The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia. The main questions it aims to answer are: * Parts A, B, and C: To determine the safety and tolerability of HMB-001 * Part A: To establish the dose level(s) and dosing interval(s) of HMB-001 to be investigated in Parts B and C * Parts B and C: To estimate the ability of HMB-001 to prevent the number and severity of bleeds Part A will assess differing singular doses of HMB-001 in small groups of participants. The dose administered to a newly enrolled participant (or groups of participants) may only increase if analysis of data from previous dosing shows it is safe to do so. The planned duration of participation in Part A is approximately 6 months, which consists of a Screening Period, an optional Run-in Observation Period, and a follow-up period of 8 weeks. Part B is similar to Part A as it involves testing different dose levels of HMB-001 in small groups of participants. However, in Part B, HMB-001 is given multiple times over a 3-month period, either weekly, every 2 weeks, or every 4 weeks. Part B consists of a Screening Period, a Run-in Observation Period, a 3-month Treatment Period, and a Safety Follow-up following the last dose of HMB-001. Part C is open to participants from Part B and consists of approximately a 18-month Treatment Period and a Safety Follow-up following the last dose of HMB-001.
Age range
18 Years – 67 Years
Sex
ALL
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Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Part A: Safety as assessed by the incidence of treatment-emergent adverse events (TEAEs)
Timeframe: From baseline to Day 57
Part A: Safety as assessed by the changes in physical examinations, vital signs, clinical laboratory assessments, and ECG parameters
Timeframe: From baseline to Day 57
Part B: Safety as assessed by the incidence of treatment-emergent AEs
Timeframe: From baseline to Day 106/Early Termination (ET)/End of Study (EOS)
Part B: Safety as assessed by the changes in physical examinations, vital signs, clinical laboratory assessments, and ECG parameters
Timeframe: From baseline to Day 106/ET/EOS
Part B: Preliminary prophylactic effect of HMB-001 as assessed via Bleed frequency: annualized bleed rate (ABR)
Timeframe: From baseline to Day 106/ET/EOS
Part B: Preliminary prophylactic effect of HMB-001 as assessed via Bleed frequency: annualized treated bleed rate (ATBR)
Timeframe: From baseline to Day 106/ET/EOS
Part C: Safety as assessed by the incidence of treatment-emergent AEs
Timeframe: Day 99 to Day 687/Early Termination (ET)/ENdo of Study (EOS)
Part C: Safety as assessed by the changes in physical examinations, vital signs, clinical laboratory assessments, and ECG parameters
Timeframe: Day 99 to Day 687/Early Termination (ET)/ENdo of Study (EOS)
Part C: Preliminary prophylactic effect of HMB-001 as assessed via Bleed frequency: annual treated bleed rate (ATBR) and annualized bleed rate (ABR)
Timeframe: Day 99 to Day 687/Early Termination (ET)/ENdo of Study (EOS)