This is a randomized, double-blind, placebo-controlled dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of intra-articular injection of RAB001 in participants with knee osteoarthritis (KOA).
The study consists of five dose groups: 1.5 mg, 4.5 mg, 9 mg, 18 mg and 27 mg. A total of 12 participants with knee osteoarthritis will be enrolled per dose group (9 receiving investigational product and 3 receiving placebo), for an overall total enrollment of 60 subjects. The 27 mg cohort serves as an optional escalation group, whose initiation is contingent upon assessment by the Safety Review Committee (SRC).
Eligible participants will first receive a single-dose administration followed by a 14-day observation period, during which safety, tolerability, PK and preliminary efficacy data will be collected.
In the multiple-dose phase, study treatment will be administered once every 4 weeks for a total of 2 injections, on Week 4 (Day 29) and Week 8 (Day 57), respectively. After the last administration, subjects will enter the follow-up period and return to the study site for safety and efficacy assessments at Weeks 12, 16 and 24.
A staggered dose-escalation design will be implemented. Escalation to the next higher dose level may proceed only after completion of the 14-day safety and tolerability evaluation for all subjects in the current dose cohort and subsequent approval from the SRC. Each participant will receive only one assigned dose level throughout the trial.