Long-Term Safety and Tolerability Evaluation of Bepranemab in Patients with Progressive Supranuclear Palsy: An Open-Label Extension Study

The primary objective of this study is the assessment of the long-term **safety** and **tolerability** of UCB0107, also known as **bepranemab**, in participants diagnosed with **Progressive Supranuclear Palsy** (PSP). This objective is clinically relevant as it aims to determine the sustained safety profile of bepranemab, which is crucial for its potential use as a therapeutic option in managing PSP, a neurodegenerative disorder characterized by motor and cognitive impairments. The study will provide valuable data on the long-term effects of the drug, which is administered as a solution for infusion via intravenous use.

The clinical trial involves a total of **2 participants** diagnosed with **Progressive Supranuclear Palsy** (PSP). The study population includes both male and female subjects, with an age range that corresponds to categories 3 and 4, indicating adult and elderly participants. The trial population was selected based on specific criteria, including the completion of a prior treatment period in PSP003 and the ability to provide informed consent. Participants are required to adhere to contraceptive guidelines if applicable. The study does not specify particular lifestyle considerations such as diet or physical activity. The trial includes a vulnerable population, emphasizing the need for careful monitoring and ethical considerations throughout the study.

The clinical trial is designed to evaluate the long-term safety and tolerability of **bepranemab** in participants with **Progressive Supranuclear Palsy** (PSP). This is a Phase 3, open-label extension study, which means that all participants will receive the investigational product, and both the researchers and participants will know the treatment being administered. The trial is expected to run from November 16, 2020, to March 30, 2027, with a maximum treatment period of 144 weeks for each participant. The study involves the administration of bepranemab as a **solution for infusion** via **intravenous use**.

Participants will undergo a series of study visits, beginning with an inclusion (screening) visit to confirm eligibility based on criteria such as a diagnosis of possible or probable PSP Richardson's Syndrome and completion of a prior treatment period in a related study. Following the screening, participants will enter the treatment phase, where they will receive regular infusions of bepranemab. Throughout the study, follow-up visits will be conducted to monitor the incidence of treatment-emergent adverse events, which is the primary endpoint of the trial. The end-of-study visit will conclude the participant's involvement, assessing the overall safety and tolerability of the treatment.

Participant involvement is expected to last up to 144 weeks, contingent upon adherence to study protocols and the absence of any adverse events that may necessitate early termination. Conditions that could lead to early withdrawal include significant adverse reactions or non-compliance with study requirements. The trial aims to gather comprehensive data on the long-term effects of bepranemab, contributing to the understanding of its potential as a therapeutic option for PSP.

The clinical trial involves the administration of **Bepranemab**, an experimental medication, to evaluate its long-term safety and tolerability in participants with **Progressive Supranuclear Palsy**. Bepranemab is provided in the form of a **solution for infusion** and is administered via the **intravenous route**. The dosing regimen is specified in milligrams per kilogram (mg/Kg), although the exact dosage and maximum daily dose are not explicitly defined in the trial data. The treatment period for Bepranemab is set to a maximum of 144 weeks. The active substance, Bepranemab, is a protein-based compound, classified under the category "Protein - Other". The pharmaceutical product is developed by UCB Biopharma SRL and is identified by the sponsor product code UCB0107.

In this study, no non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatments are mentioned. The trial is designed as an open-label extension study, focusing solely on the administration of Bepranemab. Participant compliance with the dosing schedule is monitored throughout the study duration to ensure adherence to the treatment protocol. The trial does not include a pediatric formulation, and Bepranemab is not classified as an orphan drug for this study. The primary objective is to assess the safety and tolerability of Bepranemab in the specified patient population over the long term.

The efficacy of Bepranemab in the clinical trial will be assessed primarily through the incidence of treatment-emergent adverse events from the baseline of the open-label extension to the end of the study. This endpoint is designed to evaluate the long-term safety and tolerability of Bepranemab in participants with **Progressive Supranuclear Palsy** (PSP). The trial is a Phase 3 open-label extension study, focusing on participants who have completed the treatment period in a previous study (PSP003). The assessment will involve systematic collection and analysis of adverse event data throughout the study duration, ensuring comprehensive monitoring of the participants' response to the treatment. The trial does not specify additional secondary efficacy endpoints, emphasizing its primary objective of safety and tolerability evaluation.