We are conducting a clinical trial for a new medicine that could be used to treat people with Glanzmann thrombasthenia.

• In this clinical trial we are recruiting male and female participants aged 18 to 65 years with Glanzmann thrombasthenia. We will observe how your body processes the new medicine and how long the new medicine stays in your body.
• You will be paid for your time and commitment if you participate in this clinical trial.

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Clinical trial summary

• The intention of the study drug is to prevent and reduce bleeding events in patients with Glanzmann thrombasthenia.
• The aim of the clinical trial is to evaluate the safety of the study drug when given to patients with Glanzmann thrombasthenia.
• The trial also aims to look at how long the drug will remain in the body (pharmacokinetics), how it effects the body (pharmacodynamics), and whether it works for preventing bleeding (efficacy).
• This clinical trial is split into two parts (Part A and Part B) and will require you to attend our clinical facility in London Bridge for a number of visits (see below). You may be eligible to participate in Part A, Part B, or both.

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This clinical trial will be divided into 2 parts:‍

• Part A: During this part of the study, different dose levels of the study drug will be tested. This study part will consist of four groups which will involve about 1-6 participants per group. Each participant will receive a single dose. This part will involve an in-house stay.
• Part B: This part of the study will involve 3 cohorts with a minimum of 3 participants per group, with the possibility of expanding to 12. Each cohort will test a different dose level, with each participant receiving multiple doses for a 3-month period. This part will not involve an in-house stay.
• During your in-house stays, you will receive standard meals each day including breakfast, lunch, evening meals and snacks, served at specific times. You will have to consume all your meals and snacks.

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If you are interested in participating in the trial:

Part A

• You will need to attend a screening appointment so we can check if you are suitable to take part in the trial. At this appointment, we will perform a number of tests, including taking blood samples, checking your heart and also carry out a physical examination.
• Following the screening appointment, you will complete a Run-in Observation Period or Historical Bleeding Data collection period – this will involve an observation for up to 90 days to record bleeding events or you will be asked for estimated bleeding frequency over the past 90 days. This is to determine how often you usually experience bleeds.
• If results from your screening appointment show you are suitable for the trial, you will be invited to take part.
• This will involve a stay of 5 days / 4 nights in our clinical research unit. You will arrive one day before you are dosed, this visit is called Day -1. You will then be dosed on Day 1 and stay in the unit overnight until discharge on Day 4
• After your stay with us you will be required to return for further appointments on Day 8, Day 15, Day 22, Day 29, Day 43, and Day 57 of the trial.
• This means the whole process takes approximately 25 weeks from your screening visit through to your final visit.
• In total, if you are included in the clinical trial you will come to the research unit 8 times.
• During your stay with us you will receive a single dose of the new medicine via an injection under the skin (subcutaneous), into your abdomen or thigh. We will conduct a number of assessments (like those we did at screening) to check how well you tolerate the new study drug, and to help us understand how long it takes for your body to get rid of the study drug.

Part B

• You will need to attend a screening appointment so we can check if you are suitable to take part in the trial. At this appointment, we will perform a number of tests, including taking blood samples, checking your heart and carrying out a physical examination.
• Following the screening appointment, you will complete a Run-in Observation Period or Historical Bleeding Data collection period – this will involve an observation for up to 90 days to record bleeding events or you will be asked for estimated bleeding frequency over the past 90 days. This is to determine how often you usually experience bleeds.
• If results from your screening appointment show you are suitable for the trial, you will be invited to take part.
• This will involve receiving multiple doses of the study drug for a 3-month period in our clinical research unit. Your first dose will be on Day 1 and will be at intervals based on data from Part A.
• There will be no in-house stay, but you will be required to attend the clinic on Day 8, Day 15, Day 22, Day 29, Day 43, Day 57, Day 71 and Day 85 for treatment visits and 28 days after your last dose for an end of study visit.
• This means the whole process takes approximately 33 weeks from your screening visit through to your final visit.
• In total, if you are included in the clinical trial you will come to the research unit 12 times.
• During your treatment visits, you will receive multiple doses of the study drug via an injection under the skin (subcutaneous), into your abdomen or thigh. We will conduct a number of assessments (like those we did at screening) to check how well you tolerate the new study drug, and to help us understand how long it takes for your body to get rid of the study drug.