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

Verified siteInvestigationalNo placebo
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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 the long-term safety of a treatment for Hunter Syndrome, a rare genetic disorder that affects the body's ability to break down certain sugars. The study involves patients who also have cognitive impairment, which means they experience difficulties with thinking and understanding. The treatment being tested is called Idursulfase, specifically in a form known as idursulfase-IT (HGT-2310), which is administered directly into the spinal fluid through a method called intrathecal injection. This is done using a special device called the SOPH-A-PORT Mini S Implantable access port. In addition to this, patients will continue to receive Elaprase, an enzyme replacement therapy given through an intravenous (IV) infusion.

The purpose of the study is to gather information on the long-term safety of this combined treatment approach. Participants in this study have previously been involved in earlier studies and have shown clinical benefits from the treatment. The study will monitor the safety of the treatment by observing any side effects or adverse events that may occur, including those related to the medication, the device used for administration, and the procedure itself.

Throughout the study, participants will receive regular doses of the treatment and will be closely monitored by healthcare professionals. The study aims to ensure that the treatment is safe for long-term use in individuals with Hunter Syndrome and cognitive impairment. This research is important for understanding how to best manage and treat this condition over an extended period.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing previous studies HGT-HIT-046 or SHP609-302. The participant must have received a clinical benefit from idursulfase-IT as determined by the investigator.

    Informed consent must be signed by the participant or their legally designated representative.

  2. Step 2

    Treatment administration

    The treatment involves the administration of idursulfase-IT through an intrathecal route, which means it is injected into the space around the spinal cord.

    In addition, Elaprase® is administered intravenously as part of enzyme replacement therapy.

  3. Step 3

    Monitoring and safety assessment

    The primary focus is on the long-term safety of the treatment. Safety is assessed by monitoring any adverse events, which are categorized by type, severity, and their relationship to the treatment.

    The safety of the intrathecal administration process and the intravenous infusion of Elaprase® is also evaluated.

  4. Step 4

    Duration of the study

    The study is expected to continue until January 1, 2034.

    Recruitment for the study is estimated to start on January 1, 2024.

Who can join the trial?

4 criteria

  • The patient must have completed the end of study assessments in previous studies called HGT-HIT-046 or SHP609-302 and must have received a clinical benefit from a treatment called **idursulfase-IT**, according to the opinion of the study doctor.
  • The patient, or the patient's legally designated representative, must have been informed about the nature of this study and must have voluntarily signed a consent form approved by an ethics committee. This means they agree to participate after understanding all important details of the study.
  • The patient must have continued to receive a treatment called **Elaprase** regularly in the previous studies HGT-HIT-046 or SHP609-302.
  • Only male patients are eligible to participate in this study.

Who cannot join the trial?

4 criteria

  • Only males can participate, so females are excluded.
  • Participants must have Hunter Syndrome with cognitive impairment, so those without this condition are excluded.
  • Participants must be receiving specific treatments: intrathecal idursulfase-IT and IV Elaprase® enzyme replacement therapy. Those not receiving these treatments are excluded.
  • Participants must be within a certain age range, so those outside this range are excluded.
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Investigated drugs

  • Idursulfase-IT

    is a medication administered directly into the spinal fluid. It is used in this trial to help manage symptoms in patients with Hunter syndrome, particularly those with cognitive impairment. The goal is to evaluate its long-term safety when given in this manner.

  • Elaprase

    is an enzyme replacement therapy given through an intravenous infusion. It is used to treat patients with Hunter syndrome by providing the enzyme that is missing or not working properly in their bodies. This trial aims to assess the long-term safety of using Elaprase in combination with Idursulfase-IT.

What is already known about the treatment

  • Idursulfase-IT

    This medication is administered intrathecally, meaning it is delivered directly into the spinal canal. It is currently being studied in clinical trials for its long-term safety in treating Hunter Syndrome with cognitive impairment. The main therapeutic indication is to address the symptoms of Hunter Syndrome, a rare genetic disorder. At the molecular level, Idursulfase-IT works by replacing the deficient enzyme iduronate-2-sulfatase, which helps break down certain complex molecules in the body. It is classified as an enzyme replacement therapy.

  • Elaprase®

    This medication is administered intravenously, meaning it is delivered directly into the bloodstream. It is approved for use in treating Hunter Syndrome, a rare genetic disorder. The main therapeutic indication is to manage the symptoms associated with this condition. Elaprase® works by providing the enzyme iduronate-2-sulfatase, which is lacking in individuals with Hunter Syndrome, helping to break down complex molecules that accumulate in the body. It is classified as an enzyme replacement therapy.

Investigated diseases

Hunter Syndrome – Hunter Syndrome, also known as Mucopolysaccharidosis 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 sugars in the body, affecting various organs and tissues. Symptoms often include joint stiffness, distinctive facial features, and hearing loss. Over time, individuals may experience progressive physical and neurological decline. The severity and progression of symptoms can vary widely among affected individuals. Cognitive impairment is a common feature, particularly in more severe cases.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2023-504127-90-00Protocol codeTAK-609-3001Estimated enrolment8 patientsSponsorTakeda Development Center Americas Inc.

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