RecruitingPHASE2INTERVENTIONAL

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

This research study, called HMBeacon, is testing a new medicine known as ALN-6400. It's for women and teenage girls aged 16 to 45 who have a bleeding disorder called Von Willebrand Disease (VWD) and also experience very heavy periods, known as Heavy Menstrual Bleeding (HMB). The main goals are to check if ALN-6400 is safe to use, how well people can tolerate it, and if it helps to reduce heavy bleeding during periods. Researchers will also look at how the medicine works in the body. This is a 'Phase 2' study, meaning it's still in the earlier stages of testing in a smaller group of people, after initial safety checks have been done.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Alnylam Pharmaceuticals

Enrolment target

Start

30 Jul 2026

Estimated completion

29 Nov 2028

Von Willebrand Disease (VWD)Heavy Menstrual Bleeding (HMB)

What is this study about?

This study is about helping women and teenage girls who have two related health issues: Von Willebrand Disease (VWD) and heavy periods, also known as Heavy Menstrual Bleeding (HMB). VWD is a common inherited bleeding disorder where your blood doesn't clot properly. This can make periods much heavier and last longer than usual, which can be very difficult to manage and affect daily life.

We're testing a new medicine called ALN-6400. The aim is to see if this medicine can safely and effectively reduce the heavy bleeding during periods for people with VWD. By taking part, you would be helping doctors and researchers understand more about this potential new treatment and how it might improve the lives of others with VWD and heavy periods.

This is a 'Phase 2' study. This means it's an important step where we learn more about the medicine's safety, how well it works, and the right dose. We're looking at how your body handles the medicine and whether it makes a difference to your periods. This kind of research is vital for bringing new and better treatments to people who need them.

Key takeaways

This study is for women and teenage girls (16-45) with Von Willebrand Disease and heavy periods.
It's testing a new medicine called ALN-6400 to see if it's safe and reduces heavy bleeding.
This is a 'Phase 2' study, an important step in testing new treatments.
Participation involves regular clinic visits, blood tests, and taking the study medicine.
You must meet specific health criteria and agree to use contraception.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you need to be a woman or teenage girl between the ages of 16 and 45. You must have been diagnosed with Von Willebrand Disease (any type) and experience heavy periods. Your heavy periods need to have happened during at least two recent menstrual cycles before the study starts. You'll also need to agree to use effective contraception throughout the study.

There are some reasons why you might not be able to join. For example, if you have certain problems with your liver or kidneys, as shown by blood test results. You also can't have certain other conditions that might cause heavy bleeding, like fibroids, endometriosis, or polycystic ovary syndrome, or have recently had a new hormonal or non-hormonal coil (IUD) fitted. If you regularly use a treatment called 'factor prophylaxis' more than once a week, or plan to, you wouldn't be able to join either.

It's important to remember that these are just some of the main criteria. The study team will review your full medical history to make sure the study is safe and right for you.

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 a female aged between 16 and 45?
Do you have a confirmed diagnosis of Von Willebrand Disease?
Do you experience heavy periods (Heavy Menstrual Bleeding)?
Have you had heavy periods for at least two cycles recently?
Are you able and willing to use reliable contraception during the study?
Do you *not* have certain liver or kidney problems (this would be checked by blood tests)?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be given the study medicine, ALN-6400. You will have regular visits to the clinic so the study team can check your health, how the medicine is affecting you, and if it's helping with your periods. These visits will involve physical exams, blood tests, and discussing your period experiences. You'll also need to answer questions about your symptoms and any side effects you might be having.

The total duration of your involvement in the study will vary, but typically these types of studies involve several months of regular follow-up and treatment. The study team will provide you with a detailed schedule of visits and what to expect at each one. Your safety and well-being will be monitored very closely throughout your participation.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A possible benefit of joining this study is that ALN-6400 might help to reduce your heavy periods. You would also be contributing to medical research that could help others in the future. However, there's no guarantee the medicine will work for you, and it might not improve your symptoms. Like all medicines, ALN-6400 could cause side effects, some of which might be unpleasant or serious. The study team will explain all known side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (11)

Clinical Trial Site
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Orange, United States· Recruiting
Clinical Trial Site
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San Diego, United States· Recruiting
Clinical Trial Site
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San Francisco, United States· Recruiting
Clinical Trial Site
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Miami, United States· Recruiting
Clinical Trial Site
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Lexington, United States· Recruiting
Clinical Trial Site
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Pittsburgh, United States· Recruiting
Clinical Trial Site
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Nijmegen, Netherlands· Not yet recruiting
Clinical Trial Site
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Stockholm, Sweden· Not yet recruiting
Clinical Trial Site
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Bradford, United Kingdom· Not yet recruiting
Clinical Trial Site
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Oxford, United Kingdom· Not yet recruiting
Clinical Trial Site
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Truro, United Kingdom· Not yet recruiting

Common questions

What is Von Willebrand Disease (VWD)?

It's a common inherited bleeding disorder where your blood doesn't clot as well as it should. This can lead to heavy bleeding, including very heavy periods.

What does 'Phase 2' study mean?

It means the medicine is being tested in a small group of people to see if it's safe, how well it works, and to help decide on the right dose. It's an earlier stage of testing.

Will I definitely get the new medicine?

Yes, if you join this study, you will receive doses of the new medicine, ALN-6400.

How long will I be in the study?

The study will involve regular visits and taking the medicine over several months. The exact length will be explained by the study team.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason, and it won't affect your future medical care.

How to find out more

Alnylam Clinical Trial Information Line

clinicaltrials@alnylam.com 1-877-ALNYLAM Official trial record

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