A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

To join this study, you need to be between 18 and 65 years old. You must have a confirmed diagnosis of Glanzmann Thrombasthenia, either through specific blood tests that look at how your platelets work and what's on their surface, or through a genetic test.

There are also some health checks to make sure it's safe for you to participate. For example, your heart rate and blood pressure need to be within healthy ranges. Your kidneys and liver must be working well, and your haemoglobin (a measure of red blood cells) should be above a certain level. You also need to be able to understand the study information and agree to follow all the study procedures.

If you are a woman who could become pregnant, you'll need a negative pregnancy test and must agree to use highly effective birth control throughout the study and for six months afterwards. Men who could father children will also need to use effective birth control during the study and for six months after their last dose.

If you decide to take part, the study is divided into several parts. Part A involves taking a single dose of HMB-001, with different small groups receiving different doses. Doctors will carefully monitor your health before increasing the dose for the next group. This part could last about 6 months, including initial checks, a possible observation period, and an 8-week follow-up after your dose.

Part B is similar, testing various doses but you'll receive HMB-001 multiple times over a 3-month period – either weekly, every two weeks, or every four weeks. This involves an initial check, an observation period, the 3-month treatment, and a safety check after your last dose.

If you complete Part B, you might be able to continue into Part C. This would involve taking HMB-001 for a longer period, approximately 18 months, followed by another safety check. Throughout all parts, there will be regular visits and assessments to monitor your health, how the medicine is affecting you, and if it's helping with your bleeding.