RecruitingPHASE1, PHASE2INTERVENTIONAL

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

The redePHine study is investigating a new treatment called ABO-101 for a rare inherited kidney condition known as Primary Hyperoxaluria Type 1 (PH1). This condition can cause kidney stones and serious kidney damage. The main goals are to check if ABO-101 is safe, how the body uses it, and if it helps reduce harmful substance build-up in the body. The study has two main periods. Initially, it will test different doses in adults to find the best amount, then use that dose in children. After this first phase, participants will be monitored long-term to ensure continued safety. This research aims to find a new way to manage PH1 and improve the health of those affected.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Arbor Biotechnologies

Enrolment target

Start

16 Jun 2025

Estimated completion

01 Feb 2043

Primary Hyperoxaluria Type 1 (PH1)

What is this study about?

This research study, called redePHine, is looking into a new treatment named ABO-101 for a rare illness called Primary Hyperoxaluria Type 1, often shortened to PH1. PH1 is an inherited condition that causes the body to produce too much of a substance called oxalate. This extra oxalate can build up and lead to painful kidney stones, damage to the kidneys, and other health problems throughout the body.

The main aim of this study is to find out if ABO-101 is safe for people with PH1 and how well their bodies handle it. Researchers also want to see if this new medicine can help reduce the amount of harmful oxalate in the body. By understanding more about ABO-101, doctors hope to develop a new and effective way to treat PH1, which could greatly improve the lives of those living with this condition.

The study is split into two main parts. First, adult participants will receive different doses of ABO-101 to help scientists figure out the most suitable dose. Once this dose is identified, children with PH1 will then receive it. After these initial stages, all participants will be part of a long-term monitoring programme to ensure everyone's safety and to keep track of how the treatment is working over time. This careful approach helps make sure any new medicine is thoroughly tested.

Key takeaways

This study is for a new medicine (ABO-101) for Primary Hyperoxaluria Type 1 (PH1).
It aims to check the safety and how well the medicine works in both adults and children.
Participants will have regular clinic visits, tests, and long-term monitoring.
You must have a confirmed PH1 diagnosis through genetic testing to be eligible.
You can withdraw from the study at any time, for any reason.

Who may be eligible?

To join this study, your doctor would need to confirm that you have Primary Hyperoxaluria Type 1 (PH1) through a genetic test. That means they would look at your genes to find a specific change linked to PH1. You would also need to be aged between 6 and 64 years old.

There are also some medical checks needed. Your kidneys would need to be working at a certain level, and the amount of oxalate in your urine would need to be above a specific amount. If you weigh more than 90 kilograms, you unfortunately would not be able to take part.

You cannot join if you have other types of hyperoxaluria (type 2 or 3), or if you've ever had a liver or kidney transplant. If you are currently having dialysis treatment, or have used certain other oxalate-lowering medicines in the last two years, you wouldn't be able to participate either. Women who are pregnant, breastfeeding, or planning to be in the next year also cannot join this study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Primary Hyperoxaluria Type 1 (PH1) from a genetic test?
Am I between 6 and 64 years old?
Is my kidney function at least moderately good?
Are the oxalate levels in my urine above a certain amount?
Have I ever had a liver or kidney transplant?
Am I pregnant or breastfeeding, or planning to be in the next year?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will first go through an initial phase where doctors will try to find the best dose of the medicine. This will involve adults receiving different doses to see what works best. After this, children will receive this chosen dose. You will receive the new medicine, ABO-101, which will be given in a way that the study team will explain to you.

Throughout the study, you will have regular visits to the clinic. During these visits, doctors and nurses will carry out various tests. These might include blood tests, urine tests, and other checks to see how you are responding to the medicine and to monitor your general health. They will also look for any side effects.

After the initial treatment period, all participants will enter a long-term monitoring programme. This means you’ll continue to have check-ups to make sure the medicine is still safe and working well over time. The study will last for an extended period, which the study team will discuss in detail with you, including the total duration and how often your visits will be.

Potential risks and benefits

Taking part in a study like this might offer the potential benefit of trying a new medicine that could help manage your PH1, especially if current treatments aren't fully working for you. However, as ABO-101 is still being tested, there are also potential risks involved. These could include side effects from the medicine, which doctors will carefully monitor. The study team will explain these potential risks to you in detail. Remember, choosing to participate is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Nucleus Network
Verified postcode
Saint Paul, United States· Withdrawn
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
Verified postcode
Lyon, France· Not yet recruiting
Kindernierenzentrum Bonn
Verified postcode
Bonn, Germany· Not yet recruiting
Heidi Chaker
Verified postcode
Sfax, Tunisia· Recruiting
Queen Elizabeth Hospital Birmingham
Verified postcode
Birmingham, United Kingdom· Recruiting
Royal Free Hospital
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Primary Hyperoxaluria Type 1 (PH1)?

PH1 is a rare inherited condition where your body makes too much oxalate, a substance that can lead to kidney stones and kidney damage.

What is ABO-101?

ABO-101 is a new medicine being tested in this study to see if it can help reduce the high levels of oxalate in people with PH1.

Who can take part in this study?

Adults aged 18-64 and children aged 6-17 with diagnosed PH1 and certain kidney function levels, who meet specific health criteria, might be able to participate.

Will I get paid to be in the study?

The study information doesn't mention payment, but staff can tell you if there is any help with travel costs or other expenses.

What happens after the main treatment?

After the initial treatment, participants will join a long-term monitoring programme to continue checking their safety and how the medicine is working.

How to find out more

Daniel Ory, MD

arbortrials@arbor.bio 617-500-8941 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.