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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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Heidi Chaker

    RECRUITING

    Sfax, Tunisia

    Contact Phone: •••-•••-••••

  • Hospices Civils de Lyon- Hôpital Femmes Mères Enfants

    NOT_YET_RECRUITING

    Lyon, France

    Contact Phone: •••-•••-••••

  • Kindernierenzentrum Bonn

    NOT_YET_RECRUITING

    Bonn, Germany

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact Phone: •••-•••-••••

  • Nucleus Network

    WITHDRAWN

    Saint Paul, Minnesota, 55114, United States

  • Queen Elizabeth Hospital Birmingham

    RECRUITING

    Birmingham, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Royal Free Hospital

    RECRUITING

    London, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••