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Contact Information:

Dr Catherine Rea
catherine@hemab.com


Dr Ulrike Lorch
u.lorch@richmondpharmacology.com


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Be Part of Research - Trial Details - A first-in-human study of HMB-001 in patients with Glanzmann thrombasthenia
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A first-in-human study of HMB-001 in patients with Glanzmann thrombasthenia

Not Recruiting

Open to: All Genders

Age: Adult

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Medical Conditions

Glanzmann thrombasthenia

This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


Glanzmann thrombasthenia (GT) is a rare bleeding disorder, in which patients’ bodies cannot properly form blood clots to stop bleeding. This leads to frequent bleeding, with many patients experiencing a bleed every day. This is a very serious disorder that, whilst rare (there are only around 150 patients in the UK), leads to regular and potentially life-threatening bleeding. These frequent bleeds also have a significant impact on an individual’s ability to lead a normal life, often having to miss school or work.
While there are drugs currently available to help stop bleeding once it has occurred, there are no drugs currently available to effectively prevent bleeding from occurring. Hemab Therapeutics ApS is funding this research to evaluate the ability of a new drug, HMB-001, to help patients with GT. HMB-001 is a medicine intended to prevent and reduce bleeding events in patients with GT. This will be the first time HMB-001 will be given to humans, and the main goal of this trial is to look at the safety of the drug. In addition to determining how safe and well tolerated it is in humans, this clinical trial will also look at how long the drug will remain in the body (pharmacokinetics), how it affects the body (pharmacodynamics), and whether it works for preventing bleeding (efficacy).

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

02 Nov 2022 17 Dec 2023

This is a multicenter clinical trial conducted in the UK and can have up to 15 research sites. This trial plans to enrol a maximum of 57 male and female participants. The trial is comprised of two parts (Part A and Part B). Part A will include up to 21 participants and will evaluate a single dose of HMB-001. Part B will include up to 36 participants and will evaluate multiple doses of HMB- 001. If a participant is recruited into Part A, they may also be able to enter into Part B of the study once they have completed Part A. Participants will be in the trial for approximately one and half years.


Adults with GT

You can take part if:



You may not be able to take part if:


1. Severe infection or inflammation at the time of Screening2. History of clinically significant hypersensitivity associated with monoclonal antibody therapies3. Personal history of venous or arterial thrombosis or thromboembolic disease4. Known severe congenital or acquired thrombophilia5. Has vital signs outside of the following normal range at Screening: 5.1 Supine heart rate <100 bpm (after at least 5 minutes of supine rest)5.2. Blood pressure (BP): Supine BP (after at least 5 minutes of supine rest): 5.3. Systolic blood pressure: 90 - 140 mmHg5.4. Diastolic blood pressure: 40 - 90 mmHg6. Has a positive test for Hepatitis B surface antigen (HBsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at Screening7. Is currently positive for COVID-19 based on lateral flow8. Is within 6 weeks of COVID-19 infection9. Other conditions that substantially increase risk of thrombosis by the discretion of the investigator including, but not limited to: significant family history, BMI >30 kg/m2 (moderately obese, adjusted for ethnicity), reduced mobility, active malignancy, major surgery within 6 weeks preceding first dose of study drug, post-partum within 12 weeks preceding first dose of study drug10. Women who are using estrogen-containing medication or hormone modulators (within 8 weeks pre dose to 8 weeks post dose of study drug) including: 10.1. Combined oral contraception pill 10.2. Hormone replacement therapy (excluding transdermal patches) 10.3. Oestrogen receptor modulators (eg, Tamoxifen) 10.4. Gonadotropin releasing hormone receptor (GnRH receptor) agonist 11. Clinically significant cardiovascular disease including, but not limited to: New York Heart Association Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischaemic stroke. 12. Other conditions that substantially increase risk of cardiovascular events by the discretion of the Investigator including, but not limited to: smoking, cocaine use, and uncontrolled hypertension.13. Congenital or acquired bleeding disorders other than Glanzmann thrombocythemia. 14. Concurrent disease, treatment, medication, or abnormality in clinical laboratory tests that may pose additional risk in the opinion of the investigator and preclude the participant’s safe participation in and completion of the study.15. Addiction or other diseases that prevent the participant from appropriately assessing the nature and scope of the clinical study or participating in study procedures by the discretion of the Investigator.16. Received investigational medication in another clinical study within 5 half-lives before administration of the study drug. 17. Female participants who are pregnant (including a positive serum pregnancy test at Screening) or breastfeeding.


