Actively Recruiting
Phase 1/2 Dose Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Effects of ABO-101 in Primary Hyperoxaluria Type 1
Led by Arbor Biotechnologies · Updated on 2026-02-12
23
Participants Needed
7
Research Sites
726 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ABO-101 to study its safety, tolerability, how the body processes it, and its effects in people with primary hyperoxaluria type 1 (PH1), a rare genetic condition. This Phase 1/2 clinical trial aims to find a recommended dose for adults and then assess this dose in children. The study is sponsored by Arbor Biotechnologies and includes a long-term follow-up to meet regulatory requirements. The trial has two main study periods. In the first period, Part A involves adults receiving single increasing doses of ABO-101 through an intravenous infusion to identify a suitable dose. Part B then treats pediatric participants with the chosen dose. After these parts, the second study period begins, focusing on long-term monitoring for safety and treatment effects. Participants will have regular assessments including monitoring for any treatment-related side effects over six months, measuring changes in urine oxalate and plasma glycolate levels, and tracking kidney function for up to two years. Blood and urine samples will be collected to study drug components and immune responses. The trial involves careful safety follow-up and ongoing evaluation throughout the study duration.
CONDITIONS
Brief Title
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetic confirmation of primary hyperoxaluria type 1 (PH1) with pathogenic AGXT mutations
- Age 18 to 64 years for adults (Part A)
- Age 6 to under 18 years for pediatric participants (Part B)
- 24-hour urinary oxalate excretion of at least 0.7 mmol/24 hours/1.73 m²
- Estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m²
- Weight of at least 90 kg
You will not qualify if you...
- Diagnosis of primary hyperoxaluria type 2 or type 3
- History of liver, kidney, or combined liver/kidney transplant
- Currently receiving dialysis
- Use of urinary oxalate lowering RNA interference (RNAi) or siRNA therapy within the past 24 months or currently
- Pregnant or breastfeeding females, or planning pregnancy during the first 12 months of the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 6 months
Participants receive intravenous ABO-101 as part of the study intervention.
1 or more treatment visits depending on dosing schedule and cohort
Duration - Up to 18 months after treatment
Participants are monitored for safety and effects of the treatment after dosing ends.
Periodic visits for safety and outcome assessments
Trial Site Locations
Total: 7 locations
1
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
2
Nucleus Network
Saint Paul, Minnesota, United States, 55114
Withdrawn
3
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
Lyon, France
Not Yet Recruiting
4
Kindernierenzentrum Bonn
Bonn, Germany
Not Yet Recruiting
5
Heidi Chaker
Sfax, Tunisia
Actively Recruiting
6
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Actively Recruiting
7
Royal Free Hospital
London, United Kingdom
Actively Recruiting
Research Team
D
Daniel Ory, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Frequently Asked Questions
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