Evaluation of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients with Sub-optimal Response to C5 Inhibitor Ravulizumab: A Phase 2 Study

The primary objective of this study is to evaluate the **safety**, **tolerability**, **pharmacokinetics**, **pharmacodynamics**, and preliminary **efficacy** of OMS906 in patients with **Paroxysmal Nocturnal Hemoglobinuria** (PNH) who exhibit a sub-optimal response to the C5 inhibitor, Ravulizumab. This is clinically relevant as it aims to address the unmet need for effective treatment options in PNH patients who do not adequately respond to existing therapies, potentially improving patient outcomes and quality of life.

The clinical trial involves a total of **9 participants** diagnosed with **paroxysmal nocturnal hemoglobinuria**. The study population includes both male and female subjects, with an age range spanning from 18 to 64 years. Participants were selected without targeting any vulnerable populations. The general health status of the participants is not specified, and there are no specific lifestyle considerations such as diet or physical activity mentioned. The sponsor has not provided detailed information regarding the main objective of the trial or the principal inclusion criteria.

The clinical trial is designed to evaluate the **safety**, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of OMS906 in patients with **paroxysmal nocturnal hemoglobinuria** (PNH) who exhibit a sub-optimal response to the C5 inhibitor, Ravulizumab. This is a Phase 2, randomized, double-blind, controlled study. The trial is expected to commence recruitment on November 1, 2022, and is estimated to conclude by April 12, 2025. Participants will be involved in the study for a duration that aligns with the trial's timeline, subject to their continued eligibility and adherence to the study protocol.

The sequence of study visits includes an initial screening visit to assess eligibility based on predefined criteria. Following successful screening, participants will be randomized and begin the treatment phase. Regular follow-up visits will be scheduled to monitor the participants' response to the treatment, assess any adverse events, and ensure compliance with the study protocol. The end-of-study visit will occur at the conclusion of the treatment period, where final assessments will be conducted to evaluate the primary and secondary endpoints of the trial.

Participants may be withdrawn from the study early if they experience significant adverse effects, fail to comply with the study protocol, or if the investigator deems it in the best interest of the participant's health. The study is conducted under strict ethical guidelines, ensuring the safety and well-being of all participants throughout the trial duration.

The clinical trial documentation does not provide specific details regarding the **experimental medication** used in the study. Information such as the name, pharmaceutical form, dosage, route, and frequency of administration is not available. Additionally, there is no data on whether the medication is a **paediatric formulation** or if it is classified as an **orphan drug**. The maximum daily dose, total dose, and treatment period are also unspecified.

Details about any **non-experimental treatments** used in the study, such as standard-of-care therapy, placebo, or comparator treatment, are not provided. There is no information on the administration of other medicinal products or their role in the trial.

Furthermore, the documentation lacks information on the **participant compliance monitoring** procedures, dosing schedules, and any additional relevant information about drug administration. The absence of these details limits the ability to provide a comprehensive description of the treatments used in the clinical trial.

The clinical trial is a Phase 3 study with an estimated recruitment start date of November 1, 2022, and an estimated end date of April 12, 2025. The efficacy of the investigational treatment will be assessed through predefined primary and secondary endpoints, although specific endpoints are not detailed in the provided data. The trial will follow a structured schedule for measuring and collecting efficacy data, which is typical for Phase 3 trials, but specific timepoints and methods are not specified. The analysis of efficacy parameters will be conducted using standard statistical methods appropriate for the trial phase and objectives. The trial is categorized under trial category 1, with a trial category ID of 30198, indicating its classification within the clinical trial framework.