New cystinosis drug shows promise in early trial
NCT ID NCT05994534
First seen Jan 06, 2026 · Last updated May 22, 2026 · Updated 22 times
Summary
This study tests a new oral medicine called NPI-001 for people with cystinosis, a rare disease that causes cystine buildup in cells. It compares NPI-001 to the standard drug cysteamine in 12 patients aged 10 and older. The goal is to see if NPI-001 is safe and how well it controls cystine levels over 6 hours.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital at Westmead
RECRUITINGWestmead, New South Wales, 2145, Australia
Contact Email: •••••@•••••
Conditions
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