Not recruitingRare disease

Study on the Safety of DNL310 for Children with Hunter Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II). This is a rare genetic disorder that affects the body's ability to break down certain sugars. The study is testing a treatment called DNL310, which is a special type of protein designed to help manage the symptoms of Hunter Syndrome. The treatment is given as a solution through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein.

The purpose of the study is to evaluate the safety and how the body processes and responds to DNL310 in children with Hunter Syndrome. Participants in the study will receive the treatment over a period of time, and researchers will monitor them to see how they react to the treatment. This includes checking for any side effects and measuring changes in certain substances in the body that are related to the disease. The study will also look at how the treatment affects the progression of the disease and any improvements in the participants' daily functioning.

The study will take place over several weeks, with an initial period of 24 weeks, followed by longer-term follow-ups to ensure the safety and effectiveness of the treatment. Throughout the study, researchers will collect information on how the treatment affects the participants' health and any changes in their condition. This information will help determine if DNL310 is a safe and effective treatment option for children with Hunter Syndrome.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm the diagnosis of Hunter Syndrome (Mucopolysaccharidosis Type II).
Participants are categorized into different cohorts based on age and specific characteristics of the condition.
Step 2
Treatment administration
The treatment involves the administration of DNL310, a solution for infusion, given through an intravenous infusion.
The frequency and dosage are determined based on the specific cohort and individual needs.
Step 3
Monitoring and evaluation
Throughout the study, participants are monitored for any treatment-emergent adverse events and infusion-related reactions.
Regular assessments are conducted to evaluate changes in urine total glycosaminoglycan concentration and other health indicators.
Step 4
Safety extension
The study includes a safety extension period up to Week 104 to further monitor the safety and effects of the treatment.
Participants continue to receive the treatment and undergo regular evaluations during this period.
Step 5
Open-label extension
An open-label extension is available up to Week 261, allowing for continued treatment and monitoring.
This phase aims to gather long-term data on the safety and effectiveness of the treatment.
Step 6
Final assessment
At the end of the study, a final assessment is conducted to evaluate the overall impact of the treatment on the condition.
The study is estimated to conclude by July 2027.

Who can join the trial?

7 criteria

Must have a confirmed diagnosis of **Hunter Syndrome** (also known as **Mucopolysaccharidosis Type II** or **MPS II**).
For **Cohort A**: Must be between 5 to 10 years old and have **neuronopathic MPS II** (a form of MPS II that affects the nervous system).
For **Cohort B**: Must be between 1 to 18 years old and have either **non-neuronopathic**, **neuronopathic MPS II**, or an unknown type of MPS II.
For **Cohort C**: Must be younger than 4 years old with **neuronopathic MPS II**. This group can also include participants aged 4 to 18 years if they are a blood relative with the same genetic mutation as a participant under 4 years old who is joining the study.
For **Cohort D**: Must be 18 years old or younger, have **nMPS II** (neuronopathic MPS II) or **nnMPS II** (non-neuronopathic MPS II), and have an enlarged liver (**hepatomegaly**). Must not have taken the usual enzyme replacement therapy (**ERT**).
For **Cohort E**: Must have **neuronopathic MPS II** and be 6 years or older at the time of screening, or have **non-neuronopathic MPS II** and be younger than 6 or 17 years or older at screening. Participants with a history of stem cell transplantation or gene therapy must have completed at least 48 weeks in a previous study (Study DNLI-E-0001).
If receiving **intravenous iduronate 2-sulfatase (IDS) ERT** (a type of enzyme replacement therapy), must have tolerated at least 4 months of this therapy right before the screening.

Who cannot join the trial?

3 criteria

Participants cannot be female. Only males are allowed to join the study.
Participants must be within a specific age range, which is not specified here, but typically means they must be children.
Participants must not belong to any vulnerable population that is not specified here, but generally refers to groups that might need special protection or care.

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Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands

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Investigated drugs

DNL310 is an experimental medication being studied for its safety and how it behaves in the body in children with Hunter Syndrome, also known as MPS II. This study aims to understand how well children tolerate this medication and to gather information on its effects.

Iduronate-2-Sulfatase Fused To A Fc Polypeptide That Binds To The Human Transferrin Receptor

What is already known about the treatment

DNL310 – DNL310 is administered intravenously, meaning it is delivered directly into the bloodstream through a vein. It is currently being studied in clinical trials, specifically in a Phase 1/2 study, to assess its safety and effects in children with Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II). The main therapeutic indication for DNL310 is to treat Hunter Syndrome, a rare genetic disorder. At the molecular level, DNL310 is designed to cross the blood-brain barrier and deliver therapeutic enzymes to the central nervous system, potentially addressing neurological symptoms of the disease. It is classified pharmacologically as an enzyme replacement therapy.

Investigated diseases

Hunter Syndrome – Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II), is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This enzyme deficiency leads to the accumulation of complex sugar molecules called glycosaminoglycans in the body's cells. As these molecules build up, they cause progressive damage to various tissues and organs. Symptoms often include developmental delays, joint stiffness, and distinctive facial features. Over time, individuals may experience hearing loss, heart problems, and difficulties with mobility. The progression and severity of symptoms can vary widely among those affected.