A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
This important study is testing a new medicine called tividenofusp alfa (DNL310) for people living with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. This condition affects different parts of the body, and sometimes the brain too. The new medicine is designed to get into the brain, which many current treatments don't do effectively. Researchers want to find out if tividenofusp alfa is a safe and effective treatment. The study will compare it against idursulfase, which is a common treatment already used for MPS II. Participants will be randomly given either the new medicine or idursulfase. This study is for children and young adults between 2 and 25 years old.
At a glance
What is this study about?
This study is focused on a health condition called Mucopolysaccharidosis Type II, often shortened to MPS II, and also known as Hunter syndrome. This is a rare genetic condition where the body can't properly break down certain complex sugars. These sugars then build up in cells, causing problems in various parts of the body like bones, joints, heart, and sometimes the brain.
The main goal of this particular study is to evaluate a new medicine called tividenofusp alfa (DNL310). Researchers believe this new medicine might be able to reach the brain more effectively than some existing treatments. This is important because in some forms of MPS II, the brain can be affected, leading to developmental delays. The study wants to see how well tividenofusp alfa works and if it's safe compared to a medicine currently used for MPS II, called idursulfase.
This is a 'Phase 2/3' study, which means it's one of the later stages of testing a new medicine. It tells us that initial tests have shown promise, and now researchers are looking at a larger group of people to confirm its benefits and side effects. By taking part, people could help scientists learn more about MPS II and potentially discover better treatments for those living with the condition.
Key takeaways
- This study is testing a new medicine for Hunter syndrome (MPS II).
- It compares the new medicine with an existing treatment to see if it's safe and effective.
- It's for children and young adults with MPS II, aged 2-25.
- Participation involves regular clinic visits, receiving medication, and various tests.
- You will receive expert care if you choose to take part.
- You can stop participating at any time if you wish.
Who may be eligible?
This study is looking for children and young adults aged between 2 and 25 years old who have a confirmed diagnosis of MPS II (Hunter syndrome). Depending on their age and the type of MPS II they have, they might be able to join. For example, younger children (under 6) would typically need the 'neuronopathic' form where the brain is affected, while older children and young adults (6 to 25 years old) would typically have the 'non-neuronopathic' form where the brain isn't as affected.
Participants should also not have received other enzyme replacement therapy (ERT) for a long time before the study, or they need to have been on a stable dose of idursulfase (a standard treatment) for at least four months. This helps ensure that any changes seen during the study are due to the new medicine or the comparison treatment being tested.
There are also some reasons why someone might not be able to join. For example, if they have other genetic conditions causing developmental delays, or if they've had certain gene therapies or stem cell treatments. Also, if they've received specific brain-targeted MPS treatments recently, or if they can't have procedures like spinal taps (lumbar punctures) or MRI scans, they wouldn't be able to take part. It's important not to be taking part in other drug studies at the same time.
- I am between 2 and 25 years old.
- I have been diagnosed with MPS II (Hunter syndrome).
- I haven't recently received certain other intensive MPS treatments.
- I don't have other genetic conditions causing developmental delays.
- I am able to have MRI scans and, if needed, a spinal tap.
- I am not currently taking part in another drug study.
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part, you'll be randomly assigned to receive either the new medicine, tividenofusp alfa, or the standard treatment, idursulfase. This will be done in a way that neither you, your doctors, nor the study team will know which treatment you are getting. You will receive your assigned medicine regularly through an intravenous (IV) drip, which means it will be given directly into your vein.
Throughout the study, you'll have regular visits to the clinic for various health checks. These will include physical examinations, blood tests, urine tests, and sometimes a spinal tap (lumbar puncture) to check fluid around your brain and spine. You will also have MRI scans to look at your brain. The study will last for a period of time, and after the main part, some participants might have the option to continue receiving DNL310 (the new medicine) or idursulfase in an open-label extension, where everyone knows what treatment they are getting. The total duration of participation, including follow-up, will be discussed in detail by the study team.
Potential risks and benefits
Locations (32)
- UCSF Benioff Children's Hospital OaklandOakland, United States· Recruiting
- Ann and Robert H Lurie Children's Hospital of ChicagoChicago, United States· Recruiting
- Hackensack University Medical CenterHackensack, United States· Recruiting
- UNC Children's Research InstituteChapel Hill, United States· Recruiting
- Cincinnati Children's Hospital Medical CenterCincinnati, United States· Recruiting
- Children's Hospital of PhiladelphiaPhiladelphia, United States· Recruiting
- The University of Texas Medical School at HoustonHouston, United States· Recruiting
- University of Utah, PPDSSalt Lake City, United States· Recruiting
- Sanatorio Mater DeiBuenos Aires, Argentina· Recruiting
- Women's and Children's HospitalNorth Adelaide, Australia· Withdrawn
- UZ AntwerpenAntwerp, Belgium· Recruiting
- Universitair Ziekenhuis BrusselJette, Belgium· Recruiting
+20 more sites — see the official record for the full list.
Common questions
What is MPS II (Hunter syndrome)?
MPS II, or Hunter syndrome, is a rare genetic condition where the body can't break down certain complex sugars, which then build up and cause health problems.
What is tividenofusp alfa (DNL310)?
It's a new medicine being tested that aims to help in MPS II, especially by reaching the brain more effectively than some current treatments.
Will I know which medicine I'm getting?
No, during the main part of the study, neither you nor your doctors will know if you're receiving the new medicine or the standard one. This is called 'double-blind'.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time without having to give a reason, and it won't affect your future medical care.
How long will the study last?
The study has a main treatment period, and some people may then have the option to continue onto an extended treatment phase. The full duration will be explained by the study team.
How to find out more
Clinical Trials at Denali Therapeutics
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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