Hunter syndrome study pulled before it even started

NCT ID NCT05494593

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 However, the study was withdrawn by the sponsor before enrolling any participants, so no data was collected.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The study was withdrawn before enrolling any participants, so no results are available.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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis mucopolysaccharidosis type 2

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