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Promising drug may protect kidneys in kids with rare oxalate disorder

NCT ID NCT05001269

Completed Disease control Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

primary hyperoxaluria primary hyperoxaluria type 1 primary hyperoxaluria type 2 primary hyperoxaluria type 3

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PRIMARY HYPEROXALURIA PRIMARY HYPEROXALURIA TYPE 1 PRIMARY HYPEROXALURIA TYPE 2 PRIMARY HYPEROXALURIA TYPE 3

Contacts and locations

Locations

  • Clinical Research Site

    Rochester, Minnesota, 55905, United States

  • Clinical Research Site

    Hamilton, Ontario, L8S 4K1, Canada

  • Clinical Research Site

    Heidelberg, 69120, Germany

  • Clinical Research Site

    Fukuoka, 830-0011, Japan

  • Clinical Research Site

    Nagoya, 467-8602, Japan

  • Clinical Research Site

    Beirut, 1100, Lebanon

  • Clinical Research Site

    Bialystok, 15-274, Poland

  • Clinical Research Site

    Barcelona, 08035, Spain

  • Clinical Research Site

    Yenimahalle, Ankara, 06506, Turkey (Türkiye)

  • Clinical Trial Site

    Bonn, 53127, Germany

  • Clinical Trial Site

    Roma, 00165, Italy

  • Clinical Trial Site

    Dubai, +971, United Arab Emirates

  • Clinical Trial Site

    London, WC1N 3JH, United Kingdom

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