Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
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)
Arbor Biotechnologies
23 participants
Jun 16, 2025
INTERVENTIONAL
Conditions
Summary
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
Inclusion Criteria7
- 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
- hour UOx ≥0.7 mmol/24 hours/1.73 m²
- eGFR ≥30 mL/min/1.73m²
- Weight ≤90 kg
Exclusion Criteria5
- 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)
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Interventions
Intravenous (IV) infusion
Locations(7)
View Full Details on ClinicalTrials.gov
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NCT06839235