Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

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What is this study about?

This clinical trial is focused on studying two kidney diseases: Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis. The treatment being tested is a medication called R3R01, which is taken as a tablet. The purpose of the study is to evaluate how safe and tolerable this medication is for patients, as well as its effectiveness in reducing a condition called proteinuria, which is the presence of excess protein in the urine.

Participants in the study will take the medication R3R01 orally for a period of 12 weeks. During this time, researchers will monitor the safety and tolerability of the medication by checking for any side effects and changes in health indicators such as physical exams, vital signs, and lab tests. The study will also assess how well the medication works in reducing proteinuria in patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis.

The study aims to gather information on the quality of life of participants and how it changes from the beginning to the end of the treatment. Additionally, researchers will analyze how the body absorbs and processes the medication. The study will track the number of patients who show a complete or partial response to the treatment in terms of reducing proteinuria at various points during and after the treatment period.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current health status.

Eligibility criteria include stable medication doses and specific health parameters, such as kidney function and protein levels in urine.

2 medication administration

The study involves taking a medication called R3R01 in the form of a tablet.

The medication is taken orally for a duration of 12 weeks.

3 monitoring and evaluation

Throughout the 12-week period, regular monitoring is conducted to assess safety and tolerability. This includes checking for any side effects and changes in health indicators.

Evaluations focus on the medication’s effect on reducing protein levels in urine, which is a key measure of its effectiveness.

4 follow-up assessments

After completing the medication course, follow-up assessments are conducted to evaluate any changes in quality of life and health status.

These assessments help determine the long-term effects of the medication.

Who Can Join the Study?

All patients must be able to communicate well with the study team, understand and agree to follow all study requirements, and sign a written informed consent form.
For children to participate, one or both parents or legal guardians must sign a permission form, and children who can understand must also agree to participate by signing an assent form.
If a patient has received a COVID vaccination, the first study visit must be at least one week after the second or booster shot.
Patients who had COVID symptoms within 3 months before the study and are now symptom-free for the last 2 weeks but tested positive for COVID can be rescreened after at least two weeks if they are still positive at the first screening.
Female patients and female partners of male patients who can become pregnant must agree not to become pregnant during the study and for 90 days after the last dose of the study medication.
Male patients, including those who are sterilized, whose female partners can become pregnant, must agree to use male contraception (like condoms) from the time of signing the consent form until 90 days after the last dose of the study drug. They must inform the study team immediately if their partner becomes pregnant during the study.
Patients must have an estimated glomerular filtration rate (eGFR) of at least 45 mL/min/1.73m², which is a measure of kidney function.
Patients must be on a stable dose of ACE inhibitors (ACEi) or angiotensin receptor blockers (ARB) for at least 4 weeks before the study and continue this stable dose during the study.
For patients with Alport Syndrome (AS):

Both males and females with X-Linked AS and those with autosomal inherited AS can participate. In countries enrolling children, patients must be 12 years or older. In other countries, patients must be 18 years or older.
Patients must have a confirmed diagnosis of AS through genetic testing or a kidney biopsy. In the US, if the diagnosis is not confirmed, the study sponsor will provide genetic testing.
Patients must have a urine protein-to-creatinine ratio (UPCR) of at least 1.0 g/g, which measures protein levels in urine.

For patients with Focal Segmental Glomerulosclerosis (FSGS):

Both males and females can participate. In countries enrolling children, patients must be 12 to 75 years old. In other countries, patients must be 18 to 75 years old.
Patients must have primary FSGS, which means it has no identifiable cause and is confirmed by a kidney biopsy, or FSGS with a documented genetic mutation in a podocyte protein related to FSGS.
Patients must be steroid-resistant, meaning they did not achieve partial or complete remission or had adverse effects without acceptable benefits after at least 8 weeks of corticosteroid therapy for children and 12 weeks for adults.
Patients must have a UPCR between 3.5 g/g and 12.0 g/g.
Patients must have an eGFR greater than 45 mL/min/1.73m².
If patients are taking ACEi and/or ARB treatment, it must be at a stable dose for at least 28 days before joining the study and remain stable during the study.

Who Cannot Join the Study?

Patients who are not diagnosed with either Alport Syndrome (AS) or Primary Steroid-Resistant Focal Segmental Glomerulosclerosis (FSGS) cannot participate. These are specific kidney conditions.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Patients who are not able to take the study medication orally cannot participate. The medication is given by mouth.
Patients who have other medical conditions that might interfere with the study cannot participate. This is to ensure safety and accurate results.
Patients who are pregnant or breastfeeding cannot participate. This is to protect the health of the mother and baby.
Patients who are currently participating in another clinical trial cannot participate. This is to avoid interference with study results.
Patients who have a history of allergic reactions to the study medication cannot participate. This is to prevent any harmful reactions.
Patients who are unable to follow the study procedures or attend scheduled visits cannot participate. This is important for accurate data collection.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Universitaetsmedizin Goettingen	Goettingen	Germany
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Centre hospitalier universitaire de Liege	Liege	Belgium
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Stichting Radboud University Medical Center	Nijmegen	The Netherlands
Hopital Beaujon	Clichy	France
Clwakhjxa Uikayobmzryrah Siuwpbbjv	Woluwe-Saint-Lambert	Belgium
Asodyguvs Unt	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	27.06.2022
France	Not recruiting	27.06.2022
Germany	Not recruiting	27.06.2022
The Netherlands	Not recruiting	27.06.2022

Trial locations

Investigated drugs:

R3R01

R3R01 is a medication being studied for its potential to help patients with certain kidney conditions. It is taken by mouth and is being tested to see if it is safe and well-tolerated over a period of 12 weeks. The study is particularly interested in how well this medication can reduce protein levels in the urine of patients with Alport Syndrome and those with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis, both of which are conditions that affect the kidneys.

Investigated diseases:

Alport Syndrome – Alport Syndrome is a genetic disorder that primarily affects the kidneys, ears, and eyes. It is characterized by progressive kidney disease, hearing loss, and eye abnormalities. The condition is caused by mutations in genes responsible for the production of type IV collagen, a protein essential for the normal function of the basement membranes in these organs. Over time, individuals with Alport Syndrome may experience worsening kidney function, leading to chronic kidney disease. Hearing loss typically begins in childhood or adolescence and can progress to significant impairment. Eye changes may include abnormalities in the lens and retina, but they usually do not affect vision significantly.

Primary Steroid-Resistant Focal Segmental Glomerulosclerosis – Primary Steroid-Resistant Focal Segmental Glomerulosclerosis (FSGS) is a kidney disorder characterized by scarring in the glomeruli, the tiny filtering units within the kidneys. This condition leads to proteinuria, where excess protein is lost in the urine, and can result in nephrotic syndrome. Unlike other forms of FSGS, this type does not respond to steroid treatment, making it more challenging to manage. The disease can cause progressive kidney damage, eventually leading to chronic kidney disease. Symptoms may include swelling, particularly in the legs and around the eyes, due to fluid retention. The exact cause of primary steroid-resistant FSGS is not well understood, but it is believed to involve genetic and environmental factors.

Trial ID:

2024-512964-73-00

Protocol code:

R3R01-ASFSGS-201

NCT ID:

NCT05267262

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

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