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
Published: 10/21/2024
General Information:
The objectives of this study are to evaluate the efficacy, safety, and pharmacokinetics of investigational drug in inducing and maintaining clinical remission in pediatric patients 2 to 17 years of age with moderately to severely active UC, who have had an inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.
Eligibility:
Key Eligibility Criteria
- Patient is male or female, age 2 to 17 years old at Baseline. If a patient turns 18 years old at any point after Baseline, he or she may continue in the study until the completion of the OLE Period 2.
- Patient must have total body weight at time of screening and Baseline of ≥ 10 kg.
- Documented diagnosis of UC 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 UC, in the assessment of the investigator, must be available from either the screening endoscopy or prior endoscopy within 90 days.
- Active UC with an AMS of 5 to 9 points and endoscopic subscore of 2 to 3 (confirmed by central reader).
- Demonstrated an inadequate response, loss of response, or intolerance (or have medical contraindications) to at least one of the following treatments including corticosteroids, immunosuppressants, and/or biologic therapy, in the opinion of the investigator, as defined below. For patients in the US, patients must have demonstrated an inadequate response, loss of response, or intolerance to one or more TNF inhibitors.
Study Details:
Study description/explanation of participation:
If you take part of M14-658 study, you will receive a higher dose in the 8 week induction phase and one of 2 doses of active drug in the 44 week maintenance phase. Endoscopic evaluation will also be included There is no placebo arm. Your study participation could be a maximum of 325 weeks depending on how you respond to the study drug, plus a 30 day follow up visit after your last dose.
Description of treatment or intervention (mechanism of action):
The study drug works with your body to fight the inflammation that can lead to IBD 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 trials for different inflammatory diseases. Either an oral tablet taken once daily or 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 dug is being investigated to treat pediatric ulcerative colitis. The study drug has been tested for different inflammatory diseases but is not approved to treat pediatric Ulcerative Colitis. 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 with you to help you determine if you would like to participate in the trial.
Progress to date:
Phase 3 Induction and maintenance studies in adult population complete with an adult Long Term Extension study ongoing