW up to Week 56.
• TREMFYA Dose Regimen 2
  • At the end of the induction phase, week 12 responders will be randomized into the double-blind maintenance phase to receive TREMFYA dose regimen 2 based on their BW up to Week 56.

Open-label Maintenance Phase

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

Primary outcome

• Clinical remission at week 56.
  • Clinical remission is defined per modified Mayo score as a stool frequency of 0 or 1 and not increased from induction baseline, a rectal bleeding score of 0, and endoscopy subscore of 0 or 1 with no friability present on the endoscopy.

Crohn’s disease (MACARONI-23): The clinical and endoscopic efficacy of TREMFYA is currently being evaluated in pediatric patients (ages 2-17 years) with moderately to severely active CD in a phase 3, multicenter, randomized, platform study.²

Study Design/Methods

• Please refer to clinicaltrials.gov for inclusion and exclusion criteria.

Treatment Arms

 Open-label induction phase

• TREMFYA IV
  • TREMFYA IV dose based on BW during the 12-week open-label induction phase.
•  TREMFYA SC
  • TREMFYA SC dose based on BW during the 12-week open-label induction phase.

Double-blind maintenance phase:

• TREMFYA SC or TREMFYA SC and Placebo SC
  • At the end of the induction phase, week 12 responders will be randomized into the double-blind maintenance phase to receive a TREMFYA SC dose based on their BW or a TREMFYA SC dose based on their BW and placebo SC up to Week 48.

Open-label maintenance phase:

• TREMFYA SC
  • Week 12 non-responders will enter open-label maintenance phase to receive TREMFYA SC dosing regimen based on their BW up to Week 48.

Primary Outcomes

• Clinical remission at week 52.
• Endoscopic response at week 52.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, DERWENT^® (and/or other resources, including internal/external databases) was conducted on 25 September 2024.