Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
This study is called an 'open-label study' looking at a treatment named AX 250 for a genetic condition called MPS IIIB. It's for people who have already completed an earlier study with AX 250. The main aim is to keep checking how safe AX 250 is and how well it works over a longer time, up to three more years. Doctors will give the treatment directly into the fluid around the brain every two weeks. They will be looking at things like thinking skills, how well people communicate, their daily abilities, and their overall quality of life. They will also do imaging scans and blood tests to understand the disease better. The study will happen in different hospitals across various countries.
At a glance
What is this study about?
This research study is about an ongoing treatment called AX 250 for people living with a rare genetic condition called MPS IIIB. MPS IIIB can affect development and brain function. This particular study is for people who have already been receiving AX 250 for a long time in a previous study. Think of it as a continuation, where doctors want to understand the long-term effects of this treatment.
The main goals are to keep a close eye on how safe AX 250 is over a longer period and to see if it continues to help with important aspects of daily life. This includes looking at how people think and learn, how they communicate, their abilities to do everyday tasks, and their general well-being. Doctors will also use special scans and tests to track changes in the body and brain.
AX 250 is given directly into the fluid that surrounds the brain, which is a special way to make sure the treatment reaches the right place. By continuing this study, researchers hope to gather more important information to help people with MPS IIIB in the future.
Key takeaways
- This study is a long-term look at AX 250 for MPS IIIB.
- It's only for people who have already been receiving AX 250 in a previous study.
- The treatment is given directly to the fluid around the brain every two weeks.
- The study aims to check safety and how it affects thinking and daily life over three more years.
- Regular visits include health checks, various tests, and brain scans.
- Joining is voluntary, and you can leave at any time.
Who may be eligible?
This study is specifically designed for people who have already been part of an earlier study (called Study 250-202) and have received AX 250 for 240 weeks. You'd need to start this new study within eight weeks of finishing the previous one.
To join, a parent or legal guardian needs to give their written permission, and if the person taking part is able, they will also need to agree. It's important that you can follow the study instructions. If you're a female who could become pregnant, you'll need to have a negative pregnancy test at the start and during the study.
However, you wouldn't be able to join if your thinking development is very low (like an 18-month old or less) or if your overall development is very low, and you haven't shown any signs of getting better in the earlier study. You also can't join if you've had other special treatments for MPS IIIB like stem cell or gene therapy, or if you have poorly controlled seizures, problems with fluid in your brain, or have recently taken other experimental medicines. The study doctor will make the final decision based on your health and whether they think it would be good for you.
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.
- Have you already completed Study 250-202 with AX 250 treatment?
- Can you start this new study within 8 weeks of finishing the previous one?
- Are you able to regularly attend hospital visits for treatment and check-ups?
- Do you have a history of well-controlled seizures (if applicable)?
- Have you avoided other special treatments for MPS IIIB like gene or stem cell therapy?
What does participation involve?
If you join this study, you will continue to receive the AX 250 treatment every two weeks. The treatment is given directly into the fluid around your brain. This study is expected to last for up to three years (144 weeks), but the total time you'd be involved builds on the previous study you've already completed.
During this time, you'll have regular visits to the hospital. At these visits, doctors and nurses will check your safety by doing physical exams, taking blood and urine samples, and recording any new health issues. They will also do various assessments to understand how the treatment is affecting you. This includes tests for your thinking skills (neurocognitive function), how you communicate, and how well you manage daily activities (adaptive behavior). They will also ask about your quality of life, perform imaging scans of your brain, and check for certain markers in your body related to the disease.
Potential risks and benefits
Locations (6)
- UCSF Benioff Children's Hospital OaklandVerified postcodeOakland, United States
- UPMC Children's Hospital PittburghVerified postcodePittsburgh, United States
- Fundación Cardio Infantil - Instituto de CardiologíaVerified postcodeBogotá, Colombia
- University Medical Center Hamburg-EppendorfVerified postcodeHamburg, Germany
- Gazi University Faculty of MedicineVerified postcodeAnkara, Turkey (Türkiye)
- Great Ormond Street Hospital For Children, NHS Foundation TrustVerified postcodeLondon, United Kingdom
Common questions
What is MPS IIIB?
MPS IIIB is a rare genetic condition that affects how the body breaks down certain sugars, leading to their build-up and problems, particularly in the brain.
Why is this study only for people already getting AX 250?
This study is a continuation of an earlier one to see how AX 250 works over a much longer period, specifically for those who have already started the treatment.
How is AX 250 given?
AX 250 is given directly into the fluid surrounding the brain. This is done to make sure the medicine reaches where it needs to go.
How long will I be in this study?
If you join, you could be in this study for up to an additional three years, continuing the treatment every two weeks.
What kind of tests will I have?
You'll have assessments for thinking abilities, communication, daily life skills, quality of life, brain scans, blood tests, and regular health checks.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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