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

At the moment there are no treatments to prevent the bleeds caused by Glanzmanns thrombasthenia, only ways to manage them when they happen. Whilst there are expected to be risks associated with taking part in the trial for HMB-001, everything possible has been done to control them. Participants in this trial will all have GT and so the benefit to them from finding better ways to treat the illness is expected to outweigh these risks.
Groups that represent individuals with GT have been important to the decisions made about this trial, particularly the advocacy organisation and patient community forum called Haemnet. Along with specialist doctors, they have already helped to design an approved trial that will gather the information needed for potential participants to take part in the HMB-001 trial.
The main risk expected from HMB-001 is that, rather than the blood being too slow to clot, it may cause participants to form a clot too easily. The potential impact of this could range from relatively minor changes that the participant would suffer no harm from, all the way through to clots in the heart, lungs, or brain that could potentially lead to death.
The risk of people forming these clots is thought to be minimal. HMB-001 will work in a similar way to a treatment called rFVIIa that is already used for people with GT and other, more common bleeding problems. RFVIIa is a well-understood medicine and has not been seen to frequently cause dangerous clots. In the rare cases where clots have been reported, they have been in patients who have had additional risks, like those undergoing major surgery.
To minimise the unlikely chance of a clot occurring, every potential participant will be reviewed by trained doctors to look for factors that increase their risk of forming a clot. If any are present, they will not be able to take part in the trial.
HMB-001 belongs to a type of medicines called monoclonal antibodies, these medicines work by mimicking the part of our immune system that recognises things that can do us harm. Over time, the participant’s immune system may start to react to this by forming its own antibodies that attack HMB-001. This can cause the medicine to become less effective or to behave differently.
To monitor for this, participants in all parts of the trial will have samples taken to check for these antibodies.
Finally, there are the risks associated with trial procedures. In particular, the trial for HMB-001 will involve a bleeding time assessment, this requires a small cut to be made on the individual’s forearm with the help of local anaesthetic to numb the area. In people with a bleeding disorder such as GT, this can lead to prolonged bleeding and potentially the formation of a small scar.
All bleeding time assessments will be conducted in a clinical setting by trained professionals. If the participant prefers not to undergo this specific test, they are able to opt-out. In addition to this, anyone with a history of allergy to local anaesthetic, or who has a tendency to form larger, troublesome scars will not have this test performed.
Participants will have the opportunity to individually discuss the risks and benefits involved in HMB-001 with a doctor familiar with the trial, this will take place during the informed consent process.
With regard to the burden of taking part in the trial for HMB-001, participants will be expected to attend seven visits to a clinic alongside a stay of five consecutive days. The trial has been designed to minimise the number of visits required, and where possible the exact day participants need to attend is made flexible by allowing their scheduled visit to occur between one and three days either side of it.
COVID-19 continues to pose a significant risk to certain individuals, and those with GT may be at a higher risk. To keep potential participants and other visitors safe, a lateral flow test will be performed before entering the clinic. If an individual tested positive, they would not be given HMB-001 and their involvement in the trial would be deferred. This is because COVID-19 is known to increase the risk from clots forming in the blood.
If any significant changes to the risks, benefits, or burden involved in the trial for HMB-001 emerge after participants enrol, they will be informed in a timely manner and asked whether they continue to consent to take part.

Dr Ulrike Lorch
u.lorch@richmondpharmacology.com


Dr Catherine Rea
catherine@hemab.com



The study is sponsored by Richmond Pharmacology Ltd and funded by Hemab Therapeutics ApS.



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Read full details for Trial ID: ISRCTN66310879
Last updated 28 July 2023

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