HUNTER SYNDROME
Clinical trials for HUNTER SYNDROME explained in plain language.
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Withdrawn trial aimed to stop bodies from fighting rare disease drug
Disease control TerminatedThis study aimed to see if a combination of immune-suppressing drugs could help young boys with Hunter syndrome better tolerate their main enzyme replacement therapy (ELAPRASE). The goal was to prevent their immune systems from developing antibodies that could block the treatment…
Matched conditions: HUNTER SYNDROME
Phase: PHASE4 • Sponsor: Takeda • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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Extended safety check for rare genetic disease therapy
Disease control OngoingThis study continues monitoring the safety of two treatments (idursulfase-IT and Elaprase) for Hunter syndrome, a rare genetic disorder that causes cognitive impairment. It follows 6-8 children and adults from previous studies for nearly 5 years to track any side effects. The goa…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated Mar 17, 2026 13:09 UTC
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New hope for kids with devastating genetic diseases
Disease control OngoingThis study is testing a stem cell transplant procedure for children and adults with severe, inherited metabolic disorders and a rare bone disease. The goal is to see if using a specific drug combination can help the donor cells successfully take hold in the patient's body while k…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Mar 13, 2026 15:05 UTC