RecruitingPhase IIInterventional

Study to test the safety and effectiveness of sutacimig for people with a rare bleeding condition called congenital factor VII deficiency

This study is looking into a new treatment called sutacimig for a rare bleeding disorder called congenital factor VII deficiency. If you have this condition, your blood doesn't clot properly because you have low levels of a protein called factor VII, which can lead to bleeding. Sutacimig is a new drug given by injection that's designed to help your blood clot more effectively. This Phase 2 study will check if sutacimig is safe, how well your body handles it, and if it helps to improve blood clotting. Participants will receive one dose and be observed for about 3 months. The goal is to see if this new medicine could be a helpful option for people with this condition.

At a glance

Status

Recruiting

Phase

Phase II

Sponsor

Hemab Aps

Enrolment target

Start

17 Oct 2025

Estimated completion

30 Jul 2026

Congenital coagulation factor VII deficiency

What is this study about?

Congenital factor VII deficiency is a rare condition that means your blood doesn't clot as it should. This happens because your body doesn't make enough of a special protein called factor VII, or the protein it does make doesn't work properly. When you get a cut or injury, factor VII usually helps to start the clotting process, which stops bleeding. Without enough working factor VII, your blood has trouble forming clots, which can lead to bleeding problems.

Researchers are developing a new medicine called sutacimig (also known as HMB-001) that they hope will help. This medicine is designed to work in a similar way to existing treatments by helping your blood produce a substance called thrombin, which is essential for blood clotting. Sutacimig is given as an injection under the skin. This particular study is a 'Phase 2' trial, which means it builds on earlier testing and is focused on finding out more about its safety and how it works in people.

The main goal of this study is to see if sutacimig is safe for people with congenital factor VII deficiency, how their bodies react to it (which experts call 'pharmacokinetics' and 'pharmacodynamics'), and if it helps improve blood clotting. The information gathered from this study will help researchers understand if sutacimig could be a useful new treatment option for this rare bleeding condition.

Key takeaways

This study is testing a new medicine called sutacimig for a rare bleeding condition.
The main goals are to check if the medicine is safe and how it works in the body.
Participants will receive a single injection and be monitored for about 3 months.
Potential risks include side effects like allergic reactions or blood clots, which will be watched closely.
This study is for adults aged 18-60 with a diagnosis of congenital factor VII deficiency.
Your participation could help advance understanding and treatment of this condition.

Who may be eligible?

To join this study, participants need to be between 18 and 60 years old and have a confirmed diagnosis of congenital factor VII deficiency, along with a history of bleeding problems. You also need to be able to fully understand the study details and agree to take part.

There are some reasons why you might not be able to join. For example, if you've had blood clots or a heart condition in the past, or if you have certain risk factors that make blood clots more likely, you wouldn't be eligible. Also, pregnant or breastfeeding women cannot take part. There are also some medications that could prevent you from joining.

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 60 years old?
Have you been diagnosed with congenital factor VII deficiency?
Do you have a history of bleeding problems related to this condition?
Are you able to understand the study details and give your consent to participate?
Are you NOT pregnant or breastfeeding?

Answer every question to see your result.

What does participation involve?

If you join the study, you'll visit the clinic on 'Day 1' to receive a single dose of the study medicine, sutacimig. You'll stay at the clinic for at least 4 hours after your dose so the study team can observe you and carry out some initial checks. After this, you'll need to return to the clinic for follow-up appointments at specific times. The total time you'll be involved in the study is expected to be about 3 months. This includes a screening period of up to 60 days to check if you're suitable, a single day for treatment, and then 28 days of follow-up observation. Doctors are looking to include up to 18 people in this study.

Potential risks and benefits

At the moment, the direct personal benefits of taking part in this specific study haven't been fully established. However, your participation could help researchers learn more about congenital factor VII deficiency and potentially lead to new treatments that could benefit others in the future. As with any new medicine, there are potential risks. Sutacimig might cause side effects, including an immune reaction to the drug, allergic reactions, a drop in blood platelet count (thrombocytopenia), or even the formation of blood clots. There are also risks linked to common study procedures, like giving blood samples or receiving injections. The effects of the drug on pregnancy, unborn babies, or sperm are not yet fully known, so strict contraception is required for participants who can become pregnant or have partners who can. If you decide to take part, the study doctor will explain all these potential risks in detail, and you always have the right to withdraw from the study at any time.

Locations (1)

The Royal London Hospital
Approximate
London, England

Common questions

What is congenital factor VII deficiency?

It's a rare bleeding condition where your blood doesn't clot properly because you have low levels of a protein called factor VII.

What is sutacimig?

It's a new medicine being tested as an injection to help blood clot in people with factor VII deficiency.

How long will I be in the study?

The study will last about 3 months in total, including screening, a treatment day, and follow-up visits.

What are the main risks?

Potential risks include side effects like allergic reactions, immune responses, a drop in blood platelets, or blood clot formation, which will be carefully monitored.

Can women who are pregnant or breastfeeding participate?

No, women who are pregnant or breastfeeding cannot take part in this study due to unknown risks to the baby.

How to find out more

- Hemab Aps

clinicaltrials@hemab.com +44 808 304 6409 Official trial record

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