Clinical Trials for Alport Syndrome
Currently, there are 5 ongoing clinical trials investigating new treatments for Alport syndrome, a genetic kidney disease. These studies are testing medications that may help slow disease progression and protect kidney function, taking place across several European countries and involving both children and adults with this condition.
Clinical trial locations
- Austria
- Belgium
- Czechia
- France
- Germany
- Study on Dapagliflozin for Slowing Kidney Disease in Adolescents and Young Adults with Alport Syndrome
- Study on the Safety and Effects of Sparsentan for Children with Proteinuric Kidney Diseases
- Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
- Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression
- Italy
- Lithuania
- Netherlands
- Poland
- Slovakia
- Spain
- Sweden
Study on Dapagliflozin for Slowing Kidney Disease in Adolescents and Young Adults with Alport Syndrome
This trial is testing whether Dapagliflozin, a medication normally used to manage blood sugar levels in diabetes, can help slow the progression of kidney disease in young people with Alport syndrome. The study focuses on adolescents aged 10 to 17 years and young adults aged 18 to 39 years who have early-stage chronic kidney disease.
Who can participate: Participants must have a confirmed diagnosis through genetic testing or kidney biopsy and show evidence of protein in their urine (albuminuria). Adolescents need a urine albumin-to-creatinine ratio of at least 300 mg/g, while adults need at least 500 mg/g. All participants must have stable kidney function and be on stable treatment with medications called RAS blockers for at least three months before joining.
Who cannot participate: The study excludes people without a confirmed diagnosis, those outside the specified age range, individuals with allergies to the medication, people currently in other clinical trials, those with serious interfering health conditions, and pregnant or breastfeeding women.
Study focus: The main goal is to determine whether Dapagliflozin can reduce the amount of protein in the urine and prevent worsening of kidney disease over a 48-week treatment period. Participants will take either Dapagliflozin or a placebo tablet daily, with regular check-ups to monitor kidney function and overall health. The study is double-blind, meaning neither participants nor researchers know who receives the actual medication.
Investigational drug: Dapagliflozin is a sodium-glucose co-transporter-2 inhibitor given as a 5 mg film-coated tablet taken orally once daily.
Study on the Safety and Effects of Sparsentan for Children with Proteinuric Kidney Diseases
This clinical trial evaluates Sparsentan for children with various kidney diseases that cause protein loss in the urine, including Alport syndrome, Focal Segmental Glomerulosclerosis, Minimal Change Disease, IgA Nephropathy, and IgA Vasculitis. The study runs for 108 weeks and aims to assess both safety and effectiveness.
Who can participate: Children aged 1 to 18 years in one group must have certain levels of protein in their urine and a confirmed diagnosis through kidney biopsy or genetic testing. A second group of children aged 2 to 18 years with lower protein levels and confirmed diagnoses can also join. All participants must have adequate kidney function (eGFR of at least 30 mL/min/1.73 m²) and normal blood pressure for their age and height. Informed consent from parents or guardians and assent from the child are required.
Who cannot participate: Children with other serious health conditions that might interfere with the study, those currently in another trial, pregnant or breastfeeding individuals, those with recent major surgery, people with drug or alcohol abuse history, those allergic to the study medication, individuals with uncontrolled high blood pressure, severe liver or kidney disease, certain heart conditions, or those unable to follow study procedures are excluded.
Study focus: The trial monitors how safe and well-tolerated Sparsentan is for children and measures changes in urine protein levels over time. Researchers also track how many participants achieve complete or partial remission of their symptoms and observe how the body processes the medication.
Investigational drug: Sparsentan is given as an oral suspension (liquid) taken once daily. It works by blocking specific receptors in the body to help reduce protein loss in urine. It is classified as an angiotensin receptor blocker and endothelin receptor antagonist.
Study on R3R01 for Patients with Alport Syndrome and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
This trial investigates R3R01, an oral tablet medication, for its safety and effectiveness in reducing protein in the urine for patients with either Alport syndrome or primary steroid-resistant Focal Segmental Glomerulosclerosis. The treatment period lasts 12 weeks with ongoing monitoring.
Who can participate: For Alport syndrome patients, both males and females aged 12 years and older (or 18 and older depending on the country) with confirmed genetic or biopsy diagnosis and a urine protein-to-creatinine ratio of at least 1.0 g/g can participate. For FSGS patients, those aged 12 to 75 years (or 18 to 75 depending on country) with biopsy-confirmed primary FSGS or genetic mutation, who have proven steroid resistance and protein levels between 3.5 and 12.0 g/g can join. All participants must have adequate kidney function (eGFR at least 45 mL/min/1.73m²) and be on stable doses of ACE inhibitors or ARBs for at least 4 weeks.
Who cannot participate: People without either diagnosis, those outside the specified age ranges, individuals unable to take oral medication, those with interfering medical conditions, pregnant or breastfeeding women, people currently in another trial, those with allergies to the study medication, and individuals unable to follow study procedures or attend visits are excluded.
