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Idursulfase

Clinical trials of Idursulfase are studying long-term safety and treatment effects in people with Hunter syndrome, also called mucopolysaccharidosis type II (MPS II). These studies include patients with cognitive impairment, as well as pediatric and young adult participants. The trials look at safety, brain and body outcomes, and other measures of disease control.

Table of contents

Trial overview

The trial set includes three interventional studies of Idursulfase in people with Hunter syndrome, also called mucopolysaccharidosis type II (MPS II).[1][2][3]

All three studies are listed as Authorised, and they range from Phase 3 to Phase 4.[1][2][3]

Who can participate

One study enrolls subjects with Hunter syndrome and cognitive impairment, which means the condition affects thinking or learning.[1]

Another study includes MPS II patients and is designed for pediatric and young adult participants.[3]

The open-label extension study has a very small planned group, with 8 participants, while the other two studies plan to enroll 80 and 64 participants.[1][2][3]

What the trials measure

The main safety endpoint in the extension study is the safety of intrathecal Idursulfase-IT, which is checked by tracking adverse events and whether they are linked to the treatment, the device used, the surgical procedure, or the IV Elaprase infusion.[1]

In one Phase 3 study, the main outcomes include change in BSID-III cognitive domain scores and change in CSF HS levels. BSID-III is a developmental test, and CSF HS means heparan sulfate measured in cerebrospinal fluid.[2]

In the Phase 4 study, the main outcomes are the ratio to baseline in CSF HS concentration and change from baseline in Vineland-3. Vineland-3 is a measure of adaptive behavior, such as daily living and communication skills.[3]

Trial designs and phases

The extension study is a Phase 3 open-label extension, which means everyone in the study knows which treatment is being given, and the study continues earlier research to collect long-term safety data.[1]

The JR-141 study is a Phase 3 interventional trial that aims to show efficacy, safety, and pharmacokinetics. Pharmacokinetics means how the body handles the study drug over time.[2]

The DNL310 study is a Phase 4 interventional trial comparing tividenofusp alfa (DNL310) with Idursulfase in pediatric and young adult participants with MPS II.[3]

Key trials in this set

2023-504127-90-00 is an open-label extension study for people already in studies HGT-HIT-046 and SHP609-302.[1] It looks at long-term safety of intrathecal Idursulfase-IT given together with intravenous Elaprase in subjects with Hunter syndrome and cognitive impairment.[1]

NCT04573023 studies JR-141 in people with MPS II / Hunter syndrome.[2] Its goals are to test whether the treatment helps central nervous system symptoms and body symptoms, and to assess safety and pharmacokinetics.[2]

NCT05371613 compares tividenofusp alfa (DNL310) with Idursulfase in pediatric and young adult participants with neuronopathic or non-neuronopathic MPS II.[3] The study focuses on brain-related activity and adaptive behavior, using CSF HS and Vineland-3 as key measures.[3]

What these studies may mean for patients

These trials show that researchers are studying Idursulfase in different ways, including long-term follow-up and comparison with other treatments.[1][3]

Some studies focus on the central nervous system, which is the brain and spinal cord, because MPS II can affect thinking and daily function.[2][3]

Overall, the trial data suggest a research focus on both safety and treatment effect in people living with Hunter syndrome across different age groups.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
2023-504127-90-00 Phase 3 Hunter Syndrome and Cognitive Impairment Authorised 8
NCT04573023 Phase 3 Mucopolysaccharidosis type II / Hunter Syndrome Authorised 80
NCT05371613 Phase 4 Mucopolysaccharidosis Type II [MPS II] Authorised 64

FAQ

  • What are the Idursulfase clinical trials studying? The trials are studying Idursulfase in people with Hunter syndrome (MPS II). They focus on safety, and some studies also look at brain-related and body-related outcomes.
  • Who can take part in these trials? The trials include subjects with Hunter syndrome and cognitive impairment, as well as pediatric and young adult participants with MPS II. Each study has its own enrollment rules.
  • What phases are the Idursulfase trials in? The trials listed are Phase 3 and Phase 4 studies. These later-phase trials often look at how well a treatment works and how safe it is in larger groups.
  • What kinds of results are being measured? The studies measure safety, changes in cognitive test scores, levels of heparan sulfate in cerebrospinal fluid, and adaptive behavior. Safety is also checked by tracking adverse events.
  • Are all the studies using Idursulfase in the same way? No. One study looks at intrathecal Idursulfase-IT, which means it is given into the spinal fluid space. Another study compares Idursulfase with other treatments in MPS II.

Ongoing Clinical Trials on Idursulfase

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

    Recruiting

    1 1 1 1
    Investigated drugs:
    Belgium Czechia France Germany Italy The Netherlands +2

  • Study on Long-Term Safety of Idursulfase-IT and Elaprase for Patients with Hunter Syndrome and Cognitive Impairment

    Recruiting

    1 1 1
    Investigated drugs:
    France

  • Study on the Effects and Safety of JR-141 and Idursulfase for Patients with Hunter Syndrome

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Poland Spain

Glossary

  • Hunter syndrome: A genetic condition also called mucopolysaccharidosis type II (MPS II). It can affect many parts of the body and may also affect thinking and learning.
  • Mucopolysaccharidosis type II (MPS II): The medical name for Hunter syndrome. It is the condition studied in these trials.
  • Cognitive impairment: Problems with thinking, learning, memory, or understanding. Some trials focus on people with this feature.
  • Intrathecal: Given into the space around the spinal cord, where spinal fluid is found.
  • Intravenous: Given through a vein, usually using a needle or infusion line.
  • Phase 3: A later stage of clinical research that usually studies how well a treatment works and how safe it is in a larger group.
  • Phase 4: A study done after a treatment is already being used. It often looks at real-world safety or added benefits.
  • Cerebrospinal fluid (CSF): The clear fluid around the brain and spinal cord. Some studies measure substances in this fluid to see if treatment affects the central nervous system.
  • Heparan sulfate (HS): A substance measured in the studies. Changes in its level may help show how the disease is affecting the body or brain.
  • Adaptive behavior: Daily life skills such as communication, social skills, and practical tasks.
  • Adverse events (AEs): Medical problems or unwanted events that happen during a study. They are tracked to assess safety.

References