Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome

This trial is exploring a new treatment, DNL310, for children who have Hunter Syndrome (also known as MPS II). It's a very early stage study, called a Phase 1/2, which means it's one of the first times this medicine is being given to people. The main goals are to check how safe the medicine is by looking for any side effects and reactions during infusions. Researchers also want to understand how the medicine works in the body and if it helps by measuring certain substances in urine. It's a long-term study, with different periods of observation, to get a full picture of the medicine's effects over time. The study is open to male participants within a specific age range.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Denali Therapeutics Inc.

Enrolment target

Start

06 Jun 2024

Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II])

What is this study about?

This study is about a new medicine called DNL310 for a condition known as Hunter Syndrome, or MPS II. Hunter Syndrome is a rare genetic condition that mostly affects boys. It happens when the body doesn't make enough of a special enzyme that breaks down certain sugar molecules. When these molecules build up, they can cause problems in different parts of the body, like organs, bones, and joints.

This trial is a 'Phase 1/2' study, which is an early but important step. It's designed to first make sure the new medicine, DNL310, is safe for people to take. Researchers will be carefully watching for any unexpected reactions or side effects. At the same time, they will also be trying to understand how the medicine moves through the body, how long it stays there, and if it starts to have any positive effects on the condition. This includes checking substances in the urine that are linked to Hunter Syndrome.

The study will also look at how DNL310 might affect the overall health and development of the children taking part, including their daily living skills. It's a long-term study, so researchers can see how the medicine works over many months, which helps them understand its full potential and safety profile.

Key takeaways

This is an early-stage study for a new medicine, DNL310, for Hunter Syndrome.
The main goals are to check the medicine's safety and how it works in the body.
Participants will receive the medicine through infusions and have regular health checks.
The study has long-term follow-up periods, potentially lasting several years.
It's only open to male participants with Hunter Syndrome, currently aged 18 and older.

Who may be eligible?

This study is specifically looking for male participants. The age range for taking part is quite broad, from 18 years old and up, meaning adults are currently being considered.

The main condition for joining is having Hunter Syndrome (MPS II). There will also be other health checks and requirements to make sure it's safe for participants to receive the new 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.

Are you or the participant a male?
Do you or the participant have a diagnosis of Hunter Syndrome (MPS II)?
Are you or the participant at least 18 years old?
Are you or the participant able to attend regular appointments for infusions and check-ups?

Answer every question to see your result.

What does participation involve?

If you or your child decides to take part, you'll be giving a new medicine called DNL310. This medicine is given as an infusion, which means it goes directly into a vein. The study involves regular visits to the study centre, where doctors and nurses will monitor your health very closely. They will check for any side effects, take blood and urine samples, and carry out other assessments to see how the medicine is working and how you are feeling.

The study has different observation periods over a long time. Initially, participants will be monitored for 24 weeks. This is followed by a 'safety extension' period up to 104 weeks (about two years), and then an 'open-label extension' which could last up to 261 weeks (about five years). This long follow-up allows the researchers to gather a lot of information about the medicine's longer-term effects and safety.

Potential risks and benefits

Like all medicines, DNL310 might have side effects, and some may not be known yet as it's a new treatment. The study team will carefully monitor participants for any reactions, especially during and after infusions. However, there's also a chance that DNL310 could help to ease some of the symptoms of Hunter Syndrome or slow down its progression by addressing the underlying cause. It's important to remember that participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What is Hunter Syndrome?

Hunter Syndrome is a rare genetic condition where the body can't break down certain complex sugars, causing them to build up and damage various organs and tissues.

What is DNL310?

DNL310 is a new medicine being studied to treat Hunter Syndrome. It is designed to replace the missing enzyme that causes the condition.

Why is this study only for males?

Hunter Syndrome primarily affects males. This study is focused on this group to understand how DNL310 works for those most commonly affected.

How long will the study last?

The study has different phases of monitoring, starting with 24 weeks, extending to about two years, and then potentially up to five years in total.

Will I receive the new medicine if I join?

Yes, all participants in this particular study will receive the new medicine, DNL310, as it is an 'open-label' study.