Study focus: The study evaluates the safety and tolerability of R3R01 over 12 weeks while measuring its effects on reducing urine protein levels. Researchers also assess quality of life changes and how the body absorbs and processes the medication.
Investigational drug: R3R01 is an oral tablet taken for 12 weeks, currently under investigation for its potential to reduce proteinuria in kidney disease patients.
Study on the Safety and Effects of Setanaxib for Patients with Alport Syndrome
This trial examines Setanaxib, a film-coated tablet, comparing it to a placebo over 24 weeks to assess safety and tolerability in patients with Alport syndrome. The study includes regular health monitoring throughout the treatment period.
Who can participate: Males and females aged 12 to 50 years (or 18 to 50 in the EU) with genetically confirmed Alport syndrome who weigh at least 40 kg can participate. Participants must have elevated protein levels in their urine (measured twice at least 2 weeks apart) and be taking stable doses of specific blood pressure medications for at least 8 weeks. Blood pressure must be within acceptable ranges based on age. Women who can become pregnant must use effective birth control starting 4 weeks before the study and continuing for 90 days after the last dose, with negative pregnancy tests required. Men must use condoms and ensure their partners use birth control during the same timeframe.
Who cannot participate: People without confirmed Alport syndrome, those under 18 years old, pregnant or breastfeeding women, individuals in another trial within the last 30 days, those with allergies to similar medications, people with severe liver or heart problems, and those unable to follow study procedures are excluded.
Study focus: The study assesses the safety and tolerability of Setanaxib compared to placebo through regular health checks including heart rate, blood pressure, kidney function tests, blood tests, and hearing tests. Researchers aim to understand how the body processes the medication and its effects on symptoms.
Investigational drug: Setanaxib is a NOX1/4 inhibitor given as a film-coated tablet taken orally. It works by inhibiting certain enzymes thought to play a role in disease progression.
Study on the Safety of Vonafexor for Patients with Alport Syndrome at Risk of Progression
This clinical trial studies Vonafexor, an oral tablet medication, to assess its safety and tolerability in patients with Alport syndrome who are at risk of disease progression. The study uses a fixed dose-escalation approach, with some participants receiving a placebo.
Who can participate: Adults aged 18 to 55 years (or 16 and older in the United States) with confirmed Alport syndrome diagnosis (through clinical signs, family history, biopsy findings, or genetic testing) can participate. Participants must have kidney function measured by eGFR between 30 and less than 90 ml/min/1.73m² and increased albuminuria with a urine albumin-to-creatinine ratio of 300 mg/g or higher. Those taking ACE inhibitors, ARBs, or SGLT2 medications must be on stable doses for at least 60 days. Participants must test negative for hepatitis B, hepatitis C, and HIV, and sexually active individuals must agree to use two effective birth control methods during the study and for 6 weeks after.
Who cannot participate: People without confirmed Alport syndrome, those not at risk of disease progression, individuals under 18 years old (except in the United States where 16 is allowed), and vulnerable populations including pregnant women, children, or those unable to make decisions for themselves are excluded.
Study focus: The trial monitors safety and tolerability through regular check-ups including physical examinations and laboratory tests, both during treatment and after it ends. Researchers also measure Vonafexor levels in the blood to understand how the body processes the medication.
Investigational drug: Vonafexor is an oral tablet classified as a selective modulator that targets specific receptors to help reduce kidney damage. It is in the investigational stage for treating patients at risk of Alport syndrome progression.
Summary
The five ongoing clinical trials for Alport syndrome represent diverse approaches to slowing kidney disease progression. Geographically, Germany and France show the strongest concentration of trials, each hosting four of the five studies, followed by Spain with three trials. This suggests these countries have established research infrastructure for rare kidney diseases.
The trials test four different investigational medications: Dapagliflozin (a drug already approved for diabetes and being repurposed), Sparsentan (targeting multiple pathways), R3R01 (a novel compound), Setanaxib (an enzyme inhibitor), and Vonafexor (a selective modulator). All studies share a common focus on reducing proteinuria, the excess protein in urine that indicates kidney damage and predicts disease progression.
Age ranges vary considerably across trials. The Dapagliflozin study specifically targets adolescents and young adults aged 10 to 39 years, while the Sparsentan trial includes children as young as 1 year. The R3R01 and Setanaxib trials generally focus on older children and adults, with some accepting participants aged 12 and up. The Vonafexor study is limited to adults aged 18 to 55 years.
Most trials require participants to already be on stable doses of standard kidney-protective medications such as ACE inhibitors or ARBs before joining. This reflects the current standard of care for Alport syndrome and allows researchers to evaluate whether new treatments provide additional benefits beyond existing therapies. All studies emphasize safety monitoring alongside effectiveness, recognizing that medications must be well-tolerated for long-term use in this chronic condition.
