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NCT06839235PHASE1, PHASE2Recruiting

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

Arbor Biotechnologies

Start Date

6/16/2025

Completion Date

2/1/2043

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 Criteria

Age Range: 6 years to 64 years

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)

Interventions

DRUG

ABO-101

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Conditions

Primary Hyperoxaluria Type 1 (PH1)

Locations

Mayo Clinic

Rochester, Minnesota 55905

United States

Nucleus Network

Saint Paul, Minnesota 55114

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