RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This research study is trialling a new medication called HMB-002 for adults aged 18 to 64 who have Von Willebrand Disease (VWD), specifically Type 1 or Type 2A. VWD is a condition where your blood doesn't clot properly, leading to increased bleeding. The study has two parts. The first part aims to find the safest and most effective dose of HMB-002 and understand how it works in the body after a single dose. The second part will test repeat doses, focusing on safety and how well it helps control bleeding episodes in people who frequently experience them. This is an important step to see if HMB-002 could be a future treatment option for VWD.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Hemab ApS

Enrolment target

108

Start

06 Feb 2025

Estimated completion

01 Jul 2027

Von Willebrand Disease (VWD)Von Willebrand Disease (VWD), Type 1 Von Willebrand Disease (VWD), Type 2

What is this study about?

This study is investigating a new medication called HMB-002 for people with Von Willebrand Disease (VWD). VWD is a genetic condition that affects how your blood clots. If you have VWD, your body doesn't produce enough of a protein called Von Willebrand Factor (VWF), or the VWF it does produce doesn't work correctly. This can lead to longer or heavier bleeding than usual, for example, from cuts, nosebleeds, or during periods.

Researchers are always looking for new and better ways to manage VWD. HMB-002 is an experimental medicine, which means it's still being tested and is not yet available as a standard treatment. This study is one of the first times it's being given to people, so the main goals are to make sure it's safe, to understand how the body handles it, and to see if it can help with bleeding problems. They are especially interested in Type 1 and Type 2A VWD.

The study is divided into two sections. In the first part, a small group of participants will receive a single dose of HMB-002 to check for safety and how the body responds. Once a safe dose is identified, the second part will involve giving repeat doses to a larger group. This will help scientists understand its long-term safety and whether it can effectively reduce the number of bleeding episodes in people who have frequent bleeds. This vital research helps move new treatments closer to becoming available to patients who need them.

Key takeaways

The study tests a new medicine, HMB-002, for Von Willebrand Disease (VWD).
It's for adults aged 18-64 with certain VWD types (Type 1, Type 2A).
The main goals are to check safety, how the body uses the medicine, and if it helps reduce bleeding.
Participation involves clinic visits, blood tests, and careful monitoring.
It's an early-stage study (Phase 1/2), meaning HMB-002 is an experimental medicine.
Your health and safety are the top priority throughout the study.

Who may be eligible?

To join this study, you need to be an adult between 18 and 64 years old and weigh between 50 and 110 kilograms (about 7 stone 12 pounds to 17 stone 5 pounds). You must have a confirmed diagnosis of certain types of Von Willebrand Disease (specifically Type 1 or Type 2A) and your heart rate, blood pressure, and certain blood test results (for your kidneys, liver, and blood count) need to be within a healthy range.

If you are a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use two reliable forms of contraception throughout the study. Men with partners who could become pregnant also need to use contraception. For the second part of the study, you must also have a history of frequent bleeding events, typically at least three treated bleeding episodes within the last year, showing the medicine is being tested in people who need it most.

You cannot participate if you have certain other serious health conditions or medical history that the doctors feel would make the study unsafe for you. The study team will review all your medical information carefully to make sure it's safe for you to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 64 years old?
Have you been diagnosed with Type 1 or Type 2A Von Willebrand Disease?
Do you weigh between 50kg and 110kg?
Are your heart rate, blood pressure, and routine blood tests (kidney, liver) generally healthy?
If you're a woman who could get pregnant, are you willing to use two forms of contraception?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given HMB-002, the experimental medicine, either as a single dose or as repeated doses, depending on which part of the study you are in. You will have regular visits to the clinic for medical check-ups, blood tests, and other assessments to monitor your health and how your body is reacting to the medicine. This helps the study team understand the medicine's safety and how it works.

Before you start, you'll have screening appointments to make sure you meet all the health requirements. Throughout the study, you'll be asked to keep track of any new symptoms or bleeding events. The total length of your participation will depend on which part of the study you join and the specific schedule set by the researchers, but it will involve a period of follow-up after your last dose of HMB-002.

Potential risks and benefits

Taking part in a research study means there might be potential benefits, but also potential risks. A possible benefit of this study is that HMB-002 could help reduce your bleeding episodes if it works as intended, although this is not guaranteed as it's an experimental drug. You will also receive careful medical monitoring and support during your time in the study. However, like all medicines, HMB-002 may have side effects, some of which might be serious or currently unknown. The research team will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care, by simply telling the study team.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Fiona Stanley Hospital
Verified postcode
Murdoch, Australia· Not yet recruiting
Royal Prince Alfred Hospital
Verified postcode
Camperdown, Australia· Recruiting
The Alfred Hospital
Verified postcode
Melbourne, Australia· Recruiting
Richmond Pharmacology
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Von Willebrand Disease (VWD)?

VWD is a common inherited bleeding disorder where your blood doesn't clot properly, leading to more bleeding than usual.

What is HMB-002?

HMB-002 is a new, experimental medication being tested in this study to see if it can help people with VWD.

Who can take part in this study?

Adults aged 18 to 64 with specific types of VWD and who meet other health requirements can be considered.

Will I get the actual medicine?

Yes, if you qualify and choose to participate, you will receive doses of HMB-002, as this study doesn't use a placebo (dummy drug).

What is the purpose of this study?

The study aims to understand if HMB-002 is safe, how it works in the body, and if it can help reduce bleeding in people with VWD.

How to find out more

Clinical Trials

clinicaltrials@hemab.com 080 8304 6409 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.