A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
This research study is looking at a new treatment called NXT007 for people with haemophilia A, a condition where blood doesn't clot properly. It's being compared to an existing treatment called emicizumab. The study is open to people aged 12 and older who have severe, moderate, or even mild haemophilia A, particularly if they have developed 'inhibitors' which make standard treatments less effective. Researchers want to understand if NXT007 is safe, how effective it is at preventing bleeding, and how it acts in the body, to potentially offer another helpful option for managing this condition.
At a glance
What is this study about?
This study is investigating a new medication called NXT007 for people living with haemophilia A. Haemophilia A is a condition that means your blood doesn't clot as it should, leading to longer bleeding and sometimes internal bleeding. People with haemophilia often take regular medication to help prevent these bleeds, a treatment approach known as 'prophylaxis'. This study aims to see if NXT007 can be an effective and safe option for preventing bleeds, similar to or better than an existing treatment called emicizumab.
The main goal is to find out how well NXT007 works compared to emicizumab. The researchers will be looking at several things, including how effective the treatments are at stopping bleeds, if there are any side effects (safety), and how the medications behave inside the body. This information is really important because it helps doctors understand which treatments are best for different people with haemophilia A.
The study includes people aged 12 and above. It's open to those with severe or moderate haemophilia A, and also to people with any severity (mild, moderate, or severe) who have developed 'inhibitors'. Inhibitors are antibodies that the body sometimes makes against treatment, making it less effective. Finding new treatments for people with inhibitors is particularly important as their options can be more limited.
Key takeaways
- Compares a new drug (NXT007) with an existing one (emicizumab) for haemophilia A.
- Aimed at people 12 years and older, including those with 'inhibitors'.
- Focuses on preventing bleeds, safety, and how the drugs work.
- Involves regular clinic visits and close medical monitoring.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, you generally need to be 12 years or older and have a confirmed diagnosis of haemophilia A. This includes people with severe or moderate forms of the condition, whether they have developed 'inhibitors' (which make some treatments less effective) or not. If you have mild haemophilia A, you can still participate, but only if you have developed chronic inhibitors that significantly reduce your natural clotting ability.
The doctors will also need to review your past medical records, including information about any previous treatments for haemophilia and bleeding episodes, especially over the last six months. If you currently take medication only when you have a bleed, you would need to be willing to switch to a regular preventative treatment with either NXT007 or emicizumab as part of the study.
There are also some reasons why you might not be able to join. For example, if you are allergic to any part of the study treatments or tests, or if you are taking certain other medications that could interfere with the study. Also, if you have certain heart conditions or are planning major surgery during the study period, you wouldn't be able to take part. The study team will discuss all these details with you carefully.
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?
- Have you been diagnosed with severe or moderate haemophilia A?
- If you have mild haemophilia A, do you also have 'inhibitors' that significantly reduce your clotting ability?
- Are you willing to switch to a regular preventative treatment if you're currently taking medication only when you bleed?
- Do you have any heart conditions or major surgeries planned during the study period?
What does participation involve?
If you decide to take part in this study, you will be given either the new medication NXT007 or the existing treatment emicizumab on a regular schedule, as a preventative measure against bleeding. This will be decided randomly, like flipping a coin. You will have regular visits to the clinic where doctors and nurses will monitor your health, perform various tests, and check for any side effects. You'll also be asked to keep a record of any bleeding episodes you experience.
The study involves several clinic visits over a period of time, where you'll have blood tests, physical examinations, and answer questions about how you're feeling. The exact number and frequency of visits will be explained by the study team. You'll be closely supported by the medical team throughout your participation. The total duration of the study for each person will vary, but it typically involves an initial treatment period followed by ongoing monitoring.
Potential risks and benefits
Locations (30)
- Center for Inherited Blood DisordersVerified postcodeOrange, United States· Recruiting
- University of Colorado Hemophilia and Thrombosis CenterVerified postcodeAurora, United States· Recruiting
- St Joseph's Children's Hospital of TampaVerified postcodeTampa, United States· Recruiting
- Innovative Hematology, Inc.Verified postcodeIndianapolis, United States· Recruiting
- University Of Iowa Hospitals And ClinicsVerified postcodeIowa City, United States· Recruiting
- Washington Center for Bleeding DisordersVerified postcodeSeattle, United States· Recruiting
- UZ LeuvenVerified postcodeLeuven, Belgium· Recruiting
- Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical SciencesVerified postcodeTianjin, China· Recruiting
- Hospices Civils de LyonVerified postcodeBron, France· Recruiting
- Universitätsklinikum BonnVerified postcodeBonn, Germany· Recruiting
- Debreceni Egyetem Klinikai Kozpont Nagyerdei CampusUnverifiedDebrecen, Hungary· Recruiting
- The Chaim Sheba Medical Center - PPDSUnverifiedRamat Gan, Israel· Recruiting
Common questions
What is haemophilia A?
Haemophilia A is a genetic condition where your blood doesn't clot properly due to a lack of a specific protein called Factor VIII, leading to longer bleeding after injuries.
What are 'inhibitors' in haemophilia?
Inhibitors are antibodies that your body can sometimes make against the treatment for haemophilia, which makes the standard medication less effective at stopping bleeds.
How will I know which treatment I'm getting?
You will be assigned to either NXT007 or emicizumab randomly, meaning you won't get to choose, and sometimes neither you nor your doctor will know which treatment you are on (this is called a 'blinded' study).
What kind of tests will I have?
You'll have regular physical exams, blood tests to check your clotting factors and general health, and you'll be asked about any bleeding episodes you experience.
Can I stop participating in the study?
Yes, you can leave the study at any time, for any reason. Your decision will not affect your future medical care.
How to find out more
Reference Study ID Number: BO45887 https://forpatients.roche.com/ No attachments to email below.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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