Efficacy and Safety of HK-660S in the Treatment of Primary Sclerosing Cholangitis
brief summary
The cause of PSC is unknown.To date, there is no treatment besides liver transplantation proven to improve PSC prognosis. However, there is a clear medical unmet need yet for patients with PSC, due to risks and complications of liver transplantation. This is a two-part, Phase 2b, randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of HK-660S in patients with PSC. The primary objective is to evaluate the effects of HK-660S on serum ALP improvement (reduction of 20% or more) over 12 weeks of treatment in patients with PSC.
detailed description
This is a two-part, Phase 2b, randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of HK-660S in patients with PSC.
After subjects voluntarily consent in writing to participate in the clinical study, subjects who meet the inclusion/exclusion criteria will be evaluated through a screening period. The screening period should not, as far as possible, exceed a maximum of 5 weeks including the washout period. Based on the screening results at Visit 1, subjects suitable for participation in this clinical study will be selected.
In Part A, 6 subjects will be assigned to 200 mg HK-660S twice a day for 12 weeks. After Visit 2, subjects will visit the study center at Week 4 (Visit 3), Week 8 (Visit 4), Week 12 (Visit 5 / End of Treatment), and Week 16 (Visit 6 / Follow-up) to assess the efficacy and safety (excluding MRCP at Visit 6). Enrollment will be staggered, with the first two subjects enrolled and monitored for the initial 4-week treatment period. Interim safety data from these initial two subjects will be reviewed by the Sponsor and medical monitor to assess any significant safety concerns. If no such concerns are identified, enrollment of the remaining four subjects will proceed. Safety data from the first 4-week treatment in all 6 subjects will be reviewed by the SRC, which will decide on the initiation of Part B.
In Part B, 99 subjects will be randomly assigned in a 1:1:1 ratio into one of three groups: 100 mg HK-660S, 200 mg HK-660S, or placebo to take two tablets (1 active and 1 placebo tablets, 2 active tablets, or 2 placebo tablets) twice a day for 12 weeks. After Visit 2, subjects will visit the study center at Week 4 (Visit 3), Week 8 (Visit 4), Week 12 (Visit 5 / End of Treatment), and Week 16 (Visit 6 / Follow-up) to assess the efficacy and safety (excluding MRCP at Visit 6).
The study period for each subject is approximately 21 weeks including up to 5-week screening period, 12-week treatment period, and 4-week follow-up period.
official title
A Randomized, Double-blind, Placebo-controlled, Parallel Group, 12 Weeks, Phase 2b Clinical Study to Evaluate the Efficacy and Safety of HK-660S in Patients With Primary Sclerosing Cholangitis (PSC)