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New hope for rare kidney disease: drug targets oxalate buildup in severe cases

NCT ID NCT04580420

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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Conditions

The condition(s) this trial relates to.

chronic kidney disease end stage renal failure primary hyperoxaluria type 1

As listed by the trial registrant

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

END-STAGE RENAL DISEASE PRIMARY HYPEROXALURIA TYPE 1

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Clinical Trial Site

    ACTIVE_NOT_RECRUITING

    San Francisco, California, 94143, United States

  • Clinical Trial Site

    ACTIVE_NOT_RECRUITING

    Boston, Massachusetts, 02115, United States

  • Clinical Trial Site

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Clinical Trial Site

    ACTIVE_NOT_RECRUITING

    New York, New York, 10016, United States

  • Clinical Trial Site

    WITHDRAWN

    Bron, 69677, France

  • Clinical Trial Site

    WITHDRAWN

    Paris, 75019, France

  • Clinical Trial Site

    RECRUITING

    Bonn, 53127, Germany

  • Clinical Trial Site

    WITHDRAWN

    Heidelberg, 69120, Germany

  • Clinical Trial Site

    WITHDRAWN

    Roma, 00165, Italy

  • Clinical Trial Site

    RECRUITING

    Beirut, 00001, Lebanon

  • Clinical Trial Site

    WITHDRAWN

    Casablanca, 2025, Morocco

  • Clinical Trial Site

    WITHDRAWN

    Oradea, 410469, Romania

  • Clinical Trial Site

    WITHDRAWN

    Oradea, 410562, Romania

  • Clinical Trial Site

    RECRUITING

    Barcelona, 08035, Spain

  • Clinical Trial Site

    WITHDRAWN

    Santa Cruz de Tenerife, 38320, Spain

  • Clinical Trial Site

    RECRUITING

    Dubai, +971, United Arab Emirates

  • Clinical Trial Site

    WITHDRAWN

    London, NWG 2Q3, United Kingdom

  • Clinical Trial Site

    RECRUITING

    London, WC1N3JH, United Kingdom

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