ClinicalTrials.gov record NCT06839235

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants With Primary Hyperoxaluria Type 1 (PH1)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
Age6 years old to 64 years old
SexAll
LocationFrance, Germany, Tunisia, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1/2 trial studies a single IV dose of ABO-101 in people with genetically confirmed primary hyperoxaluria type 1 (PH1). Part A enrolls people (18–64 years) in a single-ascending-dose dose-finding step; Part B enrolls children (6–17 years) at the selected dose. Key eligibility gates include genetic confirmation of PH1, specified urinary oxalate and kidney function thresholds, and exclusions such as prior liver/kidney transplant, current dialysis, recent RNAi/siRNA oxalate-lowering therapy, and pregnancy/breastfeeding.

What it is studying. The study is measuring whether a single IV dose of ABO-101 is safe and tolerable, how the body processes the drug (pharmacokinetics), how the drug affects biological markers (pharmacodynamics), and early signs of effect on urinary oxalate and related measures.

What the researchers hope to learn. Collect safety and tolerability data, establish recommended dose, measure pharmacokinetics and pharmacodynamics, and assess changes in urinary oxalate, plasma glycolate, kidney function (eGFR), plasma/urine concentrations of study components, and anti-drug/anti-Cas antibodies.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • ABO-101

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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)

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive a single intravenous (IV) infusion of ABO‑101: adults in Part A get single ascending doses to identify a recommended dose, and children in Part B receive the recommended single dose. After dosing, participants enter a long‑term monitoring period (Study Period 2) per local and national requirements. Study visits include safety monitoring, blood draws and urine collections (including 24‑hour urine) for drug levels, urinary oxalate, kidney function, and antibody testing. Some outcomes are followed for up to 6 months, and kidney function is also assessed up to 12 and 24 months as noted in the registry.

How the study is structured. Open-label, non-randomized Phase 1/2 treatment study with two study periods. Part A uses a single ascending dose (adults) to identify a recommended dose. Part B gives a single dose to pediatric participants at that recommended dose. After dosing, participants enter a long-term monitoring program for safety and biomarker follow-up.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in France, Germany, Tunisia, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on primary hyperoxaluria

Clinical trials13 trials listed for primary hyperoxaluriaEvery trial the registry lists for primary hyperoxaluria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in primary hyperoxaluria researchLatest monthly update: September 2026. Includes: Early oxalate change. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 16, 2025
Primary completion
March 2029 (estimated)
Study completion
February 2043 (estimated)
Target enrollment
23 participants (estimated)
Last updated on registry
Feb 12, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Arbor Biotechnologies

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (7)

Terms used on this page · explanations by MedicalUpdates.ai

pharmacokinetics
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
pharmacodynamics
What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
ascending dose
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
Sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
eGFR
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Key Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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