Overview
The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, pediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring program to comply with local and national requirements.
Eligibility
Key Inclusion Criteria for Parts A and B
- Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase (AGXT) gene (valid historical laboratory data will be reviewed and approved by the Sponsor)
- Age at time of signing the informed consent/assent form:
- Part A: ≥18 years to ≤64 years
- Part B: ≥6 years to <18 years
- 24-hour UOx ≥0.7 mmol/24 hours/1.73 m²
- eGFR ≥30 mL/min/1.73m²
- Weight ≤90 kg
Key Exclusion Criteria for Parts A and B
- Confirmed diagnosis of primary hyperoxaluria type 2 or type 3
- History of a liver, kidney or combined liver/kidney transplant
- Currently on dialysis
- Participant has previously used (within past 24 months) or is currently receiving an approved or investigational urinary oxalate lowering RNA interference (RNAi) or siRNA therapy
- Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)