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
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Heidi Chaker
RECRUITINGSfax, Tunisia
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Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
NOT_YET_RECRUITINGLyon, France
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Kindernierenzentrum Bonn
NOT_YET_RECRUITINGBonn, Germany
Contact Email: •••••@•••••
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
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Nucleus Network
WITHDRAWNSaint Paul, Minnesota, 55114, United States
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Queen Elizabeth Hospital Birmingham
RECRUITINGBirmingham, United Kingdom
Contact Email: •••••@•••••
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Royal Free Hospital
RECRUITINGLondon, United Kingdom