New hope for rare disease: testing alternative to harsh current treatment
NCT ID NCT05994534
Summary
This early-stage study is testing a new oral medication called NPI-001 for people with cystinosis, a rare genetic disorder. It will compare NPI-001's safety and its ability to lower harmful cystine levels in the body to the current standard treatment, cysteamine. The study involves about 12 participants aged 10 and older who can temporarily stop their current cysteamine therapy.
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Contacts and locations
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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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