First-in-human trial aims to tame rare enzyme disorder
NCT ID NCT05682144
First seen Apr 03, 2026 · Last updated May 02, 2026 · Updated 4 times
Summary
This early-stage study tests a new therapy called ISP-001 in 11 people with MPS I (Hurler-Scheie or Scheie types). The main goal is to see if the treatment is safe and tolerable. Participants must be at least 10 years old and able to stay near the study site for a few days after the infusion.
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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.
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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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact Email: •••••@•••••
Contact
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
Contact
Conditions
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