A Phase 3 Multicenter Study to Evaluate Efficacy, Safety, and Pharmacokinetics of an Investigational drug with Open-Label Induction, Randomized, Double-Blind Maintenance and Open-Label Long-Term Extension in Pediatric Subjects with Moderately to Severely
Published: 04/08/2025
General Information:
The study objective is to assess the efficacy, safety, and pharmacokinetics of an investigational drug in pediatric subjects from 2 to < 18 years of age with moderately to severely active Crohn’s disease (CD) who have had inadequate responses, loss of response, or intolerance to corticosteroids, immunosuppressants, and/or biologics, or in whom use of those therapies is medically contraindicated.
Eligibility:
Key Eligibility Criteria
- Male or female individuals, age 2 to < 18 years old at Baseline.
- Patient must have total body weight at Screening and Baseline of ≥ 10 kg.
- Documented diagnosis of CD prior to Baseline, confirmed by colonoscopy during the screening period, with exclusion of current infection, colonic dysplasia and/or malignancy. Appropriate documentation of biopsy results consistent with the diagnosis of CD, in the assessment of the investigator, must be available.
- Moderate to severe CD as defined as
- PCDAI > 30
- Evidence of mucosal inflammation, defined as SES-CD ≥ 6 (≥ 4 for subjects with isolated ileal disease), excluding the presence of narrowing component, as confirmed by the central reader.
- PCDAI > 30
- Demonstrated an inadequate response, loss of response, or intolerance to corticosteroids, immunomodulators, and/or biologic therapy or in whom use of those therapies is medically contraindicated. For patients in the US, subjects must have demonstrated an inadequate response, loss or response, or intolerance to one or more anti-TNFs.
Study Details:
Study description/explanation of participation:
If you take part of M14-671 study, you will receive a higher dose in the 12-week induction phase and one of 2 doses of active drug in the 52-week maintenance phase. Endoscopic evaluation will also be included. There is no placebo arm. Your study participation could be a maximum of 220 weeks depending on how you respond to the study drug, plus a 30 day follow up visit after your last dose of study drug.
Description of treatment or intervention (mechanism of action):
The study drug works with your body to fight the inflammation that can lead to inflammatory bowel disease symptoms. The amount of inflammation is controlled by many different proteins that are found naturally within the body. The study drug may reduce the activity of some of those proteins. The study drug has been tested in clinical trialswith different inflammatory diseases. Either an oral tablet will be taken once daily, or a liquid formulation will be taken twice daily.
Patient Participation Requirements:
●attend all study visits
●tell the study doctor if you are feeling bad or worse than before
●tell the study doctor if you have any changes in medications during the study
●follow the directions of the study doctor and research team
●refrain from participation in other research studies while you are a participant in this study
●fill out the electronic questionnaires and electronic diary completely and honestly and bring the electronic diary to the study doctor's office at each visit
●carry your patient card with you as long as you are in the study and show it to any medical staff that may be involved in your healthcare
●you must return all unused study drug. The study drug is only to be taken by you (the research participant). It is important that you keep unused study drug out of the reach of other family or household members (unless they are administering drug to you)
Possible risks and side effects:
The study drug is being investigated to treat Crohn’s disease in the pediatric population. The study drug has been tested for different inflammatory diseases but is not approved to treat pediatric Crohn’s disease. Therefore, the use of the study drug is investigational (experimental) for the purposes of this study. The clinical trial investigator will review with you all the known risks and side effects to help you determine if you would like to participate in the trial.
Progress to date:
Phase 3 induction and maintenance studies in adult Crohn’s disease population complete with an adult Long Term Extension study ongoing.