Phase 3 Multicenter Open-Label Study on Long-Term Efficacy and Safety of Mirikizumab in Pediatric Patients with Moderate-to-Severe Ulcerative Colitis or Crohn's Disease

The primary objective of this study is to evaluate the **efficacy** of **mirikizumab** in pediatric participants with **Crohn's disease** and **ulcerative colitis**. This is clinically relevant as it aims to assess the therapeutic potential of mirikizumab in managing these chronic inflammatory bowel diseases in a pediatric population, which can significantly impact the quality of life and long-term health outcomes for affected children and adolescents.

The clinical trial involves a total of **21 participants** diagnosed with conditions such as **Ulcerative Colitis**, **Chronic Ulcerative Colitis**, **Inflammatory Bowel Diseases**, and **Crohn's Disease**. The study population includes both male and female pediatric subjects, specifically within the age ranges of 2 to 3 years. Participants were selected from originating studies, and they are considered to potentially benefit from further treatment with mirikizumab. The trial includes a vulnerable population, indicating special considerations for the participants' health and safety. Lifestyle factors such as diet, physical activity, or habits are not specified in the available data. The selection criteria emphasize the continuation of treatment from prior studies, with female participants required to adhere to contraception requirements.

The clinical trial is a Phase 3, multicenter, open-label, long-term extension study designed to evaluate the long-term efficacy and safety of **mirikizumab** in pediatric participants with moderate-to-severe **ulcerative colitis** or **Crohn's disease**. The trial employs a non-randomized, open-label design, allowing all participants to receive the investigational product, mirikizumab, administered as a **solution for injection**. The study is expected to commence recruitment on January 1, 2025, and conclude by July 30, 2030, with the primary objective of assessing clinical remission in participants over a 52-week period.

Participants eligible for inclusion are those who have completed assessments and procedures in prior studies and are deemed by the investigator to benefit from continued treatment with mirikizumab. The study visits are structured as follows: an initial screening visit to confirm eligibility, followed by regular follow-up visits to monitor efficacy and safety, and a final end-of-study visit to assess long-term outcomes. The expected duration of participant involvement is approximately 52 weeks, with conditions for early termination including withdrawal of consent, adverse events, or investigator decision based on clinical judgment.

The primary endpoints focus on the percentage of participants achieving clinical remission, as measured by the Modified Mayo Score for ulcerative colitis and the Pediatric Crohn's Disease Activity Index for Crohn's disease, both evaluated at week 52. The study does not include a control group, and all participants will receive the investigational product throughout the trial duration. The trial is not categorized as low intervention, given its Phase 3 status and the investigational nature of the treatment for the specified indications.

The clinical trial involves the administration of **Mirikizumab**, an experimental medication developed by Eli Lilly and Company Limited. Mirikizumab is provided in the form of a **solution for injection**. The active substance, mirikizumab, is a protein of non-specific origin. The pharmaceutical form is specifically designed for injection or infusion, allowing for flexibility in administration. The medication is not formulated for pediatric use, and it is not classified as an orphan drug. The maximum treatment period for the trial is set at one unit of time, with no specified maximum daily or total dose amount. The sponsor product code for Mirikizumab is LY3074828.

In this study, no non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatments are utilized. The trial is focused solely on evaluating the efficacy and safety of Mirikizumab in pediatric participants with moderate-to-severe ulcerative colitis or Crohn's disease. The administration of the drug is monitored to ensure compliance, although specific compliance monitoring strategies are not detailed in the provided data. The trial does not involve any additional medicinal products or devices with a CE mark.

The efficacy of **mirikizumab** in the clinical trial will be assessed through primary endpoints that focus on clinical remission rates in pediatric participants with moderate-to-severe **ulcerative colitis** (UC) and **Crohn's disease** (CD). For participants with UC, efficacy will be evaluated by the percentage of participants achieving clinical remission based on the Modified Mayo Score (MMS) at Week 52. Similarly, for participants with CD, the efficacy will be determined by the percentage of participants achieving clinical remission based on the Pediatric Crohn’s Disease Activity Index (PCDAI) at Week 52.