First human trial targets blindness in rare genetic disorder
NCT ID NCT07269665
First seen Jan 07, 2026 · Last updated May 19, 2026 · Updated 24 times
Summary
This early-stage study tests a single dose of AXV-101 in 12 children (ages 4-17) with Bardet-Biedl syndrome type 1 who have vision loss. The main goal is to see if the treatment is safe and to find the right dose. Researchers will monitor eye health, blood and tear samples, and quality of life for up to 5 years.
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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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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
Conditions
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