A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
This study is called a Phase 1/2 clinical trial and is looking at a new medicine, NXT007, for people with moderate or severe haemophilia A. Haemophilia A is a condition where blood doesn't clot properly. The main goals are to understand if NXT007 is safe to use, how well people tolerate it, how the body handles the medicine, and if it helps prevent or treat bleeding episodes. The study will involve adult, teenage, and younger male participants. Researchers will start by giving different doses to adult and teenage participants to find the best amount, and then a number of younger boys will also take part. There are no specific details provided about the number of visits, how long the study will last, or specific treatments beyond NXT007, but participants would likely have regular check-ups and monitoring.
At a glance
What is this study about?
This study is a clinical trial, which means it's a carefully planned medical research study involving people. It's designed to test a new medicine called NXT007 for individuals who have moderate or severe haemophilia A. Haemophilia A is a lifelong condition, mostly affecting males, where their blood doesn't clot properly due to a lack of a specific protein called Factor VIII. This can lead to longer bleeding episodes and sometimes internal bleeding, which can damage joints and organs.
The study is split into two main parts. The first part will look at adult and teenage male participants. They will be given increasing doses of NXT007 to find out the safest and most effective amount. This helps researchers understand how the body reacts to different amounts of the medicine. The second part of the study will involve younger boys with haemophilia A, and they will receive multiple doses. Throughout both parts, the researchers will be closely watching for any side effects, how well the medicine is tolerated, how it moves through the body, and importantly, if it helps to control bleeding.
The overall aim is to gather as much information as possible about NXT007. This includes understanding if it's safe, how it works in the body (its 'pharmacokinetics' and 'pharmacodynamics'), whether the body develops antibodies against it (known as 'immunogenicity'), and ultimately, if it's effective in treating or preventing bleeding in people with haemophilia A. This kind of research is vital for developing new and improved treatments for serious conditions like haemophilia A.
Key takeaways
- This study is testing a new medicine (NXT007) for haemophilia A.
- It's for males aged 2 to 59 with moderate or severe haemophilia A.
- Both those with and without Factor VIII inhibitors may be eligible.
- The study aims to check the medicine's safety and how well it works.
- Participants will receive NXT007 and have regular health check-ups.
Who may be eligible?
This study is looking for male participants who have been diagnosed with either severe or moderate haemophilia A. This means your Factor VIII level is either very low (less than 1%) or moderately low (between 1% and 5%). You might have Factor VIII inhibitors (which are antibodies that stop Factor VIII from working) or you might not – both groups can be considered for the study. If you do have inhibitors, you'll need to be using a specific medicine called rFVIIa for bleeding or be willing to switch to it.
There are also some important health checks you'd need to pass. Your blood counts, liver, and kidney functions would need to be within a healthy range. If you're an adult or teenager in the first part of the study, your kidney function needs to be good. For younger boys in the second part of the study, there's a slightly different kidney function requirement. If you've had treatment to get rid of inhibitors (called immune tolerance induction) in the past, it must have been successful at least 5 years ago, and there should be no sign of the inhibitors coming back.
Finally, the study is for males between 2 and 59 years old. The researchers will also need to review your bleeding history from the 24 weeks before you join the study to understand your condition better.
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 male?
- Are you between 2 and 59 years old?
- Do you have a diagnosis of severe or moderate haemophilia A?
- Are your blood counts, liver, and kidney functions generally healthy?
- If you have Factor VIII inhibitors, are you using or willing to use rFVIIa for bleeds?
- If you had inhibitor treatment (ITI) previously, was it successful at least 5 years ago?
What does participation involve?
If you decide to take part in this study, you will receive a new medicine called NXT007. The study is divided into two main parts. In the first part, adult and teenage males will be given different doses of the medicine to find the safest and most effective amount. In the second part, younger boys will receive regular doses.
Throughout your time in the study, you will have regular check-ups with the study team. These visits will likely involve blood tests to check how your body is responding to the medicine and to monitor your overall health, including your liver and kidney function. You'll also need to tell the study team about any bleeding episodes you experience. The exact number of visits and the total length of time you'll be involved in the study are not specified here, but the study team will provide you with a detailed schedule. The goal is to carefully monitor your health and the effects of NXT007.
Potential risks and benefits
Locations (14)
- UC Davis Cancer CenterVerified postcodeSacramento, United States· Recruiting
- Georgetown Uni Medical CenterVerified postcodeWashington D.C., United States· Withdrawn
- Indiana Hemophilia & Thrombosis centerVerified postcodeIndianapolis, United States· Recruiting
- University of Iowa Hospitals and Clnics Dept of PediatricsVerified postcodeIowa City, United States· Recruiting
- British Columbia Children's HospitalVerified postcodeVancouver, Canada· Recruiting
- Hamilton Health Sciences CorporationVerified postcodeHamilton, Canada· Recruiting
- IRCCS Ca' Granda Ospedale Maggiore PoliclinicoVerified postcodeMilan, Italy· Recruiting
- Istituto Clinico HumanitasVerified postcodeRozzano (MI), Italy· Recruiting
- Auckland Cancer Trial CentreVerified postcodeAuckland, New Zealand· Recruiting
- Uniwersyteckie Centrum KliniczneCity onlyGda?sk, Poland· Recruiting
- Instytut Hematologii i TransfuzjologiiVerified postcodeWarsaw, Poland· Recruiting
- Hospital Sant Joan de DeuVerified postcodeEsplugues de Llobregat, Spain· Recruiting
Common questions
What is haemophilia A?
Haemophilia A is a genetic bleeding disorder, mainly affecting males, where the blood doesn't clot properly due to a lack of a protein called Factor VIII.
What is NXT007?
NXT007 is a new experimental medicine being tested to see if it can help people with haemophilia A manage their condition and reduce bleeding.
Can women or girls join this study?
No, this particular study is only for males between 2 and 59 years old.
What are Factor VIII inhibitors?
Factor VIII inhibitors are antibodies that a person's immune system can develop, which then stop the Factor VIII given as treatment from working properly.
Will I know if the medicine is working?
The study team will monitor your bleeding episodes and conduct regular tests to understand how NXT007 is affecting your condition. They will discuss your progress with you.
How to find out more
Reference Study ID Number: WP44714 https://forpatients.roche.com/
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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