RecruitingPHASE3INTERVENTIONAL

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

This research study is looking at a new medicine called NXT007 for people with haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly, leading to bleeding. The study will compare NXT007 with the usual treatment, Factor VIII, to see how well it prevents bleeding. Researchers will also check if NXT007 is safe and how the body handles it. They are looking for adults and teenagers (aged 12 and over) who have moderate or severe haemophilia A and are currently using Factor VIII to prevent bleeds. The goal is to find out if this new treatment could be a good option for managing haemophilia A.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Hoffmann-La Roche

Enrolment target

126

Start

15 May 2026

Estimated completion

23 Sep 2031

Hemophilia A

What is this study about?

This study is designed to understand if a new medicine, called NXT007, is a good option for people living with haemophilia A. Haemophilia A is a condition where your body doesn't produce enough of a protein called Factor VIII, which is essential for blood clotting. This means people with haemophilia A can experience longer bleeding episodes or bleeds that happen spontaneously.

Currently, many people with haemophilia A use treatments that replace the missing Factor VIII. This study wants to see how NXT007 compares to this standard Factor VIII replacement. Researchers will look at how well NXT007 prevents bleeding episodes, how safe it is to use, and how it works in the body. If NXT007 proves to be safe and effective, it could offer another way for people with haemophilia A to manage their condition.

The study is in 'Phase 3', which means it's one of the final stages of testing before a new medicine might be approved for wider use. It's an important step in potentially offering new and better treatment choices for haemophilia A.

Key takeaways

This study compares a new medicine (NXT007) to standard Factor VIII treatment for haemophilia A.
It's for people aged 12 and over with moderate or severe haemophilia A who are already on Factor VIII prophylaxis.
The main goals are to check if NXT007 is safe and effective at preventing bleeds.
You'll have regular clinic visits for health checks and blood tests.
Participation is voluntary, and you can withdraw at any time.
This is a 'Phase 3' study, meaning it's a key step towards potential new treatments.

Who may be eligible?

This study is looking for people aged 12 years and older who have a diagnosis of moderate or severe haemophilia A. It's important that you don't have something called 'inhibitors' against Factor VIII, which are antibodies that can stop Factor VIII treatments from working.

You would also need to have been on regular Factor VIII treatment to prevent bleeds for at least the past six months. The study team will check your medical records to make sure you meet these and other specific health requirements.

There are also some reasons why you might not be able to join. For example, if you have certain heart conditions, a history of unusual heart rhythms, or are planning to have major surgery during the study. You also shouldn't be taking certain medications that affect your immune system, except for HIV treatment, or be allergic to any part of the study medicine.

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 12 years old or older?
Do you have a diagnosis of moderate or severe haemophilia A?
Have you been regularly using Factor VIII treatment to prevent bleeds for at least the last 6 months?
Have you been told by your doctor that you do not have 'inhibitors' against Factor VIII?
Do you not have a serious heart condition or a history of unusual heart rhythms?
Are you not planning to have major surgery during the study period?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given either the new medicine, NXT007, or your usual Factor VIII treatment. You won't get to choose which one you receive, as this is decided randomly, like flipping a coin. You'll have regular appointments at the study clinic where the team will monitor your health, check for any bleeding episodes, and take blood samples to see how the treatment is working.

The study will involve a number of visits over a period of time, and you'll need to keep track of any bleeding events and medication you take. The doctors and nurses will explain everything in detail, including how long the study will last and what is expected of you at each visit. It's important to attend all scheduled appointments so the researchers can gather all the necessary information.

Potential risks and benefits

Taking part in this study could offer several potential benefits. You would receive close medical attention and access to a new investigational treatment (NXT007) that might be more effective or easier to use than current treatments. However, there are also potential risks, as with any new medicine. NXT007 might have side effects that are not yet fully known, or it might not work as well as expected. The study team will carefully monitor you for any side effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (19)

University of Colorado Hemophilia and Thrombosis Center
Verified postcode
Aurora, United States· Recruiting
University of Iowa Hospitals and Clinics
Verified postcode
Iowa City, United States· Recruiting
Universitätsklinikum Bonn
Verified postcode
Bonn, Germany· Recruiting
Eszak-Pesti Centrumkorhaz - Honvedkorhaz;Diabetologiai Szakrendeles
Unverified
Budapest, Hungary· Recruiting
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Verified postcode
Milan, Italy· Recruiting
IRCCS Istituto Clinico Humanitas
Verified postcode
Rozzano, Italy· Recruiting
Azienda Ospedaliera Universitaria Careggi
Verified postcode
Florence, Italy· Recruiting
Nara Medical University Hospital
Verified postcode
Kashihara-shi, Japan· Recruiting
Tokyo Medical University Hospital
Unverified
Shinjuku-Ku, Japan· Recruiting
Universitair Medisch Centrum Utrecht
Verified postcode
Utrecht, Netherlands· Recruiting
Kyung Hee University Hospital at Gangdong
Unverified
Gangdong-gu, South Korea· Recruiting
Chonnam National University Hwasun Hospital
Unverified
Hwasun-gun, South Korea· Recruiting

Common questions

What is haemophilia A?

Haemophilia A is a lifelong inherited condition where your blood doesn't clot properly due to a lack of a protein called Factor VIII, leading to easy bruising and bleeding.

What is the new medicine, NXT007?

NXT007 is an investigational medicine being tested in this study to see if it can effectively prevent bleeding in people with haemophilia A, similar to or better than standard Factor VIII.

Will I know if I'm getting NXT007 or Factor VIII?

No, this study is 'blinded,' meaning neither you nor your doctor will know whether you are receiving NXT007 or Factor VIII. This helps ensure the study results are unbiased.

How long will I need to be in the study?

The study involves a series of visits over a specific period, which will be fully explained by the study team before you decide to take part.

Can I stop participating in the study at any time?

Yes, you are free to withdraw from the study at any point, for any reason, without it affecting your current or future medical care.

How to find out more

Reference Study ID Number: WO45886 https://forpatients.roche.com/ No attachments to email below.

global-roche-genentech-trials@gene.com 888-662-6728 (U.S. Only) Official trial record

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