Phase 12 Study Evaluating Safety and Effects of ABO-101 for Primary Hyperoxaluria Type 1 in Children and Adults

What this Trial Is About

Researchers are evaluating ABO-101 to study its safety, tolerability, how the body processes it, and its effects in people with primary hyperoxaluria type 1 PH1, a rare genetic condition. This Phase 12 clinical trial aims to find a recommended dose for adults and then assess this dose in children. The study is sponsored by Arbor Biotechnologies and includes a long-term follow-up to meet regulatory requirements. The trial has two main study periods. In the first period, Part A involves adults receiving single increasing doses of ABO-101 through an intravenous infusion to identify a suitable dose. Part B then treats pediatric participants with the chosen dose. After these parts, the second study period begins, focusing on long-term monitoring for safety and treatment effects. Participants will have regular assessments including monitoring for any treatment-related side effects over six months, measuring changes in urine oxalate and plasma glycolate levels, and tracking kidney function for up to two years. Blood and urine samples will be collected to study drug components and immune responses. The trial involves careful safety follow-up and ongoing evaluation throughout the study duration.

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