Study on the Effectiveness and Safety of DNL310 vs Idursulfase for Children and Young Adults with Mucopolysaccharidosis Type II

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What is this study about?

This clinical trial is focused on studying a rare genetic condition called Mucopolysaccharidosis Type II, also known as MPS II or Hunter syndrome. This condition affects the body’s ability to break down certain sugars, leading to various health issues. The study involves two treatments: Tividenofusp Alfa (DNL310) and Idursulfase. Tividenofusp Alfa is a new treatment being tested, while Idursulfase is an existing treatment used for MPS II. Both treatments are given as a solution through a vein, known as an infusion.

The purpose of the study is to compare the effectiveness and safety of Tividenofusp Alfa with Idursulfase in children and young adults with MPS II. The study will look at how these treatments affect the brain and behavior, as well as other health aspects. Participants will receive one of the treatments and will be monitored over a period of time to see how their condition changes. The study will include regular check-ups and assessments to track progress and any side effects.

Participants in the study will be divided into two groups based on their age and specific type of MPS II. The study will last for up to 96 weeks, during which various health measurements will be taken, such as changes in certain substances in the body and improvements in daily activities. The goal is to gather information that could lead to better treatment options for those living with MPS II.

1 joining the study

Upon joining the study, participants are divided into two groups based on age: Cohort A (ages 2 to <6 years) and Cohort B (ages 6 to <26 years).

Participants must have a confirmed diagnosis of Mucopolysaccharidosis Type II (MPS II).

2 initial treatment phase

Participants receive treatment with either Tividenofusp Alfa (DNL310) or Idursulfase.

Both medications are administered as a solution for infusion through intravenous use.

3 treatment schedule

The treatment is administered regularly over a period of time, with specific intervals determined by the study protocol.

Participants in Cohort A are monitored for changes in cerebrospinal fluid (CSF) concentration of heparan sulfate (HS) at Week 24.

4 monitoring and assessments

Participants undergo various assessments to evaluate the effectiveness of the treatment.

For Cohort A, assessments include changes in adaptive behavior using the Vineland-3 scale at Week 96.

For Cohort B, assessments include changes in distance walked in the 6-minute walk test (6MWT) at Week 48.

5 additional evaluations

Both cohorts are evaluated for changes in urine concentrations of HS and dermatan sulfate (DS) at Weeks 24 and 48.

Liver and spleen volumes are measured using MRI at Week 48 to ensure they are within normal ranges.

6 completion of the study

The study is estimated to conclude by December 30, 2025.

Final assessments are conducted to determine the overall impact of the treatment on MPS II symptoms.

Who Can Join the Study?

Participants must be aged 2 to less than 6 years for Cohort A or 6 to less than 26 years for Cohort B.
Participants must have a confirmed diagnosis of Mucopolysaccharidosis Type II, also known as MPS II. For Cohort A, this is referred to as nMPS II, and for Cohort B, as nnMPS II.
For those in non-run-in Cohort A and Cohort B, participants must be on maintenance ERT (Enzyme Replacement Therapy) and have tolerated at least 4 months (or 16 weeks) of idursulfase therapy before the screening period.

Who Cannot Join the Study?

Patients who have a medical condition other than Mucopolysaccharidosis Type II (MPS II).
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial group.
Patients who are not male or female, as both genders are included in the study.
Patients who are not considered part of a vulnerable population, as the study includes vulnerable groups.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Lille	Lille	France

Other Sites

Site Name	City	Country
Azienda Sanitaria Universitaria Friuli Centrale	Udine	Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Kommunale Traegergesellschaft Cottbus mbH	Cottbus	Germany
SphinCS GmbH	Hochheim Am Main	Germany
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Vrije Universiteit Brussel	Jette	Belgium
Uxewdqfzmy Mzjlgmh Cdcasd Hublszdggmxtgivyn	Hamburg	Germany
Ecrlbmi Untupevugsuk Mdxjzsl Crkzhsx Rnqnmbldk (zbsevnf Mfw	Rotterdam	The Netherlands
Ugtmzpzqbf Ov Avegdyu	Edegem	Belgium
Hjuzuniv Vkab dptsfdfl	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	12.02.2022
Czechia	Recruiting	12.02.2022
France	Recruiting	12.02.2022
Germany	Recruiting	12.02.2022
Italy	Recruiting	12.02.2022
Spain	Recruiting	12.02.2022
Sweden	Recruiting	12.02.2022
The Netherlands	Recruiting	12.02.2022

Trial locations

Investigated drugs:

DNL310 is an investigational medication being studied for its potential effects on the central nervous system (CNS) in children with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. This medication is being evaluated to see if it can reduce the concentration of harmful substances in the cerebrospinal fluid (CSF) and improve adaptive behavior in affected children.

Idursulfase is an approved enzyme replacement therapy used to treat Mucopolysaccharidosis Type II (MPS II). It works by providing the enzyme that people with this condition lack, helping to break down certain substances in the body. In this study, it is being compared to DNL310 to assess its effectiveness and safety in treating the CNS symptoms of the disease.

Mucopolysaccharidosis Type II (MPS II) – This is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This enzyme deficiency leads to the accumulation of glycosaminoglycans (GAGs) in the body’s cells, which can cause a variety of symptoms. The disease primarily affects males and can lead to progressive damage to the heart, bones, joints, respiratory system, and central nervous system. Symptoms may include developmental delays, distinctive facial features, and joint stiffness. Over time, individuals with MPS II may experience worsening of physical and cognitive abilities. The severity and progression of symptoms can vary widely among those affected.

Trial ID:

2024-510990-21-00

Protocol code:

DNLI-E-0007

NCT ID:

NCT05371613

Trial Phase:

Therapeutic use (Phase IV)

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Study on the Effectiveness and Safety of DNL310 vs Idursulfase for Children and Young Adults with Mucopolysaccharidosis Type II

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?