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Lifeline for hunter syndrome patients: continued access to Brain-Targeting therapy

NCT ID NCT05795361

Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program offers continued treatment with idursulfase-IT (TAK-609), given directly into the spinal fluid, plus standard intravenous Elaprase, for children and adults with Hunter syndrome who have cognitive impairment. It is only open to people who completed earlier studies of this combination. The goal is to maintain any benefits they experienced and keep the disease under control.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
idursulfase-IT (TAK-609) given into the spinal fluid, plus intravenous Elaprase
What this could lead to
If successful, this program could provide ongoing disease control and cognitive benefit for people with Hunter syndrome who have cognitive impairment.
What could go wrong
This is an open-access program, not a new trial, so it does not test whether the treatment works. Risks include side effects from the spinal injections or the implanted delivery device.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Participants will have completed the treatment period of the HGT-HIT-046 (NCT01506141) or SHP609-302 (NCT02412787) study prior to the first dose on this program. 2. Participant and/or a parent(s)/legal guardian is informed of the nature of this compassionate post-trial access program and can provide written informed consent for themselves or the child to participate (with assent from the child when appropriate prior to treatment). Exclusion Criteria: 1. Participant has a condition that in the opinion of the treating physician may compromise their safety. 2. Participant has a known hypersensitivity to idursulfase-IT or its components.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hunter syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    22 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    AVAILABLE

    Chicago, Illinois, 60611, United States

  • Board of Regents of the University of Nebraska

    AVAILABLE

    Omaha, Nebraska, 68198-5450, United States

  • Division of Medical Genetics, University of Utah

    AVAILABLE

    Salt Lake City, Utah, 84112, United States

  • H.C.U. de Valladolid

    AVAILABLE

    Valladolid, 47003, Mexico

  • Hospital Universitario Reina Sofia

    AVAILABLE

    Córdoba, 14004, Spain

  • Instituto Nacional de Pediatria

    AVAILABLE

    Coyoacán, Mexico City, 4530, Mexico

  • Jackson Memorial Hospital University of Miami

    AVAILABLE

    Miami, Florida, 33101, United States

  • Joseph M. Sanzari Children's Hospital

    AVAILABLE

    Hackensack, New Jersey, 07601, United States

  • NYU Langone Medical Center

    AVAILABLE

    New York, New York, 10017, United States

  • Phoenix Childrens Hospital

    AVAILABLE

    Phoenix, Arizona, 85015, United States

  • Queensland Childrens Hospital

    AVAILABLE

    South Brisbane, Queensland, QLD 4101, Australia

  • Randall Children's Hospital at Legacy Emanuel

    AVAILABLE

    Portland, Oregon, 97227, United States

  • Royal Manchester Children's Hospital - PPDS

    AVAILABLE

    Manchester, M13 9WL, United Kingdom

  • Seattle Children's Hospital - PIN

    AVAILABLE

    Seattle, Washington, 98105, United States

  • The Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • The Cleveland Clinic Foundation

    AVAILABLE

    Cleveland, Ohio, 44195, United States

  • The Nemours Foundation

    AVAILABLE

    Wilmington, Delaware, 19803, United States

  • The Regents of the University of California

    AVAILABLE

    Oakland, California, 94609, United States

  • The University of North Carolina

    AVAILABLE

    Chapel Hill, North Carolina, 27514, United States

  • UPMC Childrens Hospital of Pittsburgh

    AVAILABLE

    Pittsburgh, Pennsylvania, 15224, United States

  • Vanderbilt Children's Hospital

    AVAILABLE

    Nashville, Tennessee, 37232-7610, United States

  • Washington University

    AVAILABLE

    Washington, Missouri, 63130, United States

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