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
Sex
ALL
Min age
6 Years
Max age
64 Years
Healthy volunteers
No
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)
Primary outcome measure(s)
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs) — Up to 6 months
Trial sites (7)
Facility
City
Region
Status
Mayo Clinic
Rochester
Minnesota
Recruiting
Nucleus Network
Saint Paul
Minnesota
Withdrawn
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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