First human trial launches for Gene-Based MPS i treatment
NCT ID NCT05682144
First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 1 time
Summary
This early-stage study tests whether ISP-001 is safe and tolerable in 11 people with a rare genetic disease called MPS I (Hurler-Scheie or Scheie types). Participants receive the experimental treatment and are monitored for side effects. The goal is to gather safety data for future research.
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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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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States