Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1

• Research type

  Research Study

• Full title

  A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO 101 in Participants with Primary Hyperoxaluria Type 1 (PH1)

• IRAS ID

  1011065

• Contact name

  Tara Naylor

• Contact email

  arbortrials@arbor.bio

• Sponsor organisation

  Arbor Biotechnologies, Inc.

• Eudract number

  2024-518144-20

• Research summary

  The ABO-101 study is being conducted in participants with primary hyperoxaluria type 1 (PH1), a rare genetic disorder. ABO-101 is a highly specific genome-editing therapeutic designed to target the HAO1 gene, which encodes the enzyme glycolate oxidase. By reducing glycolate oxidase activity, ABO-101 is designed to potentially decrease the overproduction of oxalate that leads to progressive kidney damage in PH1 patients. Participants aged 6 to 64 years with a confirmed diagnosis of PH1 will be enrolled after meeting defined eligibility criteria. Participants will be monitored for approximately 2 years to assess the treatment’s safety and efficacy and will participate in a long-term follow-up for up to 15 years. The goal of this study is to determine whether ABO-101 is safe and whether it lowers urinary oxalate excretion.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  25/LO/0015

• Date of REC Opinion

  13 Mar 2025

• REC opinion

  Further Information Favourable Opinion