New cystinosis drug shows promise in early trial
NCT ID NCT05994534
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times
Summary
This study tests a new oral solution called NPI-001 in people with cystinosis, a rare disease that causes cystine buildup in cells. Researchers want to see if NPI-001 is safe and works better than the current drug cysteamine. About 12 participants aged 10 and older will stop their usual medicine for 2 days to take part.
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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
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Locations
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Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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Other studies related to the condition(s) this trial covers.
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