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

Arbor Biotechnologies

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Primary Hyperoxaluria Type 1 (PH1)

Treatments

Drug: ABO-101

Study type

Interventional

Funder types

Industry

Identifiers

NCT06839235

ABO-101-101

Details and patient eligibility

About

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.

Enrollment

23 estimated patients

Sex

All

Ages

6 to 64 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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)