Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
This study is gathering information about how well Octapharma's haemophilia A treatments, named Nuwiq, Octanate, and Wilate, are working for patients in everyday use. It focuses on people with severe haemophilia A, especially those who are newly diagnosed or have had very little treatment before. Researchers want to see if these treatments are effective, safe, and how they are used by doctors in the real world. A key area of interest is understanding how these treatments might affect the development of 'inhibitors', which are antibodies the body can sometimes make against the treatment. This is a non-interventional study, meaning patients will receive their usual care, and researchers will simply collect information on their progress.
At a glance
What is this study about?
This study is gathering important information about certain medicines used to treat a condition called haemophilia A. Specifically, it's looking at three medicines made by Octapharma: Nuwiq, Octanate, and Wilate. Haemophilia A is a rare inherited bleeding disorder where your blood doesn't clot properly. These medicines work by replacing a natural clotting factor that's missing.
The main goal is to understand how well these medicines work, how safe they are, and how doctors use them in real life. They are particularly interested in patients who are newly diagnosed with severe haemophilia A or who have had very little treatment before. These include young children, who are often called 'previously untreated patients' (PUPs), and 'minimally treated patients' (MTPs). Because haemophilia A is rare, clinical trials usually don't involve a huge number of patients, so this study helps collect more information on how these medicines perform in a broader group of people.
One of the key things the study will look at is something called 'inhibitor development'. Sometimes, when people with haemophilia A receive these treatments, their body can develop antibodies, called inhibitors, that stop the medicine from working properly. Understanding how often this happens with these specific Octapharma treatments, especially in patients who are new to treatment, is really important for improving care. This study won't change your treatment; it will just observe what happens during your regular care.
Key takeaways
- This study collects real-life information on haemophilia A medicines.
- It focuses on patients new to treatment or with very little previous treatment.
- The goal is to understand effectiveness, safety, and how medicines are used.
- A key interest is how often 'inhibitors' develop.
- You won't receive new medicines or have extra hospital visits.
- Your regular care will continue as normal, with information being collected.
Who may be eligible?
This study is looking for both male and female patients of any age and background who have severe haemophilia A. This means your 'factor VIII' levels are very low (less than 1%).
You would be considered for the study if your doctor has already decided that you will be treated with one of Octapharma's factor VIII medicines (Nuwiq, Octanate, or Wilate) before signing up for the study.
Crucially, you should either not have had any treatment with factor VIII medicines before, or you've had very little treatment – specifically, less than 5 doses in total. If you have had previous treatment, the study would be interested if you haven't developed inhibitors, or if you did develop an inhibitor but are continuing to use the same Octapharma 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.
- I have severe haemophilia A.
- My doctor has decided I will take Nuwiq, Octanate, or Wilate.
- I have had no previous Factor VIII treatment OR less than 5 doses in total.
- If I had some treatment, I didn't develop an inhibitor OR I developed an inhibitor and am still using the same Octapharma medicine.
- I am able to (or my parent/guardian is able to) provide written consent.
What does participation involve?
If you decide to take part, you won't be given any new or experimental medicines. You will continue with your regular treatment plan for haemophilia A as decided by your doctor using one of the Octapharma medicines (Nuwiq, Octanate, or Wilate). The study simply involves your doctors collecting information from your routine hospital visits, such as how often you have bleeds, what dose of medicine you take, and any side effects you might experience. They will also keep an eye on your FVIII levels and check for inhibitor development, which they would normally do as part of your regular care. There are no additional visits or tests required specifically for this study. The information collected will be detailed in your medical notes, and then shared with the study researchers. The total duration of your participation will depend on your individual treatment plan and how long your doctor feels it's beneficial to track this information, but it will be part of your ongoing care.
Potential risks and benefits
Locations (59)
- University of Miami Miller School of MedicineVerified postcodeMiami, United States· Recruiting
- Ochsner Clinic FoundationVerified postcodeNew Orleans, United States· Active not recruiting
- St. Louis University , 221 North Grand BoulevardVerified postcodeSt Louis, United States· Not yet recruiting
- Cure 4 The Kids Foundation Children's Specialty CenterVerified postcodeLas Vegas, United States· Withdrawn
- The University of Texas Health Science Center at San AntonioVerified postcodeSan Antonio, United States· Not yet recruiting
- CENIDORVerified postcodeSalta, Argentina· Active not recruiting
- Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of HemophiliaVerified postcodeBaku, Azerbaijan· Withdrawn
- Republican Scientific Center for Radiation Medicine and Human EcologyVerified postcodeHomyel, Belarus· Withdrawn
- Republican Scientific and Practical Centre of Children Oncology, Hematology and ImmunologyVerified postcodeMinsk, Belarus· Recruiting
- Hôpital Universitaire des Enfants Reine FabiolaVerified postcodeBrussels, Belgium· Recruiting
- Cliniques Universitaires Saint-LucVerified postcodeBrussels, Belgium· Recruiting
- Cliniques Universitaires Saint-LucVerified postcodeBrussels, Belgium· Recruiting
Common questions
What is haemophilia A?
Haemophilia A is an inherited condition where your blood doesn't clot properly because you're missing a special protein called Factor VIII. This can lead to longer bleeding after injuries or sometimes spontaneous bleeding.
What are 'inhibitors'?
Inhibitors are antibodies that your body can sometimes develop against the Factor VIII medicine you receive. If you develop inhibitors, the medicine might not work as well to stop bleeding.
Will I get a new medicine if I join?
No, you will not be given a new medicine. You will continue to take the Factor VIII medicine (Nuwiq, Octanate, or Wilate) that your doctor has already prescribed for your haemophilia A.
Do I need to make extra hospital visits?
No, you do not need to make any extra hospital visits. The study will collect information from your regular, planned check-ups and treatment.
Can I stop participating in the study?
Yes, you can choose to stop being part of the study at any time, and this will not affect the medical care you receive from your doctor.
How to find out more
Cristina Solomon, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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