RECRUITING Phase 1 / Phase 2 ClinicalTrials.gov

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

Sponsor: Arbor Biotechnologies • ClinicalTrials.gov ID: NCT06839235

Registry updated: 2026-10-19

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Description

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.

Interventions

ABO-101 (Drug)
Intravenous (IV) infusion

Eligibility criteria

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)

Conditions

Primary Hyperoxaluria Type 1 (PH1)

Locations

Mayo Clinic — Rochester, Minnesota, United States
Nucleus Network — Saint Paul, Minnesota, United States
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants — Lyon, France
Kindernierenzentrum Bonn — Bonn, Germany
Heidi Chaker — Sfax, Tunisia
Queen Elizabeth Hospital Birmingham — Birmingham, United Kingdom
Royal Free Hospital — London, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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