Alport syndrome

At ENYO Pharma, patients with impaired kidney function are in the center of our discussions.

We are committed every day to develop new therapeutics for improving the quality of life of patients with Alport syndrome. Working closely with our patient advocacy partners, as key players as representatives of patients, families and caregivers, support our best strategy for clinical study development in Alport syndrome.

ALPESTRIA-1 clinical trial

We are aiming to preserve kidney function and reverse kidney damage with the investigational drug Vonafexor tested in our Phase 2 clinical study named “ALPESTRIA-1”.

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What is the ALPESTRIA-1 clinical study?

The Alport syndrome Efficacy and Safety Trial-1

• Objectives: The ALPESTRIA-1 study is a Phase 2 clinical study evaluating the safety, the tolerability and the benefit of three dose levels of the investigational drug Vonafexor on renal function and biomarkers in Alport syndrome patients.
• Design: The study is an open label and single arm study with a fixed dose-escalation. There is no placebo group (all subjects will receive study drug).
• Treatment: All participants will receive all three dose levels of Vonafexor as oral tablets once daily for 24 weeks then stop the treatment for 12 weeks in the follow-up period.
• Assessments: The study offers assessments with a mix of 5 site visits, 4 home visits and 4 phone calls, with urine and/or blood sampling.
• Enrollment: The study aims at enrolling 20 patients in the US and EU (France, Spain and Germany).

What is Vonafexor?

The investigational drug for ALPESTRIA-1 clinical study

• Drug: Vonafexor is a small molecule with strong fibrolytic and anti-inflammatory properties in both organs, kidney and liver. Learn more about Vonafexor
• Regulatory: Vonafexor was granted with Orphan Drug Designation (ODD) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) in 2023.

Can I participate in the ALPESTRIA-1 study?

I may be eligible if:

• Age: I am 18–55 years of age in EU or 16-55 years of age in the US
• Sex: I am a woman or a man
• Diagnosis: I have been diagnosed with Alport syndrome & it was confirmed with a genetic test
• Kidney function: I have a mild to moderate loss of kidney function
• Proteinuria/albuminuria: I have an increased proteinuria and/or albuminuria
• Treatment: I can be on ACEi, ARB or SGLT2i treatment

Study status and site locations

The study is conducted in both US and Europe

• Study status: RECRUITING
• Site locations: The study is currently conducted in the US and is open in France and Spain.

All study sites are displayed below. To get information on a specific site, please select on the list or click the pin in the map. Click on the +/- signs or scroll up/down to zoom in/out on the map.

Learn more about the study

• If you have any questions about the clinical study, please speak with your physician.
• For the US, please visit https://clinicaltrials.gov with NCT06425055 or Alport Syndrome Foundation website.
• For EU (France, Spain and Germany), please visit https://euclinicaltrials.eu with 2023-509638-20.

Patient advocacy partnership

Patient advocacy organizations support patients and families by providing educational resources, building community, raising awareness, providing access to information from research and clinical experts, amplifying the patient voice, creating opportunities for patients to learn from and support each other, and organizing patient participation in research.

Alport Syndrome Foundation

Alport Syndrome Foundation is a patient-led non-profit organization in the United States dedicated to improving the lives of those affected by Alport Syndrome.

About Alport syndrome

• Disease overview: Alport syndrome is a rare kidney disease based on an inherited defect in type IV collagen, a structural material required for kidney, eye, and ear function.
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  Primary symptom is hematuria (blood in the urine) early in life followed by proteinuria (presence of proteins in the urine), hypertension and oedema. Together, these symptoms lead to a progressive decline of kidney function and ultimately to end-stage renal disease (ESRD). Risk for disease progression depends on many factors including the genetic type and variant of patients. Individuals living with Alport syndrome often develop hearing loss and eye conditions.
• Mode of inheritance: There are three genetic types of Alport syndrome.
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  The most common is X-Linked Alport Syndrome (XLAS) based on COL4A5 collagen IV gene mutation where hematuria history (+/- proteinuria) or renal failure often occur in the family. XLAS represents approximately 74% of identified patient population. The other genetic types are Autosomal Dominant Alport syndrome (ADAS) and Autosomal Recessive Alport syndrome (ARAS) based on COL4A3 or COL4A4 collagen IV gene mutation, respectively. ARAS type represents 17% and ADAS type represents 9% of Alport syndrome patients.
• Incidence: Alport syndrome is the second most common inherited kidney disease, slightly behind autosomal-dominant polycystic kidney disease (ADPKD).
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  The global incidence of Alport syndrome is unknown as individuals are underdiagnosed or misdiagnosed. However, Alport syndromes is estimated to affect 1:5000 – 10,000 live births in the US (XLAS: 1/2000 births; ARAS: 1/10,000 births; ADAS: 1/100 births).
• Treatment: There is currently no cure for Alport syndrome and no currently available treatment options have demonstrated efficacy in helping patients avoid ESRD.
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  Current standard of care are therapeutics used for Chronic Kidney Disease (CKD), including angiotensin converting enzyme inhibitors (ACEis) or angiotensin receptor blockers (ARBs). These medications have negative side effects, are not tolerated by all patients, can exacerbate hyperkalemia, and do not ultimately lead to patients avoiding dialysis and kidney transplant.