Phase 12 Study to Evaluate Safety and Effects of HMB-002 in People with Von Willebrand Disease

What this Trial Is About

Researchers are studying HMB-002 in people with Von Willebrand Disease VWD to evaluate its safety, tolerability, how the body processes the drug, its effects, and preliminary efficacy. This first-in-human Phase 12 trial includes three parts to explore different dosing schedules and combinations with standard factor concentrates for various VWD types. The study aims to understand how HMB-002 works alone and alongside standard treatments in participants aged 16 to under 70 years. The study is divided into three parts Part A tests single ascending doses of HMB-002 administered under close monitoring to assess safety and drug behavior over about 12 weeks. Part B evaluates repeated doses over approximately 21 weeks to assess safety and initial effects on bleeding events. Part C studies a single dose of HMB-002 given with a regular factor concentrate dose in participants who already receive this treatment, lasting about 17 weeks. Dosing schedules in Part B depend on Part A results. Participants will be monitored closely throughout the study with safety assessments, blood tests, and evaluations of bleeding rates and factor activity from Day 1 up to Day 113. Researchers will track any treatment-emergent adverse events and measure pharmacokinetic parameters like maximum drug concentration and elimination half-life. They will also assess pharmacodynamic markers such as von Willebrand factor antigen and activity, and factor VIII activity. Overall participation lasts up to about 17 to 21 weeks depending on the study part.

A Study Assessing HMB-002 in Participants With Von Willebrand Disease