Evaluation of Long-Term Safety and Tolerability of Brivaracetam in Pediatric Patients with Childhood or Juvenile Absence Epilepsy

The primary objective of this study is to investigate the long-term **safety** and **tolerability** of **brivaracetam** in pediatric participants diagnosed with **Childhood Absence Epilepsy** (CAE) or **Juvenile Absence Epilepsy** (JAE). This is clinically relevant as it aims to ensure that the therapeutic use of brivaracetam is safe and well-tolerated over extended periods, which is crucial for managing these chronic conditions in a young population. The study does not list any secondary objectives.

The clinical trial involves a total of **39 participants** diagnosed with either **Juvenile Absence Epilepsy** or **Childhood Absence Epilepsy**. The study population includes both male and female subjects, with an age range corresponding to categories 2 and 3, which typically encompass children and adolescents. Participants were selected based on their previous involvement in studies EP0132 and/or N01269, and their eligibility for entry into the current study, EP0224, as per the protocols of the aforementioned studies. The trial population is characterized by individuals for whom a reasonable benefit from the long-term administration of **brivaracetam** is anticipated. The study includes a vulnerable population, and participants are required to provide informed consent or assent, with additional consent from a parent, legal representative, or caregiver for minors. The trial does not specify particular lifestyle considerations such as diet or physical activity. The inclusion of both genders and the focus on a pediatric population are significant aspects of this study.

The clinical trial is designed to evaluate the long-term safety and tolerability of **brivaracetam** in participants with **childhood absence epilepsy** or **juvenile absence epilepsy**. This is a multicenter, open-label, single-arm study. The trial is expected to commence recruitment on June 30, 2024, and conclude by March 1, 2030. Participants will be administered brivaracetam either as an oral solution or film-coated tablet, with a maximum daily dose of 200 mg and a total dose not exceeding 100 mg. The treatment period is set for a maximum of 36 months.

Participants eligible for this study are those who have previously participated in specific trials (EP0132 and/or N01269) and meet the inclusion criteria, which include a confirmed diagnosis of childhood or juvenile absence epilepsy. The study will involve several visits, starting with a screening visit to confirm eligibility. Follow-up visits will be scheduled to monitor the incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), as well as to assess the overall safety and tolerability of the treatment. The primary endpoints focus on the incidence of TEAEs and TEAEs leading to discontinuation of the investigational medicinal product. Secondary endpoints include the incidence of serious adverse events and IMP-related TEAEs.

The expected length of participant involvement is up to 36 months, with conditions for early termination including the occurrence of significant adverse events or if the investigator deems it necessary for the participant's safety. Participants must agree to use contraception during the treatment period and for a specified duration after the final dose. The study aims to provide valuable data on the long-term use of brivaracetam in the specified population, contributing to the understanding of its safety profile in treating nervous system disorders.

The clinical trial involves the administration of **brivaracetam**, a chemical compound developed by UCB Biopharma SRL, to evaluate its long-term safety and tolerability in pediatric participants with childhood absence epilepsy or juvenile absence epilepsy. The experimental medication is available in two pharmaceutical forms: an **oral solution** and a **film-coated tablet**. The oral solution is designed for ease of administration, particularly in populations that may have difficulty swallowing tablets. The film-coated tablets provide an alternative form for those who prefer or require a solid dosage form. Both forms are administered orally.

The maximum daily dose of **brivaracetam** is 200 mg, with a total maximum dose of 100 mg per administration. The treatment period extends up to 36 months, allowing for comprehensive evaluation of long-term effects. The dosing schedule is designed to maintain therapeutic levels of the drug while minimizing potential side effects. Participant compliance is monitored through regular follow-ups and assessments to ensure adherence to the dosing regimen.

In this study, **brivaracetam** is the sole investigational product, and no non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatments are utilized. The focus is solely on assessing the safety and tolerability of **brivaracetam** in the specified patient population. The trial does not include any additional medicinal products or devices, and all substances involved are of chemical origin. The study is structured as a multicenter, open-label, single-arm trial, emphasizing the collection of safety data over an extended period.

The efficacy of **brivaracetam** in the clinical trial will be assessed through the evaluation of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the study. The primary endpoints include the incidence of TEAEs and the incidence of TEAEs leading to discontinuation of the investigational medicinal product (IMP). Secondary endpoints focus on the incidence of serious adverse events and IMP-related TEAEs. These parameters will be systematically collected and analyzed to determine the safety and tolerability of **brivaracetam** in participants with childhood absence epilepsy (CAE) or juvenile absence epilepsy (JAE).