Promising drug may protect kidneys in kids with rare oxalate disorder
NCT ID NCT05001269
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a monthly injection called nedosiran in 27 children from birth to 11 years old with primary hyperoxaluria, a rare condition that causes harmful oxalate buildup and kidney damage. The goal was to see if the drug safely lowers oxalate levels in the urine. Early results suggest it may help control the disease, but more research is needed to confirm long-term benefits.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nedosiran (a monthly injection that lowers oxalate production)
What this could lead to
If successful, this could offer a new treatment option for young children with primary hyperoxaluria, potentially reducing kidney damage and delaying or avoiding the need for dialysis or transplant.
What could go wrong
This is a small, early-phase study with only 27 children, so results may not apply to all patients. The drug may cause side effects or fail to lower oxalate enough to prevent long-term kidney problems.
Disclaimer
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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.
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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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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Clinical Research Site
Rochester, Minnesota, 55905, United States
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Clinical Research Site
Hamilton, Ontario, L8S 4K1, Canada
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Clinical Research Site
Heidelberg, 69120, Germany
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Clinical Research Site
Fukuoka, 830-0011, Japan
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Clinical Research Site
Nagoya, 467-8602, Japan
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Clinical Research Site
Beirut, 1100, Lebanon
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Clinical Research Site
Bialystok, 15-274, Poland
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Clinical Research Site
Barcelona, 08035, Spain
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Clinical Research Site
Yenimahalle, Ankara, 06506, Turkey (Türkiye)
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Clinical Trial Site
Bonn, 53127, Germany
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Clinical Trial Site
Roma, 00165, Italy
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Clinical Trial Site
Dubai, +971, United Arab Emirates
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Clinical Trial Site
London, WC1N 3JH, United Kingdom