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Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare genetic condition called Primary Hyperoxaluria Type 1 (PH1). PH1 is a disorder that leads to the overproduction of a substance called oxalate, which can cause kidney stones and other kidney-related issues. The trial will investigate a new treatment known as ABO-101, which is a solution for infusion. This treatment uses advanced gene editing technology to target and disrupt a specific gene called the Hydroxyacid oxidase 1 (HAO1) gene. By doing so, it aims to reduce the activity of an enzyme called glycolate oxidase in liver cells, which in turn decreases the production of oxalate.

The purpose of the study is to evaluate the safety and tolerability of ABO-101 in individuals with PH1. Participants in the study will receive ABO-101 through an intravenous infusion, which means the solution will be delivered directly into the bloodstream. The study will monitor participants over time to assess any side effects and to see how the body processes the treatment. Additionally, the study will measure changes in the levels of oxalate in the urine and other related substances in the blood to understand the treatment's impact.

This trial is an important step in exploring new treatment options for people with PH1, as current treatments are limited. By participating in this study, researchers hope to gather valuable information that could lead to better management of the condition in the future. The study will involve a series of visits and assessments to ensure the safety and well-being of all participants throughout the trial period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide signed informed consent. If you are below the legal age, consent must be obtained from your parent or legal representative, and you must provide assent as per local and national requirements.

    You must confirm your willingness to comply with the requirements of the trial.

  2. Step 2

    Initial assessment

    You will undergo an initial assessment to confirm your diagnosis of Primary Hyperoxaluria Type 1 (PH1). This includes genetic analysis to confirm mutations in the AGXT gene.

    Your medical history will be reviewed, including any history of kidney stones or related symptoms.

  3. Step 3

    Baseline measurements

    Baseline measurements will be taken, including 24-hour urine collections to measure urinary oxalate levels.

    Blood tests will be conducted to ensure normal liver function and kidney function, and to check platelet counts and other parameters.

  4. Step 4

    Medication administration

    You will receive the study medication, ABO-101, as a solution for intravenous infusion. The dosage and frequency will be determined by the study protocol.

    If you are taking pyridoxine (vitamin B6) for PH1, you must have been on a stable regimen for at least 90 days prior to receiving ABO-101 and continue this regimen during the study.

  5. Step 5

    Monitoring and follow-up

    You will be monitored for any treatment-emergent adverse events, including those related to ABO-101.

    Regular follow-up visits will be scheduled to assess changes in urinary oxalate excretion, plasma glycolate levels, and kidney function.

    Blood and urine samples will be collected periodically to measure drug concentrations and check for antibodies against the study medication.

  6. Step 6

    Completion of study

    Upon completion of the study, final assessments will be conducted to evaluate the overall effects of the treatment.

    You will be required to continue using effective contraception for a specified period after receiving the study medication, as outlined in the study protocol.

Who can join the trial?

21 criteria

  • Must have a confirmed diagnosis of Primary Hyperoxaluria Type 1 (PH1).
  • Must be able to follow directions and provide 24-hour urine collections.
  • If taking pyridoxine (vitamin B6) for PH1, must have been on a stable dose for at least 90 days before starting the study and be willing to continue this dose during the study.
  • Must meet certain laboratory test results:
    • AST, ALT, and total bilirubin levels should be within normal limits for age. If you have Gilbert’s syndrome, total bilirubin should be less than twice the normal limit.
    • eGFR (a measure of kidney function) should be at least 30 mL/min/1.73m².
    • Platelet count should be more than 100,000/mm³.
    • Normal results for PT, aPTT, INR, D-dimer, and fibrinogen tests.
    • Must not have received any experimental treatment for PH1 for a certain period before the study.
    • Must agree not to participate in another interventional trial during the screening period and for at least 6 months after receiving the study drug.
    • Must have a history of kidney stones or related symptoms, or a history of nephrocalcinosis (calcium deposits in the kidneys).
    • All available standard treatments for PH1 should have been considered.
    • Must weigh at least 40 kg (about 88 pounds).
    • For female participants:
      • If able to have children, must agree to use at least one highly effective method of contraception from the time of signing the consent form through 12 months after receiving the study drug.
      • Must be postmenopausal or surgically sterile for at least one month before screening.
      • For male participants: Must agree to use at least one highly effective method of contraception from the time of signing the consent form through 4 months after receiving the study drug, and not donate sperm during this time.
      • Must be able to provide informed consent. If under the legal age, consent must be obtained from a parent or legal representative, and the participant must agree to participate according to local laws.
      • Must be willing to follow the study requirements.
      • Age requirements:
        • Cohorts 1-3: Must be between 18 and 64 years old.
        • Cohort 4: Must be between 6 and 18 years old.
        • Must have documentation of PH1 through genetic testing confirming mutations in the AGXT gene.
        • Must have a certain level of urinary oxalate (a substance in urine) measured over 24 hours.

Who cannot join the trial?

9 criteria

  • Patients who do not have Primary Hyperoxaluria Type 1 (PH1) cannot participate. This is a specific medical condition that the study is focusing on.
  • Patients who are not within the specified age range cannot participate. The study is open to certain age groups only.
  • Patients who are not willing to follow the study procedures and requirements cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients who have allergies to any of the study medications or components cannot participate.
  • Patients who have a history of substance abuse or alcohol abuse cannot participate.
  • Patients who are unable to provide informed consent cannot participate. This means they must understand the study and agree to take part.
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Investigated drugs

ABO-101 is a medication being studied for its potential to help people with a condition called Primary Hyperoxaluria Type 1 (PH1). This condition causes the body to produce too much of a substance called oxalate, which can lead to kidney stones and other health problems. The goal of using ABO-101 in this trial is to see if it is safe and well-tolerated by patients, and to understand how it works in the body. Researchers are also looking at how effective it might be in reducing the symptoms of PH1. By participating in this trial, patients help scientists learn more about how ABO-101 could be used to treat this condition in the future.

What is already known about the treatment

ABO-101 – ABO-101 is administered as an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein. It is currently being studied in clinical trials, specifically in a Phase 1/2 study, to assess its safety and effectiveness for treating Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder. The main goal of ABO-101 is to reduce the production of oxalate, a substance that can accumulate and cause kidney damage in people with PH1. At the molecular level, ABO-101 works by delivering a healthy copy of the gene responsible for the disorder, helping the body to produce the enzyme needed to break down oxalate. It is classified as a gene therapy, a type of treatment that aims to correct or replace faulty genes.

Investigated diseases

Primary Hyperoxaluria Type 1 (PH1) – This is a rare genetic disorder that affects the liver's ability to produce a specific enzyme called alanine-glyoxylate aminotransferase. The lack of this enzyme leads to the overproduction of a substance called oxalate. Excess oxalate combines with calcium to form calcium oxalate, which can accumulate in the kidneys and other organs. Over time, this accumulation can lead to the formation of kidney stones and damage to the kidneys. As the disease progresses, it can cause kidney function to decline, leading to a condition known as renal failure. The buildup of oxalate can also affect other parts of the body, causing various complications.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-518144-20-00Protocol codeABO-101-101Estimated enrolment23 patientsSponsorArbor Biotechnologies Inc.

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This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).