Trial Outcomes & Findings for Study to Investigate the Safety of LT5001 Drug Product for Uremic Pruritus in Hemodialysis Patients (NCT NCT04393675)

The study was terminated after completion of Part A (18 participants). Part B was planned but not initiated.

Participants underwent a 7-day run-in period prior to Part A. Only participants meeting eligibility criteria after the run-in period were enrolled into Part A. No participants entered Part B, as Part B was not initiated.

Study to Investigate the Safety of LT5001 Drug Product for Uremic Pruritus in Hemodialysis Patients

The number of reported AE events by severity

Adverse events (AEs) were collected and categorized by severity (mild, moderate, or severe) according to standard clinical criteria. The outcome measure is defined as the number of patients who experienced at least one adverse event of each severity level during the study period.

Adverse events were assessed for their causal relationship to the study treatment by the investigator and categorized as related or not related. The outcome measure is defined as the number of patients who experienced at least one adverse event in each causality category during the study period.

The number of reported events by causality

The systemic trough (Ctrough) concentration of LT5001 was measured in hemodialysis patients at specified time points prior to the next scheduled dose using validated bioanalytical methods. The outcome measure is defined as the plasma concentration of LT5001 immediately before each dosing interval.

The intensity of itch will be measured using the Numerical Rating Scale (NRS), a standardized scale for assessing the worst itching over the past 24 hours. The scale ranges from 0 to 10, where 0 represents no itching and 10 represents the worst itching imaginable. Higher scores indicate worse itch severity. The outcome measure is defined as the change in mean worst itching intensity from baseline to the end of Week 4.

The Numerical Rating Scale (NRS) is an 11-point scale used to assess itch intensity, where scores range from 0 to 10, with 0 indicating no pain and 10 indicating the worst itch imaginable. A decrease in NRS score represents an improvement in itch severity. This outcome measures the proportion of patients who achieved a reduction of at least 2 points, 3 points, or 4 points from baseline in NRS score at Week 4.

The 5-D Pruritus Scale (5-D Itch Scale) is a multidimensional questionnaire assessing itching across five key domains: Duration, Degree, Direction, Disability, and Distribution, to comprehensively evaluate the impact of itch on daily life. The scale ranges from 5 to 25, with 5 representing no itch and 25 representing severe itch. Each domain is scored from 1 (lowest) to 5 (highest). Higher scores indicate worse itch and poorer itch-related quality of life, whereas lower scores indicate better quality of life. The outcome measure is defined as the change from baseline in 5-D Pruritus Scale total score at the end of Week 4, with improvement reflected by a decrease in total score.

The Skindex-10 Scale is a multidimensional questionnaire assessing itch-related quality of life over the past week. The questions cover 3 domains: disease, mood/emotional distress, and social functioning domain. Each of the 10 items is scored from 0 to 6, resulting in a total score ranging from 0 to 60, where 0 represents never bothered/no impact and 60 represents always bothered/severe impact. Higher scores indicate worst itch-related quality of life, whereas lower scores indicate better quality of life. The outcome measure is defined as the change from baseline in total Skindex-10 Scale Score at the end of Week 4, with improvement reflected by a decrease in total score.