HUNTER SYNDROME
Clinical trials for HUNTER SYNDROME explained in plain language.
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Extended safety check for rare genetic disease therapy
Disease control OngoingThis study continues monitoring the safety of two drugs (idursulfase-IT and elaprase) for children and adults with Hunter syndrome who have cognitive impairment. It follows participants from two previous trials for nearly five years to track any side effects from the monthly spin…
Matched conditions: HUNTER SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Withdrawn trial aimed to stop bodies from fighting rare disease drug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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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