NCT06839235 · Arbor Biotechnologies
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
What this study is about
The goal of the redePHine study is to evaluate the safety, tolerability, how the drug moves through the body, and how the drug affects the body 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.
View original scientific 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.
Interventions
DRUG
ABO-101
Intravenous (IV) infusion
Primary outcome measures
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)
Time frame: Up to 6 months
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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)
Where
- Rochester, Minnesota
- Saint Paul, Minnesota
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 12, 2026 · Source of record for eligibility and locations