Active not recruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

This study is looking at an investigational medicine called tividenofusp alfa (also known as DNL310) for children and young people with Hunter Syndrome (Mucopolysaccharidosis II). Hunter syndrome is a rare genetic condition that can affect many parts of the body, including the brain. This new medicine is designed to get into the brain, aiming to help with both the physical and neurological symptoms. Researchers want to understand if it's safe, how much is needed, and how it affects the body. Participants who are doing well on the treatment may have the opportunity to continue receiving it in longer-term safety studies.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Denali Therapeutics Inc.

Enrolment target

Start

16 Jul 2020

Estimated completion

01 Feb 2031

Mucopolysaccharidosis II

What is this study about?

This research study is investigating a new medication called tividenofusp alfa (also known as DNL310) for children and young people who have Hunter syndrome, a condition also known as Mucopolysaccharidosis II (MPS II). Hunter syndrome is a rare inherited disorder where the body is missing an important enzyme. This enzyme is crucial for breaking down certain sugar molecules, and without it, these molecules build up in the body, which can lead to various health problems affecting many organs, and sometimes the brain.

The study medicine, tividenofusp alfa, is a special kind of enzyme replacement therapy. What makes it different is that it's designed to cross into the brain, which many current treatments for Hunter syndrome don't do effectively. This means it has the potential to help with both the physical symptoms (like effects on organs and bones) and the neurological symptoms (those affecting the brain and nervous system) of the condition.

The main goals of this study are to check if the new medicine is safe for young people with Hunter syndrome, and to understand how it behaves in the body – for example, how much is absorbed and how long it stays active. By studying these aspects, researchers hope to determine if tividenofusp alfa could be an effective new treatment option that addresses a broader range of Hunter syndrome symptoms.

Key takeaways

This study is testing a new medicine (tividenofusp alfa) for Hunter syndrome.
The medicine aims to treat both physical and brain-related symptoms of Hunter syndrome.
It's for male children and young people up to 18 years old with Hunter syndrome.
Researchers want to understand the medicine's safety and how it works in the body.
All participants in this study will receive the active investigational treatment.

Who may be eligible?

This study is specifically for male children and young people up to 18 years old who have been diagnosed with Hunter syndrome (MPS II). There are different groups within the study that might be suitable depending on the specific type of Hunter syndrome they have (whether it affects the brain or not), their age, and whether they've had previous treatments. For example, some groups are for younger children, while others are for those who haven't received certain standard treatments before.

To be considered, participants must generally be in stable health without other serious medical conditions that could interfere with the study. If they are already receiving a standard enzyme replacement therapy, they would need to have been on it for at least four months and be tolerating it well. Importantly, participants cannot have received certain brain-targeted MPS II treatments recently (within 3 to 6 months, depending on age), and generally cannot have had gene therapy or stem cell therapy for MPS II, with some specific exceptions.

There are also a few conditions that would prevent someone from joining, such as significant bleeding problems, a history of stroke or severe seizures, or if they cannot safely undergo procedures like a lumbar puncture (spinal tap). The study team will carefully review each potential participant's medical history to ensure their safety throughout the trial.

Quick self-check

Is the person male with a confirmed diagnosis of Hunter syndrome (MPS II)?
Is the person 18 years old or younger?
Does the person have a stable health condition overall?
Has the person not received certain brain-targeted MPS II treatments in the last 3-6 months (depending on age)?
Does the person not have any significant bleeding issues or a history of severe stroke?
Is the person able to safely undergo all required study procedures?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

Taking part in this study would involve regular visits to a study clinic. During these visits, the research team would perform various assessments. This includes physical examinations, taking blood and urine samples, and possibly undergoing imaging scans or other specialised tests to check how the study medicine is affecting the body and the condition. The study medicine, tividenofusp alfa, would be given to participants, and the team would monitor how well it's tolerated.

The total duration of taking part in the main study is not specified but is typically several months. After this initial period, if the participant and their doctors feel they are benefiting from the treatment, there may be opportunities to continue receiving the medicine in longer-term safety and open-label (where everyone receives the active drug) extension studies. This allows for ongoing evaluation of the medicine's effects over a longer time.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new investigational treatment that might help with symptoms of Hunter syndrome, especially those affecting the brain, before it's widely available. However, there are also potential risks involved. The study medicine, tividenofusp alfa, is investigational, meaning its full safety profile and side effects are not yet completely known. There could be side effects or allergic reactions, or the treatment might not be effective. All medical procedures and assessments also carry their own small risks. Participants are free to withdraw from the study at any time, for any reason, without it affecting their usual medical care.

Locations (7)

UCSF Benioff Children's Hospital
Oakland, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
UNC Children's Research Institute
Chapel Hill, United States
UPMC | Children's Hospital of Pittsburgh
Pittsburgh, United States
McGill University Health Centre - Royal Victoria Hospital
Montreal, Canada
Erasmus Medical Center
Rotterdam, Netherlands
St Mary's Hospital, Manchester Academic Health Science Centre
Manchester, United Kingdom

Common questions

What is Hunter syndrome?

Hunter syndrome is a rare genetic condition mostly affecting males, where the body can't break down certain sugar molecules, leading to them building up and causing various health problems.

What is tividenofusp alfa?

It's an investigational medicine designed to replace a missing enzyme in Hunter syndrome patients, specifically developed to reach the brain as well as other parts of the body.

Who can join this study?

This study is for male children and young people up to 18 years old with a confirmed diagnosis of Hunter syndrome, meeting specific age and medical criteria.

Will I get the study medicine or a placebo?

This is an 'open-label' study, meaning all participants who join will receive the active study medicine, tividenofusp alfa.

Can I stop participating in the study at any time?

Yes, you have the right to withdraw from the study at any point without it affecting your child's medical care.