New hope for rare kidney disease: drug targets oxalate buildup in severe cases
NCT ID NCT04580420
First seen Jan 03, 2026 · Last updated Jun 23, 2026 · Updated 28 times
Summary
This phase 2 study tests a drug called nedosiran (DCR-PHXC) in 28 people with primary hyperoxaluria type 1 (PH1) who have severe kidney impairment, with or without dialysis. The goal is to see if the drug safely lowers plasma oxalate levels, which can cause kidney stones and damage. The trial is currently only enrolling children under 6 years old.
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This is a summary of
the original study
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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
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Clinical Trial Site
ACTIVE_NOT_RECRUITINGSan Francisco, California, 94143, United States
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Clinical Trial Site
ACTIVE_NOT_RECRUITINGBoston, Massachusetts, 02115, United States
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Clinical Trial Site
RECRUITINGRochester, Minnesota, 55905, United States
Contact
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Clinical Trial Site
ACTIVE_NOT_RECRUITINGNew York, New York, 10016, United States
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Clinical Trial Site
WITHDRAWNBron, 69677, France
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Clinical Trial Site
WITHDRAWNParis, 75019, France
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Clinical Trial Site
RECRUITINGBonn, 53127, Germany
Contact
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Clinical Trial Site
WITHDRAWNHeidelberg, 69120, Germany
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Clinical Trial Site
WITHDRAWNRoma, 00165, Italy
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Clinical Trial Site
RECRUITINGBeirut, 00001, Lebanon
Contact
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Clinical Trial Site
WITHDRAWNCasablanca, 2025, Morocco
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Clinical Trial Site
WITHDRAWNOradea, 410469, Romania
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Clinical Trial Site
WITHDRAWNOradea, 410562, Romania
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Clinical Trial Site
RECRUITINGBarcelona, 08035, Spain
Contact
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Clinical Trial Site
WITHDRAWNSanta Cruz de Tenerife, 38320, Spain
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Clinical Trial Site
RECRUITINGDubai, +971, United Arab Emirates
Contact
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Clinical Trial Site
WITHDRAWNLondon, NWG 2Q3, United Kingdom
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Clinical Trial Site
RECRUITINGLondon, WC1N3JH, United Kingdom
Contact
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nedosiran (DCR-PHXC)
What this could lead to
If it works, this could point toward a treatment to lower harmful oxalate levels in patients with PH1 and severe kidney problems, potentially reducing complications.
What could go wrong
This is a small, early-phase trial with only 28 participants, so results may not apply to everyone. The drug may not work well in patients on dialysis, and safety risks are still being studied.
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.