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 However, the study was withdrawn by the sponsor before enrolling any participants, so no data was collected.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- The study was withdrawn before enrolling any participants, so no results are available.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.
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
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Started
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Feb 2023
- Finished
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Aug 2025
- Lead sponsor
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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
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Up to 6 years
- Sex
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Male participants only
- Healthy volunteers
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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 Hide 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
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Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611, United States
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Children's Hospital and Research Center at Oakland
Oakland, California, 94609, United States
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Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
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NewYork-Presbyterian Morgan Stanley Children's Hospital
New York, New York, 10032, United States
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Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
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Rady Childrens Hospital San Diego - PIN
San Diego, California, 92123, United States
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The Cleveland Clinic Foundation
Twinsburg, Ohio, 44087, United States
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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.
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- Early enzyme therapy may help kids with rare disease grow better
- Home infusions may help patients stick to treatment
- MPS patients help design exercise program to boost mental health
- Hunterase extended trial shows promise for managing hunter syndrome
- New stem cell approach aims to tame rare genetic diseases