BRIGHT - A Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Multiple Doses of LT3001 Drug Product in Subjects with Acute Ischemic Stroke (AIS)

The primary objective of this study is to determine the **safety** of multiple doses of the LT3001 drug product in subjects with **Acute Ischemic Stroke (AIS)**. Evaluating the safety profile is crucial as it ensures that the therapeutic intervention does not pose undue risk to patients, thereby facilitating its potential use in clinical practice.

Secondary objectives include determining the efficacy of multiple doses of the LT3001 drug product in subjects with AIS. Assessing efficacy is essential to establish the therapeutic benefit of the drug, which could lead to improved clinical outcomes for patients suffering from AIS.

The clinical trial involves a total of **104 participants** diagnosed with **Acute Ischemic Stroke (AIS)**. The study population includes both male and female subjects, aged between 18 to 80 years. Participants were selected based on specific criteria, including the ability to receive the first investigational product within 24 hours after the onset of stroke symptoms and having an NIHSS score of 4 to 25. All subjects are required to have adequate renal function and no history of severe allergic reactions to contrast agents, as they must be able to undergo a contrast brain perfusion with either MRI or computed tomography (CT). The trial includes a vulnerable population, and lifestyle factors such as diet and physical activity were not specified as part of the selection criteria. The selection process ensures a diverse representation of individuals affected by AIS, focusing on safety assessments of the LT3001 drug product.

The clinical trial is designed as a **Phase II**, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of multiple doses of the investigational product, LT3001, in subjects diagnosed with **acute ischemic stroke**. The trial aims to determine the safety profile of LT3001 when administered intravenously as a solution for infusion. The study will involve a placebo group to ensure the reliability of the results. The trial is expected to commence recruitment on September 1, 2023, and conclude by March 31, 2025.

Participants will be involved in the study for a maximum treatment period of 3 days, with the investigational product administered at a maximum daily dose of 0.1 mg/kg and a total dose of 0.3 mg/kg. The study will include several key visits: an initial screening visit to confirm eligibility, follow-up visits to monitor safety and efficacy, and an end-of-study visit to assess the overall outcomes. The primary endpoint is the proportion of subjects experiencing adverse events related to the investigational product within 90 days post-administration.

Inclusion criteria require participants to be aged 18 to 80 years, have a National Institutes of Health Stroke Scale (NIHSS) score between 4 and 25, and be able to receive the first dose within 24 hours of stroke symptom onset. Adequate renal function and the ability to undergo contrast brain perfusion imaging are also necessary. Participants may be withdrawn from the study if they experience severe adverse reactions or fail to comply with study protocols. The trial's design ensures rigorous assessment of the investigational product's safety and efficacy in a controlled environment.

The clinical trial involves the administration of **LT3001 Drug Product**, which is a **solution for infusion**. The active substance in this experimental medication is LT3001, a chemical compound developed by Lumosa Therapeutics Co. Ltd. The drug is administered via **intravenous infusion**. The dosing regimen includes a maximum daily dose of 0.1 mg/kg and a maximum total dose of 0.3 mg/kg over a treatment period of up to 3 days. The primary objective of the trial is to evaluate the safety of multiple doses of LT3001 in subjects with **acute ischemic stroke**. Participant compliance with the dosing schedule is monitored throughout the study to ensure adherence to the protocol.

The study also includes a **placebo** as a comparator treatment. The placebo is used to maintain the double-blind nature of the trial, ensuring that neither the participants nor the investigators know which treatment is being administered. The placebo is administered in the same pharmaceutical form and route as the LT3001 Drug Product, which is a solution for infusion given intravenously. This allows for a direct comparison of the safety and efficacy outcomes between the experimental drug and the placebo group.

The efficacy of the LT3001 Drug Product in the treatment of **Acute Ischemic Stroke (AIS)** will be assessed in a Phase II, double-blind, randomized, placebo-controlled clinical trial. The primary endpoint for evaluating efficacy is the proportion of subjects experiencing Adverse Events (AEs) that are judged to be probably or definitely related to the investigational product within 90 days following the first administration of the investigational product. This endpoint will be measured and collected systematically throughout the trial duration, ensuring comprehensive data collection and analysis.