Study on the Safety and Effects of Sparsentan for Children with Proteinuric Kidney Diseases

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What is this study about?

This clinical trial is focused on studying certain kidney diseases known as proteinuric glomerular diseases. These include conditions like Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), Immunoglobulin A Nephropathy (IgAN), Immunoglobulin A Vasculitis (IgAV), and Alport Syndrome (AS). The treatment being tested is a medication called Sparsentan, which is given as an oral suspension. The purpose of the study is to evaluate the safety and effectiveness of Sparsentan in children with these kidney diseases.

Participants in the study will receive Sparsentan once daily for a period of 108 weeks. During this time, researchers will monitor the safety of the medication and observe any changes in the levels of protein in the urine, which is a key indicator of kidney health. The study aims to understand how well Sparsentan works in reducing protein levels in the urine and how the body processes the medication over time.

The study will also look at how many participants experience a complete or partial remission of their kidney disease symptoms, such as a significant reduction in protein levels in the urine. Additionally, researchers will track any side effects that occur during the study, including any that might lead to stopping the treatment. This information will help determine the overall safety and potential benefits of Sparsentan for treating these specific kidney diseases in children.

1 joining the study

Upon joining the study, the participant or their parent/legal guardian must provide signed informed consent. The participant must also provide assent before any procedures begin.

2 initial assessment

An initial assessment is conducted to ensure eligibility. This includes checking that the participant has an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m² and a mean seated blood pressure within the normal range for their age and height.

3 medication administration

The participant will receive a medication called sparsentan in the form of an oral suspension. This medication is taken once daily.

4 treatment duration

The treatment with sparsentan will continue for a period of 108 weeks. During this time, the participant’s response to the medication will be monitored.

5 monitoring and assessments

Throughout the study, regular assessments will be conducted to monitor the participant’s health and the effects of the medication. This includes measuring changes in protein levels in the urine and other relevant health indicators.

6 completion of the study

At the end of the 108-week period, a final assessment will be conducted to evaluate the overall effects of the treatment.

Who Can Join the Study?

The person or their parent/legal guardian must be willing and able to sign a consent form, and the person must agree to participate before any tests are done.
The person must have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m². eGFR is a test that measures how well the kidneys are working.
The person must have a blood pressure that is within the normal range for their age, sex, and height.
For Population 1: The person must be a boy or girl who is at least 1 year old but younger than 18 years old at the start of the study.
For Population 1: The person must have a urine protein/creatinine ratio (UP/C) of at least 1.5 g/g at screening. UP/C is a test that measures the amount of protein in the urine. They must also have one of the following:
- A kidney biopsy showing specific patterns related to certain kidney diseases, and they must have protein in their urine despite treatment with certain medications.
- Documentation of a genetic mutation in a specific kidney cell protein related to certain kidney diseases. If they have this mutation, a kidney biopsy is not needed.
- A kidney biopsy showing a specific pattern with a medical history and symptoms that match a certain type of kidney damage.
For Population 2: The person must be a boy or girl who is at least 2 years old but younger than 18 years old at the start of the study.
For Population 2: The person must have a UP/C of 0.6 g/g or less at screening. They must also have one of the following:
- A kidney biopsy confirming certain kidney diseases.
- A diagnosis of a specific kidney disease through genetic testing, which involves certain mutations in specific genes.

Who Cannot Join the Study?

Patients with other serious health conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have had a recent major surgery.
Patients who are currently participating in another clinical trial.
Patients with a history of drug or alcohol abuse.
Patients who have an allergy to the study medication.
Patients with uncontrolled high blood pressure.
Patients with severe liver or kidney disease.
Patients who are unable to follow the study procedures.
Patients who have a history of certain heart conditions.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Hospital Universitario 12 De Octubre	Madrid	Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Uniwersytecki Szpital Dzieciecy W Krakowie	Cracow	Poland
Azienda Ospedaliera di Padova	Padua	Italy
Karolinska University Hospital	Huddinge	Sweden
Virgen del Rocío University Hospital	Sevilla	Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Stichting Radboud University Medical Center	Nijmegen	The Netherlands
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Unjdowzzgo Mytwojl Cqzmvc Hqufrnrylatubfacv	Hamburg	Germany
Uzclmawrud Hqyouyfe Ccbdfob	Cologne	Germany
Agylgdaxk Ucs	Amsterdam	The Netherlands
Fvdmvnkym Psjh Lh Igibxefbltpae Biroregrh Dqy Hudfaodq Uniohqbyucyvm Lm Pia	Madrid	Spain
Hohzmrih Vmix dpzgsfql	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	12.08.2021
Italy	Recruiting	12.08.2021
Poland	Recruiting	12.08.2021
Spain	Recruiting	12.08.2021
Sweden	Recruiting	12.08.2021
The Netherlands	Recruiting	12.08.2021

Trial locations

Investigated drugs:

Sparsentan

Sparsentan is an investigational medication being studied for its potential to treat certain kidney diseases that cause high levels of protein in the urine, known as proteinuric glomerular diseases. In this clinical trial, sparsentan is given as an oral suspension, which means it is a liquid form that is taken by mouth. The main goals of the trial are to evaluate how safe and well-tolerated sparsentan is for children with these kidney conditions and to see how it affects the levels of protein in their urine over a period of 108 weeks.

Investigated diseases:

Focal Segmental Glomerulosclerosis (FSGS) – A kidney disorder characterized by scarring in the glomeruli, which are tiny structures within the kidneys that filter waste from the blood. This scarring leads to protein leaking into the urine, a condition known as proteinuria. Over time, FSGS can cause the kidneys to lose their ability to filter blood effectively.

Minimal Change Disease (MCD) – A kidney disease that primarily affects children, causing nephrotic syndrome, which is characterized by high levels of protein in the urine, low levels of protein in the blood, and swelling. The disease is termed “minimal change” because the kidney tissue appears nearly normal under a regular microscope, but changes can be seen with an electron microscope.

Immunoglobulin A Nephropathy (IgAN) – A kidney disease that occurs when IgA, a type of antibody, builds up in the kidneys, leading to inflammation that can affect the kidneys’ ability to filter waste from the blood. This condition often progresses slowly over many years and can result in blood and protein in the urine.

Immunoglobulin A Vasculitis (IgAV) – A disease that causes small blood vessels to become inflamed and can affect the skin, joints, intestines, and kidneys. It is often characterized by a purplish rash, joint pain, and abdominal pain, and can lead to kidney issues similar to those seen in IgA nephropathy.

Alport Syndrome (AS) – A genetic condition characterized by kidney disease, hearing loss, and eye abnormalities. It affects the glomeruli in the kidneys, leading to progressive loss of kidney function and often results in blood and protein in the urine. The condition is caused by mutations in genes that affect the type IV collagen found in the kidneys, ears, and eyes.

Trial ID:

2023-505497-14-00

Protocol code:

RTRX-RE021-201

NCT ID:

NCT05003986

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effects of Sparsentan for Children with Proteinuric Kidney Diseases

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