Gene therapy breakthrough for rare kidney disease enters human trials

NCT ID NCT06839235

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

Summary

This study tests a new gene therapy called ABO-101 for people with Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder that causes kidney stones and kidney damage. The trial will first test single doses in adults to find the safest dose, then test that dose in children. Researchers will monitor side effects and measure changes in urine oxalate levels to see if the treatment works.

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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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Heidi Chaker

    RECRUITING

    Sfax, Tunisia

  • Hospices Civils de Lyon- Hôpital Femmes Mères Enfants

    NOT_YET_RECRUITING

    Lyon, France

  • Kindernierenzentrum Bonn

    NOT_YET_RECRUITING

    Bonn, Germany

    Contact Email: •••••@•••••

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Nucleus Network

    WITHDRAWN

    Saint Paul, Minnesota, 55114, United States

  • Queen Elizabeth Hospital Birmingham

    RECRUITING

    Birmingham, United Kingdom

    Contact Email: •••••@•••••

  • Royal Free Hospital

    RECRUITING

    London, United Kingdom