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Hunter syndrome study pulled before it even started

NCT ID NCT05494593

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 Results may not be published yet.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 This study
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study aimed to see if giving a combination of immune-suppressing drugs (rituximab, methotrexate, and IVIG) alongside the standard enzyme therapy ELAPRASE could prevent patients with Hunter syndrome from developing harmful antibodies. It was designed for boys who had never received ELAPRASE before. However, the study was withdrawn by the sponsor before enrolling any participants, so no data was collected.

What this could mean

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

Active substance
Idursulfase (ELAPRASE), rituximab, methotrexate, intravenous immunoglobulin
What this could lead to
If successful, this approach could help prevent the body from rejecting enzyme replacement therapy, making treatment more effective for Hunter syndrome patients.
What could go wrong
The study was withdrawn before enrolling any participants, so no results are available. The immune tolerizing regimen adds significant medication and potential side effects without proven benefit.

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.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Started

Feb 2023

Finished

Aug 2025

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

Up to 6 years

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: * Participant is male. * Participant is ELAPRASE-naïve at study entry. * Participant must have a documented diagnosis of MPS II. The following combination will be accepted as diagnostic of MPS II: * Participant has a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of less than or equal to (\<=) 10 percent (%) of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). The participant has a normal enzyme activity level of at least 1 other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). * Participant has a documented mutation in the IDS gene; additionally, participants must have a severe mutation (example, large deletion or complex gene rearrangement), which is predicted to lead to development of a persistent anti-idursulfase antibody response. * Participant will be less than (\<) 6 years of age at enrollment. * Participant has a negative test result for serum anti-idursulfase antibodies. Exclusion Criteria: * Participant has received treatment with any investigational drug within the 30 days prior to study entry. * Participant has received or is receiving treatment with idursulfase-IT. * Participant has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time. * Participant has received blood product transfusions within 90 days prior to screening. * Participant is unable to comply with the protocol as determined by the investigator. * Participant has known or suspected intolerance or hypersensitivity to the investigational product(s), closely related compounds, or any of the stated ingredients, including the prophylactic ITR. * Participant has current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments. * Participant has current or relevant history of physical or psychiatric illness, or any medical disorder that may require treatment or make the participant unlikely to fully complete the study, or any condition that presents undue risk from the investigational product or procedures. * Participant has current use of any medication (including over-the-counter, herbal, or homeopathic preparations) that could affect (improve or worsen) the condition being studied, or could affect the action, absorption, or disposition of the investigational product(s), or clinical or laboratory assessment (Current use is defined as use within 30 days). * Within 30 days prior to the first dose of investigational product, the participant has been enrolled in a clinical study (including vaccine studies) that, in the investigator's opinion, may impact this study.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Hospital and Research Center at Oakland

    Oakland, California, 94609, United States

  • Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • NewYork-Presbyterian Morgan Stanley Children's Hospital

    New York, New York, 10032, United States

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Rady Childrens Hospital San Diego - PIN

    San Diego, California, 92123, United States

  • The Cleveland Clinic Foundation

    Twinsburg, Ohio, 44087, United States

  • The Lundquist Institute for BioMedical Innovation at Harbor-UCLA Medical Center

    Torrance, California, 90502, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.