New cystinosis drug shows promise in early trial
NCT ID NCT05994534
First seen Jan 06, 2026 · Last updated Apr 25, 2026 · Updated 18 times
Summary
This study tests a new oral medicine called NPI-001 for people with cystinosis, a rare disease that causes cystine crystals to build up in the body. It compares NPI-001 to the current standard treatment, cysteamine, to see if it is safe and how well it controls cystine levels. The trial involves 12 participants aged 10 and older who will stop their usual medication for two days during testing.
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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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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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