Phase 1b Evaluation of CTPS1 Inhibitor STP938 in High-Risk Essential Thrombocythaemia Resistant or Intolerant to Hydroxycarbamide Therapy

The primary objective of this open-label, Phase 1b study is to evaluate the **safety** and **tolerability** of the CTPS1 inhibitor STP938 in adult subjects with high-risk **Essential Thrombocythaemia** who are resistant to or intolerant of hydroxycarbamide therapy. This evaluation is clinically relevant as it aims to determine the potential of STP938 as an alternative treatment option for patients who do not respond to or cannot tolerate the standard therapy, thereby addressing an unmet medical need in this patient population.

The clinical trial involves a total of **10 participants** diagnosed with **thrombocythaemia**. The study population includes both male and female subjects, with an age range encompassing adults and older adults. Participants were selected to include a vulnerable population, although specific inclusion or exclusion criteria were not provided by the sponsor. The general health status of the participants is not detailed, nor are any lifestyle considerations such as diet, physical activity, or habits. The trial aims to gather data from a diverse group, ensuring representation across different age groups and genders.

The clinical trial is designed to evaluate the safety, tolerability, and preliminary activity of the CTPS1 inhibitor STP938 in adult subjects diagnosed with high-risk **essential thrombocythaemia** who are resistant to or intolerant of hydroxycarbamide therapy. This study is structured as an open-label, Phase 1b trial, with an estimated recruitment start date of March 17, 2025, and an anticipated completion date of March 17, 2027. The trial will involve a series of study visits, beginning with an inclusion visit where participants will be screened for eligibility based on predefined criteria. Following successful screening, participants will undergo a series of follow-up visits to monitor safety and efficacy outcomes. The trial will conclude with an end-of-study visit to assess the overall impact of the treatment and gather final data.

Participants are expected to be involved in the study for the duration of the trial, which spans approximately two years. However, certain conditions may lead to early termination from the study, such as adverse reactions to the investigational product, non-compliance with study protocols, or withdrawal of consent by the participant. The trial's methodology does not include randomization or blinding, as it is an open-label study, allowing both researchers and participants to be aware of the treatment being administered. The primary focus is on assessing the investigational product's safety profile and its preliminary therapeutic activity in the target population. Data collected throughout the trial will contribute to understanding the potential benefits and risks associated with the CTPS1 inhibitor STP938 in treating essential thrombocythaemia.

No specific information regarding the experimental medication, including its name, pharmaceutical form, dosage, route, and frequency of administration, is provided in the available data. Additionally, there is no information on any non-experimental treatments, such as standard-of-care therapy, placebo, or comparator treatment, used in the study. Details about drug administration, dosing schedules, and participant compliance monitoring are also not available. Therefore, a comprehensive description of the treatments used in this clinical trial cannot be constructed based on the provided data.

The clinical trial is designed to assess efficacy in a Phase 3 study. The trial is scheduled to commence recruitment on March 17, 2025, with an estimated completion date of March 17, 2027. The efficacy assessment will be conducted through a series of predefined parameters, although specific endpoints are not detailed in the available data. The trial will follow a structured timeline to ensure systematic data collection and analysis. The study will adhere to rigorous standards typical of Phase 3 trials, focusing on evaluating the therapeutic benefits of the investigational product. The trial's design will incorporate validated methodologies to ensure the reliability and validity of the efficacy outcomes. The results will contribute to the understanding of the investigational product's potential benefits in the targeted medical condition.