NCT06848296

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of of VSA012 in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH)

Phase Ib Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VSA012 Injection in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH) Who Are Complement Inhibitor Naïve or Have Not Received Complement Inhibitor Therapy Recently

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 16 (estimated)
• Sponsor: Visirna Therapeutics HK Limited · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The complement system is an important component of the innate immune system. Abnormal activation, inadequate regulation and control of the complement system, as well as impaired and dysfunctional effector functions, underlie complement mediated diseases including PNH. VSA012 targeting complement system has the potential to treat a variety of diseases associated with abnormal activation of the complement system.The purpose of VSA012-1002 is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamics and efficacy of VSA012 Injection in subjects with PNH.

Detailed description

This is a multi-center, non-randomized, open-label Phase 1b study to evaluate the safety and tolerability, pharmacokinetic and pharmacodynamic profiles of VSA012 Injection in Chinese subjects with PNH, as well as to explore preliminary efficacy. The VSA012 60 mg dose group and VSA012 120 mg dose group were initially proposed in this study; adjustments will be made based on data obtained in a single ascending dose study in healthy adult Chinese subjects (VSA012-1001) and stepwise data from this study, taking full account of subject safety. Enrollment in the 60 mg dose group of this study was not initiated until 15 days postdose safety assessments were completed for subjects in the 60 mg dose group of Study VSA012-1001; enrollment in the 120 mg dose group of Study VSA012-1001 was not initiated until 15 days postdose safety assessments were completed for subjects in the 120 mg dose group. About 8 subjects with PNH will be enrolled in each group in this study and will receive VSA012 Injection 60 mg or V SA012 Injection 12 0 mg on D1 and D29, respectively; based on full consideration of the safety of subjects, the dosing regimen may be adjusted in combination with the data gradually obtained from the VSA012-1001 study and this study. Participants may receive concomitant supportive care permitted in this study throughout the study and continue to be followed for 24 weeks following completion of D29 dosing. This study includes: Screening Period, Treatment Period, Follow-up Period.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Timeline

• Start date: 2025-04-01
• Primary completion: 2026-04-01
• Completion: 2026-04-01
• First posted: 2025-02-27
• Last updated: 2025-02-27