Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

This study is looking into a new medicine called R3R01 for people dealing with two types of kidney disease: Alport Syndrome and Focal Segmental Glomerulosclerosis (FSGS). These are conditions where the kidneys don't filter blood properly, leading to protein leaking into the urine. For this study, participants with Alport Syndrome will already be taking standard medications (ACE inhibitors or ARBs) but still have too much protein in their urine. The study's main goals are to check if the new medicine is safe, if people can tolerate it well, and if it helps reduce the amount of protein in their urine after 12 weeks. Researchers will also learn how the body processes the medicine.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

River 3 Renal Corp.

Enrolment target

Start

25 Jun 2024

Alport Syndrome (AS) and Primary Steroid-Resistent Focal Segmental Glomerulosclerosis (FSGS)

What is this study about?

This research study is investigating a new medication, called R3R01, for two specific kidney conditions: Alport Syndrome (AS) and a type of kidney scarring called Focal Segmental Glomerulosclerosis (FSGS). Both of these conditions can lead to serious kidney damage over time, including kidney failure. In both AS and FSGS, the tiny filters in the kidneys get damaged, allowing important proteins to leak out into the urine, which is a sign of kidney distress.

The study is called a 'Phase II' study, which means it's an early but important step in developing new medicines. At this stage, researchers are mainly focused on understanding if the medicine is safe for people to take, how well their bodies cope with it, and if it shows any early signs of helping with the kidney problems. For people with Alport Syndrome, they will already be on standard treatments but still have a lot of protein in their urine, and the study hopes to see if R3R01 can reduce this further.

We will be carefully monitoring participants through various tests, including checking their urine for protein levels, looking at their general health, and making sure the medicine isn't causing any serious side effects. We'll also be studying how the body absorbs, breaks down, and removes the medicine. The aim is to gather enough information to decide if R3R01 could be a promising new treatment option for these kidney conditions in the future.

Key takeaways

This study is for adults with Alport Syndrome or FSGS whose kidneys are leaking protein.
It's testing a new medicine called R3R01 to see if it's safe and helps reduce protein in urine.
Participants will receive the new medicine and have regular health checks.
The study helps us understand how the body uses the medicine.
This is an early-stage study to gather important information for future treatments.

Who may be eligible?

This study is looking for adults who are 18 years old or older. There is no upper age limit, so people of all adult ages are welcome to consider taking part.

Participants must have a confirmed diagnosis of either Alport Syndrome or a specific type of kidney scarring called Primary Steroid-Resistant Focal Segmental Glomerulosclerosis (FSGS).

If you have Alport Syndrome, you must also be currently taking standard medications (like ACE inhibitors or ARBs) but still have too much protein in your urine (this is called 'uncontrolled proteinuria'). This shows that your current treatment isn't fully managing the protein leak, and you might benefit from additional help.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of Alport Syndrome or Primary Steroid-Resistant FSGS?
If you have Alport Syndrome, are you currently taking ACE inhibitors or ARBs?
If you have Alport Syndrome, do you still have too much protein in your urine despite this treatment?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will be given the study medicine, R3R01, as part of the research. You will have regular visits to the clinic where the study team will carry out various checks. These will include physical examinations, measuring your vital signs (like blood pressure and heart rate), taking ECGs (heart tracings), and doing blood and urine tests. These tests help the researchers understand how your body is reacting to the medicine, if it's helping, and if there are any side effects. The study will measure the amount of protein in your urine after 12 weeks to see if the medicine is making a difference. There will also be follow-up appointments after you finish taking the medicine to ensure your health is stable. We do not have the exact number of visits or the total duration, but research studies typically involve multiple visits over several months.

Potential risks and benefits

Participating in a study like this may offer potential benefits, such as receiving a new investigational medication that might help your kidney condition where current treatments aren't fully effective. You will also receive close medical attention and monitoring from a specialist team. However, there are potential risks, as any new medicine can cause side effects that are not yet fully known. These side effects could be mild or, in rare cases, more serious. All risks will be fully explained before you decide to join. It's important to remember that joining the study is entirely voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (4)

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France
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Belgium
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Netherlands
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Germany

Common questions

What is Alport Syndrome?

Alport Syndrome is a genetic kidney disease that damages the tiny filters in your kidneys, leading to protein and blood in your urine over time.

What is Focal Segmental Glomerulosclerosis (FSGS)?

FSGS is a kidney disease where parts of the kidney's filtering units develop scarring, causing protein to leak into the urine.

What does a 'Phase II' study mean?

A Phase II study is an early stage of testing a new medicine. It focuses on finding out if the medicine is safe, if people can tolerate it, and if it shows early signs of working.

What is 'uncontrolled proteinuria'?

This means there's still too much protein leaking into your urine, even when you're already taking standard medications for your kidney condition.

Will I get the new medicine or a placebo?

This study is 'open-label', which means everyone who enrolls will receive the new investigational medicine, R3R01. There is no placebo group in this study.